| 1 | RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome) | Not yet recruiting | No Results Available | Mucopolysaccharidosis Type II (MPS II) | Genetic: RGX-121 | | https://ClinicalTrials.gov/show/NCT04571970 | ICM AAV9 |
| 2 | A Treatment Extension Study of Mucopolysaccharidosis Type IIIB | Active, not recruiting | No Results Available | Mucopolysaccharidosis Type IIIB|MPS III B | Drug: AX 250 | Children's Hospital and Research Center Oakland, Oakland, California, United States|Fundación Cardio Infantil - Instituto de CardiologÃa, Bogotá,, Colombia|University Medical Center Hamburg-Eppendorf, Hamburg, Germany|Complexo Hospitalario Universitario de Santiago (CHUS) - Hospital ClÃnico Universitario, Santiago de Compostela, Spain|MacKay Memorial Children's Hospital, Taipei, Taiwan|Gazi University Faculty of Medicine, Ankara, Turkey|Great Ormond Street Hospital For Children, NHS Foundation Trust, London, United Kingdom | https://ClinicalTrials.gov/show/NCT03784287 | ICV ERT |
| 3 | RGX-111 Gene Therapy in Patients With MPS I | Recruiting | No Results Available | Mucopolysaccharidosis Type I (MPS I) | Genetic: RGX-111 | Children's Hospital of Orange County, Orange, California, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States|University of Pennsylvania, Philadelphia, Pennsylvania, United States | https://ClinicalTrials.gov/show/NCT03580083 | ICM AAV9 |
| 4 | Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB | Recruiting | No Results Available | Mucopolysaccharidosis Type 3 B | Biological: rAAV9.CMV.hNAGLU | Nationwide Children's Hospital, Columbus, Ohio, United States|Armand-Trousseau Hospital, Paris, France|University Hospital Hamburg-Eppendorf, Hamburg, Germany|Hospital Clinico Universitario de Santiago, Santiago De Compostela, Spain | https://ClinicalTrials.gov/show/NCT03315182 | IV AAV9 |
| 5 | A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB) | Recruiting | No Results Available | Mucopolysaccharidosis Type IIIB | | UCSF Benioff Children's Hospital Oakland, Oakland, California, United States|Hospital Universitario Austral, Pilar, Argentina|Murdoch Childrens Research Institute and Royal Children's Hospital, Melbourne, Victoria, Australia|Medical Genetics Service/HCPA, Department of Genetics/UFRGS, Pôrto Alegre, Rio Grande Do Sul, Brazil|Fundación Cardio Infantil - Instituto de CardiologÃa, Bogotá, Colombia|University Medical Center Hamburg-Eppendorf, Hamburg, Germany|Hospital ClÃnico Universitario de Santiago, Santiago de Compostela, A Coruña, Spain|MacKay Memorial Children's Hospital, Taipei, Taiwan|Gazi University Faculty of Medicine, Ankara, Turkey | https://ClinicalTrials.gov/show/NCT03227042 | |
| 6 | Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) | Active, not recruiting | No Results Available | Mucopolysaccharidosis Type IIIA | Drug: LYS-SAF302 | CHOC Children's, Orange, California, United States|University of Minnesota, Minneapolis, Minnesota, United States|Weill Cornell Medical College, New York, New York, United States|Baylor college of medicine / Texas children's hospital, Houston, Texas, United States|Armand Trousseau Public Hospital, Paris, France|University Medical Center Hamburg-Eppendorf, Hamburg, Germany|Amsterdam UMC, Amsterdam, Netherlands|Great Ormond Street Hospital, London, United Kingdom | https://ClinicalTrials.gov/show/NCT03612869 | IC rhAAV10 |
| 7 | Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIIA | Recruiting | No Results Available | Mucopolysaccharidosis Type IIIA | Drug: Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene | Manchester University NHS Foundation Trust, Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT04201405 | OCT-201 |
| 8 | Respiratory Cathepsins, Proteases Inhibitors and Glycosaminoglycans (GAG) in Mucopolysaccharidosis | Not yet recruiting | No Results Available | Mucopolysaccharidoses | Other: Sputum|Other: Tracheal aspiration | Metabolic Disease Competence Centre - Medical Genetics Department - University Hospital, Angers, Angers, France|Metabolic Disease Reference Centre - Medical Genetics Department - University Hospital, Bordeaux, Bordeaux, France|Metabolic Disease Competence Centre - Medical Genetics Department - University Hospital, Brest, Brest, France|Metabolic Disease Competence Centre - Pediatrics Department - University Hospital, Rennes, Rennes, France|Metabolic Disease Reference Centre - Pediatrics Department - University Hospital, Toulouse, Toulouse, France|Metabolic Disease Reference Centre - Pediatrics Department - University Hospital, Tours, Tours, France|Pediatric Resuscitation Unit - Universty Hospital, Tours, Tours, France | https://ClinicalTrials.gov/show/NCT04112602 | |
| 9 | RGX-121 Gene Therapy in Patients With MPS II (Hunter Syndrome) | Recruiting | No Results Available | Mucopolysaccharidosis Type II (MPS II) | Genetic: RGX-121 | University of California San Francisco, Benioff Children's Hospital, Oakland, California, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States|Children's Hospital of Pittsburgh - UPMC: Program for Neurodevelopment in Rare Disorders, Pittsburgh, Pennsylvania, United States|Hospital de Clinicas de Porto Alegre, Porto Alegre, RS, Brazil | https://ClinicalTrials.gov/show/NCT03566043 | |
| 10 | An Extension Study of JR-171-101 Study in Patients With MPS I | Not yet recruiting | No Results Available | Mucopolysaccharidosis I | Drug: JR-171 | | https://ClinicalTrials.gov/show/NCT04453085 | |
| 11 | Assessment of Factors That Affected Respiratory Parameters in Mucopolysaccharidoses Patients | Completed | No Results Available | Mucopolysaccharidoses | Diagnostic Test: functional capacity | Hacettepe University, Ankara, Turkey | https://ClinicalTrials.gov/show/NCT04491747 | |
| 12 | Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project) | Terminated | No Results Available | Mucopolysaccharidosis (MPS) | | Children's Hospital Los Angeles, Los Angeles, California, United States|Nemours Children's Clinic, Jacksonville, Florida, United States|Children's Hospital and Clinic, Minneapolis, Minnesota, United States|Saint Louis Children's Hospital, Saint Louis, Missouri, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States|Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, United States|Intermountain Ear Nose and Throat Center, Salt Lake City, Utah, United States|Montreal Children's Hospital, Montreal, Quebec, Canada|Ospedale San Gerardo, Monza, Italy|Ospedale Pediatrico Bambino Gesù, Passoscuro, Italy|Christus Muguerza del Parque, S.A. de C.V., Chihuahua, Mexico|Hospital de Cruces, San Vicente de Baracaldo, Spain|Hacettepe Universitesi Tip Fakultesi Hastanesi, Ankara, Turkey|Royal Manchester Children's Hospital, Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT02095015 | |
| 13 | Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II | Active, not recruiting | No Results Available | Mucopolysaccharidosis II|MPS II | Biological: SB-913 | UCSF Benioff Children's Hospital Oakland, Oakland, California, United States|Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States|NYU School of Medicine, Neurogenetics Division, New York, New York, United States|University of North Carolina, Chapel Hill, North Carolina, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States | https://ClinicalTrials.gov/show/NCT03041324 | |
| 14 | Open-label Study of Anakinra in MPS III | Active, not recruiting | No Results Available | Mucopolysaccharidosis III | Biological: anakinra | The Lundquist Institute at Harbor-UCLA Medical Center, Torrance, California, United States | https://ClinicalTrials.gov/show/NCT04018755 | |
| 15 | A Phase â…¢ Study of JR-141 in Patients With Mucopolysaccharidosis II | Not yet recruiting | No Results Available | Mucopolysaccharidosis II | Drug: JR-141|Drug: Idursulfase|Drug: JR-141 or Idursulfase | | https://ClinicalTrials.gov/show/NCT04573023 | |
| 16 | Mucopolysaccharidosis Type II Observational | Not yet recruiting | No Results Available | Mucopolysaccharidosis II | Other: Observational | | https://ClinicalTrials.gov/show/NCT04591834 | |
| 17 | A Treatment Study of Mucopolysaccharidosis Type IIIB | Completed | No Results Available | MPS III B|Mucopolysaccharidosis Type IIIB | Drug: AX 250 | Children's Hospital Oakland, Oakland, California, United States|Fundación Cardio Infantil - Instituto de CardiologÃa, Bogotá, Colombia|University Medical Center Hamburg Eppendorf, Department of Pediatrics, Hamburg, Germany|Hospital Clinico Universitario de Santiago, Santiago de Compostela, Spain|Mackay Memorial Hospital, Taipei, Taiwan|Gazi Ãœniversitesi Tıp Fakültesi, Ankara, Turkey|Somers Clinical Research Facility, Great Ormond Street Hospital, London, United Kingdom | https://ClinicalTrials.gov/show/NCT02754076 | ICV ERT |
| 18 | An Extension Study of JR-141 in Patients With Mucopolysaccharidosis II | Enrolling by invitation | No Results Available | Mucopolysaccharidosis II | Drug: JR-141 | Fukui Clinical site, Fukui, Japan|Fukuoka Clinical site 2, Fukuoka, Japan|Fukuoka Clinical site, Fukuoka, Japan|Gifu Clinical site, Gifu, Japan|Hokkaido Clinical site, Hokkaido, Japan|Kananagawa Ckinical site, Kanagawa, Japan|Kumamoto Clinical site, Kumamoto, Japan|Okayama Clinical site, Okayama, Japan|Okayama Clinical site 2, Okayama, Japan|Okinawa Clinical site, Okinawa, Japan|Osaka Clinical site 3, Osaka, Japan|Osaka Clinical site 2, Osaka, Japan|Osaka Clinical site, Osaka, Japan|Saitama Clinical site, Saitama, Japan|Shizuoka Clinical site, Shizuoka, Japan|Shizuoka Clinical site 2, Shizuoka, Japan|Tochigi Clinical site, Tochigi, Japan|Tokyo Clinical site, Tokyo, Japan|Tottori Clinical site, Tottori, Japan | https://ClinicalTrials.gov/show/NCT04348136 | |
| 19 | A Study of JR-171 in Patients With Mucopolysaccharidosis I | Not yet recruiting | No Results Available | Mucopolysaccharidosis I | Drug: JR-171 | | https://ClinicalTrials.gov/show/NCT04227600 | |
| 20 | A Study in MPS VI to Assess Safety and Efficacy of Odiparcil | Completed | No Results Available | Mucopolysaccharidosis VI | Drug: Odiparcil|Other: Placebo | Hôpital Femme-Mère-Enfant, Bron, France|Villa Metabolica, Mainz, Germany|Centro Hospitalar S. João, Porto, Portugal|Royal Free Hospital, London, United Kingdom | https://ClinicalTrials.gov/show/NCT03370653 | |
| 21 | Gene Therapy in Patients With Mucopolysaccharidosis Disease | Recruiting | No Results Available | Mucopolysaccharidosis Type VI | Biological: AAV2/8.TBG.hARSB | Federico II University, Napoli, Naples, Italy|Erasmus Medical Center, Rotterdam Center for Lysosomal and Metabolic disease, Rotterdam, Netherlands|Children's Hospital Hacettepe University, Ankara, Turkey | https://ClinicalTrials.gov/show/NCT03173521 | |
| 22 | Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS I | Completed | No Results Available | Mucopolysaccharidosis I | Drug: AGT-181 | HCPA - Hospital das Clinicas de Porto Alegre, Porto Alegre, RS, Brazil | https://ClinicalTrials.gov/show/NCT03053089 | |
| 23 | An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of Study Drug UX003 Recombinant Human Beta-glucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) | Completed | Has Results | Mucopolysaccharidosis Type 7 | Drug: UX003 | Manchester Academic Health Science Centre, Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT01856218 | |
| 24 | Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant | Recruiting | No Results Available | Mucopolysaccharidosis IH | Genetic: Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the α-L-iduronidase cDNA, in their final formulation medium. | Ospedale San Raffaele, Milano, Italy | https://ClinicalTrials.gov/show/NCT03488394 | |
| 25 | A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB) | Completed | No Results Available | Mucopolysaccharidosis Type IIIB|Mucopolysaccharidosis Type 3 B|MPS III B|MPS 3 B | | Children's Hospital and Research Center Oakland, Oakland, California, United States|Melbourne Children's Trials Centre, Melbourne, Victoria, Australia|Fundacion Cardioinfantil-Instituto de Cardiologia, Bogota, Colombia|University Medical Center Hamburg Eppendorf, Department of Pediatrics, Hamburg, Germany|Hospital Clinico Universitario de Santiago, Santiago de Compostela, Spain|MacKay Memorial Children's Hospital, Taipei, Taiwan|Gazi University Faculty of Medicine, Ankara, Turkey|Somers Clinical Research Facility, Great Ormond Street Hospital, London, United Kingdom | https://ClinicalTrials.gov/show/NCT02493998 | |
| 26 | An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II | Enrolling by invitation | No Results Available | Mucopolysaccharidosis II | Drug: JR-141 | Grupo de Pesquisa ClÃnica em Genética Médica - HCPA, Porto Alegre, Brazil|Igeim - Unifesp, São Paulo, Brazil | https://ClinicalTrials.gov/show/NCT03708965 | |
| 27 | Mucopolysaccharidosis Type II Natural History | Active, not recruiting | No Results Available | Mucopolysaccharidosis II | | Children's Hospital of Pittsburgh - UPMC: Program for Neurodevelopment in Rare Disorders, Pittsburgh, Pennsylvania, United States|Hospital de Clinicas de Porto Alegre, Porto Alegre, RS, Brazil|Manchester Centre for Genomic Medicine, Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT03529786 | |
| 28 | A Study of JR-141 in Patients With Mucopolysaccharidosis II | Active, not recruiting | No Results Available | Mucopolysaccharidosis II | Drug: JR-141 | Fukui Clinical site, Fukui, Japan|Fukuoka Clinical site 2, Fukuoka, Japan|Fukuoka Clinical site, Fukuoka, Japan|Gifu Clinical site, Gifu, Japan|Hokkaido Clinical site, Hokkaido, Japan|Kanagawa Clinical site, Kanagawa, Japan|Kumamoto Clinical site, Kumamoto, Japan|Okayama Clinical site, Okayama, Japan|Okayama Clinical site 2, Okayama, Japan|Okinawa Clinical site, Okinawa, Japan|Osaka Clinical site 3, Osaka, Japan|Osaka Clinical site 2, Osaka, Japan|Osaka Clinical site, Osaka, Japan|Saitama Clinical site, Saitama, Japan|Shizuoka Clinical site, Shizuoka, Japan|Shizuoka Clinical site 2, Shizuoka, Japan|Tochigi Clinical site, Tochigi, Japan|Tokyo Clinical site, Tokyo, Japan|Tottori Clinical site, Tottori, Japan | https://ClinicalTrials.gov/show/NCT03568175 | |
| 29 | A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102 | Recruiting | No Results Available | Mucopolysaccharidosis III-A | Biological: ABO-102 | Nationwide Children's Hospital, Columbus, Ohio, United States|Women's and Children's Hospital, North Adelaide, South Australia, Australia|Hospital ClÃnico Universitario de Santiago, Santiago De Compostela, Spain | https://ClinicalTrials.gov/show/NCT04360265 | |
| 30 | Mucopolysaccharidosis VII Disease Monitoring Program | Recruiting | No Results Available | Mucopolysaccharidosis VII|MPS VII|MPS 7|Sly Syndrome | Other: No Intervention | Children's Hospital of Orange County, Orange, California, United States|Children's National Health System, Washington, District of Columbia, United States|Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States|New York University Langone Medical Center, New York, New York, United States|Laboratorio de Neuroquimica Dr. N.A. Chamoles S.R.L., Buenos Aires, Argentina|Hospital de ClÃnicas de Porto Alegre, Porto Alegre, Rio Grande Do Sul, Brazil|Centre Hospitalier Universitaire La Timone, Marseille, Provence Alpes Cote D'Azur, France|Centro Hospitalar do Porto, Porto, Portugal|Hospital Universitario Virgen del RocÃo Pabellón Infantil, Sevilla, Spain | https://ClinicalTrials.gov/show/NCT03604835 | |
| 31 | A Study of JR-141 in Patients With Mucopolysaccharidosis II | Completed | No Results Available | Mucopolysaccharidosis II | Drug: JR-141 | Grupo de Pesquisa ClÃnica em Genética Médica - HCPA, Porto Alegre, Brazil|Igeim - Unifesp, São Paulo, Brazil | https://ClinicalTrials.gov/show/NCT03359213 | |
| 32 | Natural History Study of Patients With MPS IIIA | Unknown status | No Results Available | Mucopolysaccharidosis IIIA | | Hospital de Clinicas de Porto Alegre, Porto Alegre, Brazil|Armand Trousseau Public Hospital, Paris, France|University Medical Center Hamburg-Eppendorf, Hamburg, Germany|Academic Medical Center, Emma Children's Hospital, Amsterdam, Netherlands|Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom | https://ClinicalTrials.gov/show/NCT02746341 | |
| 33 | Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I | Unknown status | No Results Available | Mucopolysaccharidosis I | Drug: AGT-181 (HIRMAb-IDUA) | Children's Hospital Oakland, Oakland, California, United States|Children's Hospital of Orange County, Orange, California, United States|Emory Healthcare, Decatur, Georgia, United States|Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States|Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, United States|University of Utah Hospital, Salt Lake City, Utah, United States | https://ClinicalTrials.gov/show/NCT02371226 | |
| 34 | A Cross-specialty Collaboration Platform for Mucopolysaccharidosis Confirmative Diagnosis | Not yet recruiting | No Results Available | Mucopolysaccharidoses | | | https://ClinicalTrials.gov/show/NCT03017677 | |
| 35 | Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB) | Completed | Has Results | Mucopolysaccharidosis IIIB | Drug: SBC-103 | Minneapolis, Minnesota, United States|Pittsburgh, Pennsylvania, United States|Barcelona, Spain|Birmingham, United Kingdom | https://ClinicalTrials.gov/show/NCT02324049 | |
| 36 | A Study of JR-141 in Patients With Mucopolysaccharidosis Type II | Completed | No Results Available | Mucopolysaccharidosis II | Drug: JR-141 | Gifu Clinical site, Gifu, Japan|Fukuoka Clinical site, Kurume, Japan|Tokyo clinical site1, Minato, Japan|Osaka Clinical site1, Osaka, Japan|Saitama Clinical site, Saitama, Japan|Tokyo clinical site2, Setagaya, Japan|Osaka Clinical site2, Suita, Japan|Tottori Clinical site, Yonago, Japan | https://ClinicalTrials.gov/show/NCT03128593 | |
| 37 | Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I | Completed | No Results Available | Mucopolysaccharidosis I | Drug: AGT-181 | HCPA - Hospital das Clinicas de Porto Alegre, Porto Alegre, RS, Brazil | https://ClinicalTrials.gov/show/NCT03071341 | |
| 38 | Natural History Studies of Mucopolysaccharidosis III | Completed | No Results Available | Mucopolysaccharidosis Type IIIA|Mucopolysaccharidosis Type IIIB | Procedure: Lumbar puncture|Device: Magnetic Resonance Imaging (MRI) of the brain | Nationwide Children's Hospital, Columbus, Ohio, United States | https://ClinicalTrials.gov/show/NCT02037880 | |
| 39 | Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects With MPS I | Active, not recruiting | No Results Available | MPS I | Biological: SB-318 | UCSF Benioff Children's Hospital Oakland, Oakland, California, United States | https://ClinicalTrials.gov/show/NCT02702115 | |
| 40 | An Open-Label Treatment Protocol With UX003 rhGUS Enzyme Replacement Therapy for an Advanced Stage MPS 7 Patient | No longer available | No Results Available | Mucopolysaccharidosis Type 7 | Drug: UX003 | Cohen Children's Medical Center, New Hyde Park, New York, United States | https://ClinicalTrials.gov/show/NCT02097251 | |
| 41 | MRS to Determine Neuroinflammation and Oxidative Stress in MPS I | Completed | No Results Available | Mucopolysaccharidosis Type I | | University of Minnesota, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT03576729 | |
| 42 | Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients | Completed | Has Results | Mucopolysaccharidosis (MPS) | Biological: Recombinant human heparan N-sulfatase (rhHNS) | Emma Children's Hospital, Academic Medical Center, Amsterdam, Netherlands|St. Mary's Hospital, Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT01155778 | |
| 43 | Longitudinal Study of Bone Disease in Children With Mucopolysaccharidoses (MPS) I, II, and VI | Completed | No Results Available | Mucopolysaccharidoses | | Children's Hospital & Research Center Oakland, Oakland, California, United States|Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, California, United States|University of Minnesota, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT01521429 | |
| 44 | An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. | Terminated | Has Results | Sanfilippo Syndrome|Mucopolysaccharidosis (MPS) | Drug: HGT-1410 | University of Minnesota, Minneapolis, Minnesota, United States|University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States|Chu Bicetre, Le Kremlin-Bicêtre, Paris, France|Universitätsklinikum Hamburg Eppendorf, Hamburg, Germany|Azienda Socio Sanitaria Territoriale - Asst di Monza, Monza, Italy|Academisch Medisch Centrum Amsterdam, Amsterdam, Netherlands|Hospital Universitario Vall D'hebron - Ppds, Barcelona, Spain|Great Ormond Street Hospital, London, United Kingdom | https://ClinicalTrials.gov/show/NCT02350816 | |
| 45 | Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis | Unknown status | No Results Available | Mucopolysaccharidosis | Drug: Conditioning regimen|Drug: Graft-versus-host disease (GVHD) prophylaxis|Procedure: Stem Cell Transplantation | Hematology-Oncology & SCT Research Center, Teharn, Tehran, Iran, Islamic Republic of | https://ClinicalTrials.gov/show/NCT01238328 | |
| 46 | Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome | Completed | No Results Available | Mucopolysaccharidosis II | Drug: AGT-182 | Children's Hospital Oakland, Oakland, California, United States|Children's Hospital of Orange County, Orange, California, United States|Emory University, Decatur, Georgia, United States|ZKJM MC University of Mainz, Mainz, Germany|Institute of Human Genetics, National Inst of Health, University of the Philippines, Manila, Philippines | https://ClinicalTrials.gov/show/NCT02262338 | |
| 47 | A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7) | Completed | Has Results | MPS 7|Sly Syndrome|Mucopolysaccharidosis|MPS VII | Drug: UX003|Other: Placebo | Children's Hospital Oakland, Oakland, California, United States|Children's Hospital of Orange County, Orange, California, United States|Miami Children's Hospital, Miami, Florida, United States|University of Minnesota, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT02230566 | |
| 48 | Mucopolysaccharidosis I (MPS I) Registry | Recruiting | No Results Available | Mucopolysaccharidosis I (MPS I) | | Investigational Site Number 840109, Little Rock, Arkansas, United States|Investigational Site Number 840108, Los Angeles, California, United States|Investigational Site Number 840042, Sacramento, California, United States|Investigational Site Number 840117, San Bernardino, California, United States|Investigational Site Number 840047, New Haven, Connecticut, United States|Investigational Site Number 840067, Washington, District of Columbia, United States|Investigational Site Number 840083, Gainesville, Florida, United States|Investigational Site Number 840112, Jacksonville, Florida, United States|Investigational Site Number 840011, Chicago, Illinois, United States|Investigational Site Number 840027, Indianapolis, Indiana, United States|Investigational Site Number 840032, Iowa City, Iowa, United States|Investigational Site Number 840030, Louisville, Kentucky, United States|Investigational Site Number 840044, Baltimore, Maryland, United States|Investigational Site Number 840062, Boston, Massachusetts, United States|Investigational Site Number 840093, Boston, Massachusetts, United States|Investigational Site Number 840002, Springfield, Massachusetts, United States|Investigational Site Number 840107, Ann Arbor, Michigan, United States|Investigational Site Number 840114, Minneapolis, Minnesota, United States|Investigational Site Number 840100, Saint Louis, Missouri, United States|Investigational Site Number 840090, Helena, Montana, United States|Investigational Site Number 840084, Omaha, Nebraska, United States|Investigational Site Number 840098, Camden, New Jersey, United States|Investigational Site Number 840101, Hackensack, New Jersey, United States|Investigational Site Number 840099, Morristown, New Jersey, United States|Investigational Site Number 840016, New Brunswick, New Jersey, United States|Investigational Site Number 840057, Paterson, New Jersey, United States|Investigational Site Number 840102, Manhasset, New York, United States|Investigational Site Number 840040, New York, New York, United States|Investigational Site Number 840110, New York, New York, United States|Investigational Site Number 840105, Rochester, New York, United States|Investigational Site Number 840052, Syracuse, New York, United States|Investigational Site Number 840037, Durham, North Carolina, United States|Investigational Site Number 840041, Cincinnati, Ohio, United States|Investigational Site Number 840048, Cleveland, Ohio, United States|Investigational Site Number 840091, Columbus, Ohio, United States|Investigational Site Number 840094, Dublin, Ohio, United States|Investigational Site Number 840080, Portland, Oregon, United States|Investigational Site Number 840034, Philadelphia, Pennsylvania, United States|Investigational Site Number 840023, Pittsburgh, Pennsylvania, United States|Investigational Site Number 840053, Providence, Rhode Island, United States|Investigational Site Number 840055, Greenville, South Carolina, United States|Investigational Site Number 840078, Charlottesville, Virginia, United States|Investigational Site Number 840072, Norfolk, Virginia, United States|Investigational Site Number 840115, Tacoma, Washington, United States|Investigational Site Number 840054, Milwaukee, Wisconsin, United States|Investigational Site Number 153130, Buenos Aires, Argentina|Investigational Site Number 153032, Ciudad Autónoma de Buenos Aires, Argentina|Investigational Site Number 152096, Brisbane, Australia|Investigational Site Number 151069, Westmead, Australia|Investigational Site Number 150944, Brussel, Belgium|Investigational Site Number 150517, Bruxelles, Belgium|Investigational Site Number 151668, Leuven, Belgium|Investigational Site Number 154008, Roeselare, Belgium|Investigational Site Number 076007, Campinas, Brazil|Investigational Site Number 076001, Porto Alegre, Brazil|Investigational Site Number 076004, Rio de Janeiro, Brazil|Investigational Site Number 076002, São Paulo, Brazil|Investigational Site Number 124020, Halifax, Canada|Investigational Site Number 124022, Moncton, Canada|Investigational Site Number 152003, Los Angeles, Chile|Investigational Site Number 152002, Punta Arenas, Chile|Investigational Site Number 152001, Talcahuano, Chile|Investigational Site Number 170001, Bogotá, Colombia|Investigational Site Number 150659, Praha 2, Czechia|Investigational Site Number 151229, København Ø, Denmark|Investigational Site Number 818002, Cairo, Egypt|Investigational Site Number 818001, Cairo, Egypt|Investigational Site Number 250001, BRON Cedex, France|Investigational Site Number 250005, Paris Cedex 12, France|Investigational Site Number 250003, Paris, France|Investigational Site Number 250006, Vandoeuvre Les Nancy Cedex, France|Investigational Site Number 344001, Kowloon, Hong Kong|Investigational Site Number 356001, Kolkata, India|Investigational Site Number 356002, Vellore, India|Investigational Site Number 360001, Jakarta, Indonesia|Investigational Site Number 380002, Ancona, Italy|Investigational Site Number 380004, Cusano Milanino, Italy|Investigational Site Number 380009, Firenze, Italy|Investigational Site Number 152013, Genova, Italy|Investigational Site Number 380003, Genova, Italy|Investigational Site Number 380007, Milano, Italy|Investigational Site Number 380005, Napoli, Italy|Investigational Site Number 380008, Padova, Italy|Investigational Site Number 380006, Roma, Italy|Investigational Site Number 380001, Udine, Italy|Investigational Site Number 392102, Nangoku-shi, Japan|Investigational Site Number 392101, Setagaya, Japan|Investigational Site Number 153178, Busan, Korea, Republic of|Investigational Site Number 154286, Changwon-si, Korea, Republic of|Investigational Site Number 153191, Daejeon, Korea, Republic of|Investigational Site Number 153228, Jeju-si, Korea, Republic of|Investigational Site Number 153225, Jeonnam, Korea, Republic of|Investigational Site Number 153180, Seoul, Korea, Republic of|Investigational Site Number 153196, Seoul, Korea, Republic of|Investigational Site Number 153192, Seoul, Korea, Republic of|Investigational Site Number 151149, Seoul, Korea, Republic of|Investigational Site Number 153188, Seoul, Korea, Republic of|Investigational Site Number 153212, Seoul, Korea, Republic of|Investigational Site Number 153222, Seoul, Korea, Republic of|Investigational Site Number 154148, Yangsan, Korea, Republic of|Investigational Site Number 154208, Yangsan, Korea, Republic of|Investigational Site Number 154225, Kuwait, Kuwait|Investigational Site Number 458001, Jalan Pahang, Malaysia|Investigational Site Number 458002, Kuala Lumpur, Malaysia|Investigational Site Number 151171, Amsterdam, Netherlands|Investigational Site Number 152029, Rotterdam, Netherlands|Investigational Site Number 608001, Manila, Philippines|Investigational Site Number 150575, Warszawa, Poland|Investigational Site Number 6200002, Coimbra, Portugal|Investigational Site Number 643001, Moscow, Russian Federation|Investigational Site Number 643002, Moscow, Russian Federation|Investigational Site Number 702001, Singapore, Singapore|Investigational Site Number 703001, Bratislava, Slovakia|Investigational Site Number 154093, Chiayi, Taiwan|Investigational Site Number 152152, Hualien, Taiwan|Investigational Site Number 152246, Kaohsiung Hsien,, Taiwan|Investigational Site Number 152244, Kaohsiung, Taiwan|Investigational Site Number 151534, New Taipei City, Taiwan|Investigational Site Number 151500, Putz City, Chia-yi, Taiwan|Investigational Site Number 151179, Taichung City, Taiwan|Investigational Site Number 151181, Taichung, Taiwan|Investigational Site Number 152153, Tainan, Taiwan|Investigational Site Number 153248, Tainan, Taiwan|Investigational Site Number 151182, Taipei, Taiwan|Investigational Site Number 152321, Taipei, Taiwan|Investigational Site Number 153158, Tao Yuan County, Taiwan|Investigational Site Number 764002, Bangkok, Thailand|Investigational Site Number 764003, Bangkok, Thailand|Investigational Site Number 764001, Bangkok, Thailand|Investigational Site Number 764004, Bangkok, Thailand|Investigational Site Number 764005, Khon Kaen, Thailand|investigational site number TBC, Dubai, United Arab Emirates|Investigational Site Number 152287, Birmingham, United Kingdom|Investigational Site Number 1232456, Birmingham, United Kingdom|Investigational Site Number 154240, Birmingham, United Kingdom|Investigational Site Number 154212, Hanoi, Vietnam | https://ClinicalTrials.gov/show/NCT00144794 | |
| 49 | The Natural History of Mucolipidosis Type IV | Recruiting | No Results Available | Mucolipidosis Type IV | | Baylor Institute of Metabolic Disease, Dallas, Texas, United States | https://ClinicalTrials.gov/show/NCT01067742 | |
| 50 | A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB | Completed | No Results Available | MPS IIIB (Sanfilippo Syndrome) | | Pittsburg, Pennsylvania, United States|Porte Alegre, Brazil|Amsterdam, Netherlands|Barcelona, Spain|Birmingham, United Kingdom | https://ClinicalTrials.gov/show/NCT02293382 | |
| 51 | Observational Study of Patients With Mucopolysaccharidosis (MPS) VI Who Previously Participated in ASB-00-02 | Completed | No Results Available | MPS VI | | Children's Hospital and Research Center, Oakland, California, United States|Women's and Children's hospital, North Adelaide, Australia|Hospital de Clinicas de Porto Alegre, Porto Alegre, Brazil|Hôpital Femme Mère Enfant, Lyon, France|Prof Michael Beck, Mainz, Germany|Al. Prof Hernani Monteiro, Porto, Portugal|Manchester Academic Health Sciences Centre, Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT01387854 | |
| 52 | Discovering New Biomarkers For Monitoring Disease Progression in Patients With Mucopolysaccharidosis IVA | Terminated | No Results Available | Mucopolysaccharidosis Type IVA | | Emory University, Dept of Human Genetics, Decatur, Georgia, United States | https://ClinicalTrials.gov/show/NCT01733615 | |
| 53 | Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I and II | Recruiting | No Results Available | Mucopolysaccharidosis I|Mucopolysaccharidosis II | Drug: Adalimumab Injection [Humira]|Drug: Saline Solution for Injection | Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, California, United States | https://ClinicalTrials.gov/show/NCT03153319 | |
| 54 | A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) | Completed | Has Results | Sly Syndrome|MPS VII|Mucopolysaccharidosis|Mucopolysaccharidosis VII | Drug: UX003 | Children's Hospital Oakland, Oakland, California, United States|Children's Hospital of Orange County, Orange, California, United States|University of Minnesota, Minneapolis, Minnesota, United States|Baylor College of Medicine, Houston, Texas, United States|Hospital Infantil Candido Fontoura Sao Paulo, Sao Paulo, Brazil|Centenario Hospital Miguel Hidalgo, Pediatrics, Aguascalientes, Mexico|Unidade de Doenças Metabólicas - Centro Hospitalar do Porto, Porto, Portugal | https://ClinicalTrials.gov/show/NCT02432144 | |
| 55 | Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of Age | Completed | Has Results | Sly Syndrome|MPS VII|Mucopolysaccharidosis|Mucopolysaccharidosis VII | Drug: UX003 | Children's National Health System, Washington, District of Columbia, United States|New York University Langone Medical Center, New York, New York, United States|University of Utah Hospital, Salt Lake City, Utah, United States|Centro Hospitalar do Porto, Porto, Portugal|Hospital Universitario Virgen Del Rocio, Sevilla, Spain | https://ClinicalTrials.gov/show/NCT02418455 | |
| 56 | A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously | Terminated | Has Results | Mucopolysaccharidosis III, Type B (MPS IIIB)|Sanfilippo B | Drug: SBC-103 | Birmingham, United Kingdom | https://ClinicalTrials.gov/show/NCT02618512 | |
| 57 | Mucopolysaccharidosis (MPS) VI Clinical Surveillance Program (CSP) | Recruiting | No Results Available | Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy Syndrome) | | University of California, Irvine, Irvine, California, United States|Children's Hospital of Los Angeles, Los Angeles, California, United States|Children's Hospital and Research Center Oakland, Oakland, California, United States|Emory University, Decatur, Georgia, United States|Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States|Indiana University School of Medicine, Indianapolis, Indiana, United States|Baton Rouge Clinic, Baton Rouge, Louisiana, United States|Tulane University Medical Center, New Orleans, Louisiana, United States|Johns Hopkins Univeristy School of Medicine, Baltimore, Maryland, United States|Children's Health Care, Minneapolis, Minnesota, United States|University of Minnesota - Fairview University Medical Center, Minneapolis, Minnesota, United States|Children's Mercy Hospital, Kansas City, Missouri, United States|St. Joseph's Healthcare, Paterson, New Jersey, United States|New York University, New York, New York, United States|Fullerton Genetic Center, Asheville, North Carolina, United States|Carolinas Medical Center, Charlotte, North Carolina, United States|Oregon Health and Science University, Portland, Oregon, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States|Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States|Sioux Valey Children's Speciality Clinics, Sioux Falls, South Dakota, United States|University of Utah Medical Center, Salt Lake City, Utah, United States|Children's Hospital of the King's Daughters, Norfolk, Virginia, United States|Seattle Children's Hospital, Seattle, Washington, United States|Westmead Hospital, Wentworthville, New South Wales, Australia|Royal Children's Hospital, Brisbane, Brisbane, Queensland, Australia|Women and Children's Hospital, Adelaide, South Australia, Australia|Royal Children's Hospital, Parkville, Victoria, Australia|Princess Margaret Hospital for Children, Subiaco, Western Australia, Australia|LKH-Universitätsklinik Graz, Kinderklinik, Graz, Steiermark, Austria|Institute of Pathology and Genetics, Metabolic Unit, Charleroi, Wallonie, Belgium|Hôpital Femme Mère Enfant, Bron cedex, Rhône-alpes, France|Hopital Necker - Enfants Malades, Paris, France|Universitatsklinikum Freiburg, Klinik II, Kammin, Mecklenburg-vorpommern, Germany|Oberarzt Pädiatrie, Hannover, Niedersachsen, Germany|Universitätsmedizin, Mainz, Rheinland-Pfalz RP, Germany|Charité-Universitätsmedizin Berlin, Berlin, Germany|Universitätsklinikum Hamburg Eppendorf, Hamburg, Germany|MidWestern Regional Hospital, Limerick, Ireland|Azienda Ospedaliero Universitaria, Cibali, Catania, Italy|Department of Woman's and Child's Health, Padova, Veneto, Italy|Università Milano Bicocca, Resp. Centro "Fondazione Mariani", Monza, Italy|Vilnius University Hospital, Santariskiu Klinikos, Vilnius, Lithuania|Rotterdam University Hospital - Sophia's Children's Hospital, Rotterdam, Netherlands|Hospital de Sao Joao, Unidae de Doencas Metabolicas, Porto, Portugal|Astrid Lindgrens Children's Hospital, Stockholm, Sweden|Queen Elizabeth Hospital, University Hospitals Birmingham NHS, Birmingham, United Kingdom|Birmingham Children's Hospital, Steelhouse, Birmingham, United Kingdom|Great Ormond Street Hospital For Children, NHS Foundation Trust, London, United Kingdom|St Mary's Hospital, Willink Biochemical Genetics Unit, Manchester, United Kingdom|Salford Royal Hospital NHS Trust, Salford, United Kingdom | https://ClinicalTrials.gov/show/NCT00214773 | |
| 58 | Immune Tolerance Study With Aldurazyme® (Laronidase) | Completed | Has Results | Mucopolysaccharidosis I | Biological: Laronidase|Drug: Cyclosporine A (CsA)|Drug: Azathioprine (Aza) | HCPA, Porto Alegre, Brazil|Moscow Research Institute for Pediatrics and Children Surgery, Moscow, Russian Federation | https://ClinicalTrials.gov/show/NCT00741338 | |
| 59 | Natural History Study to Characterise the Course of Disease Progression in Participants With Mucopolysaccharidosis Type IIIB | Terminated | No Results Available | MPS IIIB (Sanfilippo B Syndrome) | | Minneapolis, Minnesota, United States|Pittsburgh, Pennsylvania, United States|Porto Alegre, Brazil|Monza, Italy|Coimbra, Portugal|Barcelona, Spain|Birmingham, United Kingdom | https://ClinicalTrials.gov/show/NCT02293408 | |
| 60 | Effects of Adalimumab in Mucopolysaccharidosis Types I, II and VI | Completed | Has Results | Mucopolysaccharidosis Type I|Mucopolysaccharidosis Type II|Mucopolysaccharidosis Type VI | Drug: Adalimumab|Other: Placebo | Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, California, United States | https://ClinicalTrials.gov/show/NCT02437253 | |
| 61 | Ultrasound Findings of Finger, Wrist and Knee Joints in Mucopolysaccharidosis | Completed | No Results Available | MPS I|MPS IV | | Children's Hospital of Eastern Ontario, Ottawa, Ontario, Canada | https://ClinicalTrials.gov/show/NCT02067650 | |
| 62 | A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | Completed | Has Results | MPS IV A | Drug: BMN 110 Weekly|Drug: Placebo|Drug: BMN 110 Every Other Week | Oakland, California, United States|Wilmington, Delaware, United States|Washington, District of Columbia, United States|Chicago, Illinois, United States|New York, New York, United States|Seattle, Washington, United States|Cordoba, Argentina|Campina Grande, Brazil|Porto Alegre, Brazil|Montreal, Canada|Sherbrooke, Canada|Toronto, Canada|Bogota, Colombia|Copenhagen, Denmark|Lyon, France|Paris, France|Mainz, Germany|Monza, Italy|Tokyo, Japan|Seoul, Korea, Republic of|Amsterdam, Netherlands|Coimbra, Portugal|Doha, Qatar|Riyadh, Saudi Arabia|Taipei, Taiwan|Birmingham, United Kingdom|London, United Kingdom|Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT01275066 | |
| 63 | Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease | Completed | No Results Available | Mucopolysaccharidosis IV A|Mucopolysaccharidosis VI | Other: Enzyme testing | Children's Hospitals and Clinics of Minnesota, Minneapolis, Minnesota, United States|Gillette Children's Specialty Healthcare, St Paul, Minnesota, United States | https://ClinicalTrials.gov/show/NCT01707433 | |
| 64 | Study of BMN 110 in Pediatric Patients < 5 Years of Age With Mucopolysaccharidosis IVA (Morquio A Syndrome) | Completed | Has Results | Mucopolysaccharidosis IVA|Morquio A Syndrome|MPS IVA | Drug: BMN 110 | Oakland, California, United States|Manhasset, New York, United States|Monza, Italy|Taipei, Taiwan|Central Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT01515956 | |
| 65 | Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II) | Completed | No Results Available | Mucopolysaccharidosis (MPS)|Hunter Syndrome | | Childrens Hospital & Research Center Oakland, Oakland, California, United States|Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States|University of North Carolina Division of Genetics and Metabolism, Chapel Hill, North Carolina, United States|Hospital Universitario Austral, Pilar, Buenos Aires, Argentina|Instituto Nacional De Pediatria, Mexico City, Mexico|Hospital Infantil Universitario, Madrid, Spain|Central Manchester University Hospitals NHS Foundation Trust Willink Biochemical Genetics Unit, St. Mary's Hospital, Manchester, M13 9wl, United Kingdom | https://ClinicalTrials.gov/show/NCT01822184 | |
| 66 | A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA | Completed | Has Results | MPS IV A | Drug: BMN 110 | Birmingham, United Kingdom|London, United Kingdom|Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT00884949 | |
| 67 | A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | Terminated | Has Results | MPS IV A|Mucopolysaccharidosis IVA|Morquio A Syndrome | Drug: BMN 110 | Birmingham, United Kingdom|Dumfries, United Kingdom|London, United Kingdom|Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT01242111 | |
| 68 | Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome) | Completed | Has Results | Mucopolysaccharidosis IV A|Morquio A Syndrome|MPS IVA | Drug: BMN 110 - Weekly|Drug: BMN 110 - Every Other Week | Phoenix, Arizona, United States|Oakland, California, United States|Orange, California, United States|Wilmington, Delaware, United States|Washington, District of Columbia, United States|Orlando, Florida, United States|Honolulu, Hawaii, United States|Chicago, Illinois, United States|New York, New York, United States|Seattle, Washington, United States|Cordoba, Argentina|Campina Grade, Brazil|Porto Alegre, Brazil|Rio de Janeiro, Brazil|Montreal, Canada|Sherbrooke, Canada|Toronto, Canada|Bogota, Colombia|Copenhagen, Denmark|Lyon, France|Marseille, France|Paris, France|Paris, France|Mainz, Germany|Monza, Italy|Tokyo, Japan|Seoul, Korea, Republic of|Amsterdam, Netherlands|Oslo, Norway|Coimbra, Portugal|Lisbon, Portugal|Doha, Qatar|Riyadh, Saudi Arabia|Santiago de Compostela, Spain|Taipei, Taiwan|Ankara, Turkey|Belfast, United Kingdom|Birmingham, United Kingdom|Birmingham, United Kingdom|London, United Kingdom|London, United Kingdom|London, United Kingdom|Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT01415427 | |
| 69 | Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II) | Completed | Has Results | Mucopolysaccharidosis II | Biological: Iduronate-2-sulfatase enzyme replacement therapy|Biological: iduronate-2-sulfatase enzyme replacement therapy|Biological: Placebo | Children's Hospital Oakland, Oakland, California, United States|St. Louis Children's Hospital, Washington University, St. Louis, Missouri, United States|University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States|Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, United States|Hospital de Clinicas de Porto Alegre, Porto Alegre, Brazil|Children's Hospital, Johannes-Gutenburg Universitaet Mainz, Mainz, Germany|Addenbrooke's Hospital, Cambridge, England, United Kingdom|Great Ormond Street Hospital for Sick Children, London, England, United Kingdom|Royal Manchester Children's Hospital, Manchester, England, United Kingdom | https://ClinicalTrials.gov/show/NCT00069641 | |
| 70 | Study of Idursulfase-beta (GC1111) in Hunter Syndrome | Unknown status | No Results Available | Mucopolysaccharidosis II | Biological: idursulfase beta|Biological: idursulfase | | https://ClinicalTrials.gov/show/NCT02663024 | |
| 71 | Study of rhASB in Patients With Mucopolysaccharidosis VI | Completed | Has Results | Mucopolysaccharidosis VI | Drug: N-acetylgalactosamine 4-sulfatase|Drug: Placebo/rhASB | BioMarin Pharmaceutical Inc., Novato, California, United States | https://ClinicalTrials.gov/show/NCT00104234 | |
| 72 | Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease | Completed | Has Results | Mucopolysaccharidosis I|Hurler Syndrome|Hurler-Scheie Syndrome|Scheie Syndrome | Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) | Osaka City University Hospital, Osaka, Japan|National Center for Child Health and Development, Tokyo, Japan | https://ClinicalTrials.gov/show/NCT00258011 | |
| 73 | A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome) | Terminated | No Results Available | MPS IV A|Mucopolysaccharidosis IVA|Morquio A Syndrome | | Oakland, California, United States|Chicago, Illinois, United States|Cordoba, Argentina|Porto Alegre, Brazil|Sao Paulo, Brazil|Montreal, Quebec, Canada|Lyon, France|Paris, France|Mainz, Germany|Monza, Italy|Amsterdam, Netherlands|Taipei, Taiwan|Birmingham, United Kingdom|London, United Kingdom|Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT00787995 | |
| 74 | Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network | Completed | No Results Available | MPS I|MPS II|MPS VI|Mucopolysaccharidoses | | Children's Hospital of Orange County, Orange, California, United States|University of Minnesota, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT01586871 | |
| 75 | To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients | Completed | No Results Available | Mucopolysaccharidosis II | Drug: GC1111_0.5mg/kg|Drug: GC1111_1.0mg/kg|Drug: Elaprase_0.5mg/kg | Samsung Medical Center, Seoul, Irwon-dong, Gangnam-Gu, Korea, Republic of | https://ClinicalTrials.gov/show/NCT01301898 | |
| 76 | Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH | Recruiting | No Results Available | MPS IIIA|Sanfilippo Syndrome|Sanfilippo A|Mucopolysaccharidosis III | Biological: ABO-102 | Nationwide Children's Hospital, Columbus, Ohio, United States|Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States|Women's and Children's Hospital, North Adelaide, South Australia, Australia|Hospital ClÃnico Universitario de Santiago, Santiago De Compostela, Spain | https://ClinicalTrials.gov/show/NCT02716246 | |
| 77 | Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic Resonance Imaging | Completed | No Results Available | MPS IIIB (Sanfilippo B Syndrome) | | Birmingham, United Kingdom | https://ClinicalTrials.gov/show/NCT02090179 | |
| 78 | Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) | Completed | No Results Available | Sanfilippo Syndrome Type B | | Children's Hospital and Research Center, Oakland, California, United States|University of Minnesota, Minneapolis, Minnesota, United States|Johannes Gutenberg University Medical Center, Mainz, Germany|Great Ormond Street Hospital for Children, London, United Kingdom | https://ClinicalTrials.gov/show/NCT01509768 | |
| 79 | Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I | Completed | Has Results | Mucopolysaccharidosis I|Hurlers Syndrome|Hurler-Scheie Syndrome | Biological: rhIDU (recombinant human-Alpha-L-Iduronidase)|Biological: Placebo | New York, New York, United States|Chapel Hill, North Carolina, United States|Vancouver, British Columbia, Canada|Mainz, Germany | https://ClinicalTrials.gov/show/NCT00912925 | |
| 80 | A Study to Assess the Safety, Tolerability, and Efficacy of Long-term SOBI003 Treatment in Pediatric MPS IIIA Patients | Active, not recruiting | No Results Available | Sanfilippo Syndrome Type A (MPS IIIA) | Drug: SOBI003 | Children´s Hospital and research center, Oakland, California, United States|University of North Carolina hospitals, Chapel Hill, North Carolina, United States|Gazi University Hospital, Ankara, Turkey | https://ClinicalTrials.gov/show/NCT03811028 | |
| 81 | Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase | Completed | Has Results | Hunter Syndrome|Mucopolysaccharidosis II (MPS II) | Biological: Idursulfase | St. Joseph's Hospital, Phoenix, Arizona, United States|Pediatric Clinical Research Center, Children's Hospital Oakland, Oakland, California, United States|The Children's Hospital, Denver, Colorado, United States|Harbin Clinic, Rome, Georgia, United States|Mid-Illinois Hematology and Oncology Associates, Normal, Illinois, United States|University of Iowa Hospitals and Clinics, Iowa City, Iowa, United States|Children's Hospital Boston, Boston, Massachusetts, United States|Saint Louis University Cardinal Glennon Children's Hospital, St. Louis, Missouri, United States|University of Nebraska Medical Center, Omaha, Nebraska, United States|Comprehensive Cancer Centers of Nevada, Las Vegas, Nevada, United States|Upstate Medical University, State University of New York (SUNY), Syracuse, New York, United States|University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States|Baylor College of Medicine Texas Children's Hospital, Houston, Texas, United States|University of Utah Hospital, Salt Lake City, Utah, United States|Franciscan Skemp Healthcare, La Crosse, Wisconsin, United States|Fundacao Universidade de Ciencias da Saude de Alagoas Governador Lamenha Filho / UNCISAL, Maceio, AL, Brazil|Clinica Casa de Saude Sao Joao, Barreiras, BA, Brazil|c-HUPES/UFBA, Salvador, BA, Brazil|Hospital Universitario da Faculdade de Medicina da Universidade Federal de Mato Grosso do Sul, Campo Grande, MS, Brazil|Instituto de Puericultura e Pediatria Martagao Gesteira / Hospital Pediatrico, Rio de Janeiro, RJ, Brazil|Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica, Porto Alegre, RS, Brazil|UNIFESP Instituto de Oncologia Pediatrica, Sao Paulo, SP, Brazil|Instituto da Crianca / Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo, Sao Paulo, SP, Brazil|The Hospital for Sick Children Research Institute, Toronto, Ontario, Canada|University of Montreal / Hopital Ste-Justine, Montreal, Quebec, Canada|Hopital Edouard Herriot, Lyon, France|Hopital de Hautepierre, Strasbourg Cedex, France|Hospital Ducuing, Toulouse Cedex, France|Universitatsklinikum Aachen Kinderklinik, Aachen, Germany|Universitatsklinik Dusseldorf Kinderklinik, Dusseldorf, Germany|Justus-Liebig Universitat, Giessen, Germany|Universitatsklinikum Gottingen, Goettingen, Germany|Universitatsklinikum Hamburg Eppendorf, Hamburg, Germany|Children's University Hospital Mainz AG, Mainz, Germany|Universita Milano Bicocca / Ospedale S. Gerardo, Monza (Milano), Italy|Universita degli Studi di Napoli Federico II, Napoli, Italy|Universita di Padova, Padova, Italy|Ospedale S. S. Annunziata, Savigliano (Cuneo), Italy|Spitalul Clinic de Copii, Cluj Napoca, Cluj, Romania|University Hospital Germans Trias i Pujol, Badalona, Spain|Servicio de Pediatria, Linares (Jaen), Spain|Drottning Silvias Barnsjukhus, Gothenberg, Sweden|Karolinska University Hospital, Stockholm, Sweden|Bath and NE Somerset Primary Care Trust, Bath, United Kingdom|Addenbrooke's Hospital, Cambridge, United Kingdom|Derbyshire Children's Hospital, Derby, United Kingdom|Royal Hospital for Sick Children, Glasgow, United Kingdom|Great Ormond Street Hospital for Sick Children, London, United Kingdom|Royal Manchester Children's Hospital, Manchester, United Kingdom|Royal Victoria Infirmary, Newcastle, United Kingdom|Royal Surrey County Hospital, Surrey, United Kingdom | https://ClinicalTrials.gov/show/NCT00630747 | |
| 82 | The Natural History and Pathogenesis of Mucolipidosis Type IV | Completed | No Results Available | Mucolipidosis Type IV | | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, United States | https://ClinicalTrials.gov/show/NCT00015782 | |
| 83 | A Study to Assess the Safety and Tolerability of SOBI003 in Pediatric MPS IIIA Patients | Completed | No Results Available | Sanfilippo Syndrome Type A (MPS IIIA) | Drug: SOBI003 | Childrens's Hospital and Research Center, Oakland, California, United States|University of North Carolina Hospitals, Chapel Hill, North Carolina, United States|University Medical Center Hamburg-Eppendorf, Hamburg, Germany|Gazi University Hospital, Ankara, Turkey | https://ClinicalTrials.gov/show/NCT03423186 | |
| 84 | A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants | Unknown status | No Results Available | Mucopolysaccharidosis I|Hurler's Syndrome|Hurler-Scheie Syndrome|Scheie | Biological: Aldurazyme (laronidase) | Dipartimento di Scienze Pediatriche Medico - Chirurgiche e Neuro Scienze dello Suiluppo, Rome, Italy | https://ClinicalTrials.gov/show/NCT00418821 | |
| 85 | Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I | Terminated | No Results Available | Mucopolysaccharidosis I|Cognitive Decline | Drug: Intrathecal recombinant human alpha iduronidase | Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center, Torrance, California, United States | https://ClinicalTrials.gov/show/NCT02232477 | |
| 86 | Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease | Recruiting | No Results Available | MPS IIIA|Sanfilippo Syndrome|Sanfilippo A|Mucopolysaccharidosis III | Drug: ABO-102 | Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States|Adelaide Women's and Children's Hospital, North Adelaide, South Australia, Australia|Hospital ClÃnico Universitario de Santiago, Santiago De Compostela, Spain | https://ClinicalTrials.gov/show/NCT04088734 | |
| 87 | Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I | Terminated | No Results Available | Mucopolysaccharidosis I|Lysosomal Storage Diseases|Spinal Cord Compression | Drug: laronidase | Los Angeles Biomedical Research Institute at Harbor-UCLA ( LA BioMed ), Torrance, California, United States|Helsinki University Central Hospital, Helsinki, Finland | https://ClinicalTrials.gov/show/NCT00215527 | |
| 88 | Long-term Follow-Up for RGX-121 | Not yet recruiting | No Results Available | Mucopolysaccharidosis II | Other: Long-term Follow-Up | | https://ClinicalTrials.gov/show/NCT04597385 | |
| 89 | A Study of DNL310 in Pediatric Subjects With Hunter Syndrome | Recruiting | No Results Available | Mucopolysaccharidosis II | Drug: DNL310 | UCSF Benioff Children's Hospital, Oakland, California, United States|UNC Children's Research Institue, Chapel Hill, North Carolina, United States|UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States | https://ClinicalTrials.gov/show/NCT04251026 | |
| 90 | Study of Recombinant Human N-Acetylgalactosamine 4-Sulfatase in Patients With MPS VI | Completed | No Results Available | Mucopolysaccharidosis VI | Drug: N-acetylgalactosamine 4-sulfatase | BioMarin Pharmaceutical Inc., Novato, California, United States | https://ClinicalTrials.gov/show/NCT00048620 | |
| 91 | Open-Label Study of Efficacy and Safety of Recombinant Human N-acetylgalactosamine 4-sulfatase in Patients With MPS VI | Completed | No Results Available | Mucopolysaccharidosis VI | Drug: N-acetylgalactosamine 4-sulfatase | BioMarin Pharmaceutical Inc., Novato, California, United States | https://ClinicalTrials.gov/show/NCT00048711 | |
| 92 | BMN 110 Phase 3B in Australian Patients | Completed | No Results Available | Mucopolysaccharidosis IVA (Morquio A Syndrome) | Drug: BMN 110 | Children's Hospital at Westmead, Westmead, New South Wales, Australia|Lady Cilento Children's Hospital (previous: Royal Children's Hospital), Brisbane, Queensland, Australia|Murdoch Childrens Research Institute and Royal Children's Hospital, Melbourne, Victoria, Australia|Princess Margaret Hospital for Children, Perth, Western Australia, Australia|Pain and Anaesthesia Research Clinic, Royal Adelaide Hospital, Adelaide, Australia | https://ClinicalTrials.gov/show/NCT01966029 | |
| 93 | Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation | Terminated | Has Results | Mucopolysaccharidosis IVA|Morquio A Syndrome|MPS IVA | Drug: BMN 110 | Children's Hospital & Research Center Oakland, Oakland, California, United States|Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States|Universitätsklinikum Hamburg, Hamburg, Germany|University Medical Center Mainz, Center of Pediatric and Adolescent Medicine Villa Metabolica, Mainz, Germany|NIHR/Wellcome Trust Birmingham CRF, Queen Elizabeth Hospital, Birmingham, United Kingdom|Central Manchester University Hospitals NHS Foundation Trust, Manchester, United Kingdom|Salford Royal NHS Foundation Trust, Salford, United Kingdom | https://ClinicalTrials.gov/show/NCT01697319 | |
| 94 | Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome | Terminated | Has Results | Mucopolysaccharidosis IVA|Morquio A Syndrome|MPS IVA | Drug: BMN 110 | Oakland, California, United States|Chicago, Illinois, United States|New York, New York, United States|Houston, Texas, United States|Calgary, Alberta, Canada|Toronto, Ontario, Canada|Montreal, Quebec, Canada|Sherbrook, Quebec, Canada|Hamburg, Germany|Belfast, Northern Ireland, United Kingdom|Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT01609062 | |
| 95 | Dynamic Gait Analysis in Children With Mucopolysaccharidosis Type IVa | Withdrawn | No Results Available | Mucopolysaccharidosis IV|Morquio A Disease | | Birmingham Children's Hospital NHS Foundation Trust, Birmingham, West Midlands, United Kingdom | https://ClinicalTrials.gov/show/NCT02153255 | |
| 96 | Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome) | Completed | No Results Available | Mucopolysaccharidosis II | Genetic: lymphocyte gene therapy | University of Minnesota Medical School, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT00004454 | |
| 97 | BMN 110 US Expanded Access Program | Approved for marketing | No Results Available | Mucopolysaccharidosis IVA|Morquio A Syndrome|MPS IVA | Drug: BMN 110 | Birmingham, Alabama, United States|Little Rock, Arkansas, United States|Oakland, California, United States|Orange, California, United States|Aurora, Colorado, United States|Washington, District of Columbia, United States|Hollywood, Florida, United States|Miami, Florida, United States|Atlanta, Georgia, United States|Chicago, Illinois, United States|Louisville, Kentucky, United States|New Orleans, Louisiana, United States|Minneapolis, Minnesota, United States|Paterson, New Jersey, United States|Manhasset, New York, United States|New York, New York, United States|Portland, Oregon, United States|Philadelphia, Pennsylvania, United States|Pittsburgh, Pennsylvania, United States|Nashville, Tennessee, United States|Houston, Texas, United States|Salt Lake City, Utah, United States|Seattle, Washington, United States|Tacoma, Washington, United States|Santurce, Puerto Rico | https://ClinicalTrials.gov/show/NCT01858103 | |
| 98 | MT2018-18: Sleeping Beauty Transposon-Engineered Plasmablasts for Hurler Syndrome Post Allo HSCT | Not yet recruiting | No Results Available | Mucopolysaccharidosis Type IH (MPS IH, Hurler Syndrome)|Mucopolysaccharidosis Type IH|MPS IH, Hurler Syndrome | Drug: Autologous Plasmablasts | | https://ClinicalTrials.gov/show/NCT04284254 | |
| 99 | A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease | Completed | Has Results | Mucopolysaccharidosis I|Hurler's Syndrome|Hurler-Scheie Syndrome|Scheie Syndrome | Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) | Hospital Infantil Joana de Gusmao, Florianopolis, Santa Catarina, Brazil|Universidade Federal de Minas Gerais, Belo Horizonte, Brazil|Hospital de Clinical de Porto Alegre, Porto Alegre, Brazil|Universidade Federal de Sao Paulo, San Paulo, Brazil|Division of Clinical and Metabolic Genetics, Toronto, Ontario, Canada | https://ClinicalTrials.gov/show/NCT00144781 | |
| 100 | Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients | Completed | Has Results | Mucopolysaccharidosis I|Hurler's Syndrome|Hurler-Scheie Syndrome|Scheie Syndrome | Biological: Aldurazyme|Biological: placebo | University of South Alabama, Mobile, Alabama, United States|Emory University School of Medicine, Atlanta, Georgia, United States|University of Rochester, Rochester, New York, United States|University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States|Toledo Children's Hospital, Toledo, Ohio, United States|Merle West Medical Center, Klamath Falls, Oregon, United States|The Childrens Hospital of Philadelphia, Philadelphia, Pennsylvania, United States|Rhode Island Hospital, Providence, Rhode Island, United States|Hospital Universatario de Universidade Federal de Santa Catarina, Florianopolis, Brazil|Hospital Infantil Joana de Gusmao, Florianopolis, Brazil|The Hospital for Sick Children, Toronto, Ontario, Canada|Alberta Children's Hospital, Calgary, Canada|Children's Hospital Klinikum Nord Heidberg, Hamburg, Germany|Medizinishe Hochshule Hannover, Hannover, Germany|Children's Hospital at the University Hospital of Heidelberg, Heidelberg, Germany|Children's Hospital Klinikum der F.S. Universitat, Jena, Germany|Catholic University Sacro Cuore, Rome, Italy|Academisch Ziekenhuis Rotterdam, Rotterdam, Netherlands|Blackpool Victoria Hospital, Blackpool, Lancashire, United Kingdom|Belfast City Hospital, Belfast, United Kingdom|Birmingham Children's Hospital, Birmingham, United Kingdom|Bristol Royal Hospital for Children and Frenchay Hospital, Bristol, United Kingdom|Gartnavel Hospital, Glasgow, United Kingdom|Great Ormond Street Hospital for Sick Children and NHS Trust, London, United Kingdom|Royal Victoria Hospital, Newcastle upon Tyne, United Kingdom | https://ClinicalTrials.gov/show/NCT00146770 | |
| 101 | Non-invasive Assessment of Intraocular Pressure in MPS by Use of the Ocular Response Analyzer. | Unknown status | No Results Available | Mucopolysaccharidosis|Fabry Disease | | Department of Ophthalmology, University Medical Center, Johannes Gutenberg-, Mainz, Germany | https://ClinicalTrials.gov/show/NCT01695161 | |
| 102 | Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them? | Recruiting | No Results Available | Morquio Disease|MPS - Mucopolysaccharidosis | Other: Interview | Emory University, Decatur, Georgia, United States | https://ClinicalTrials.gov/show/NCT03150069 | |
| 103 | Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI | Recruiting | No Results Available | Mucopolysaccharidosis IV A|Mucopolysaccharidosis VI|Mucopolysaccharidoses|MPS IV A|MPS VI|MPS - Mucopolysaccharidosis|Morquio A Syndrome|Morquio Syndrome A|Morquio Syndrome | Drug: Losartan|Drug: Placebo | Hospital de Clinicas de Porto Alegre, Porto Alegre, Rio Grande Do Sul, Brazil | https://ClinicalTrials.gov/show/NCT03632213 | |
| 104 | Gait Analysis in MPS IVA | Completed | No Results Available | MPS IVA|Morquio Syndrome | | Oregon Health and Science University, Portland, Oregon, United States | https://ClinicalTrials.gov/show/NCT01920828 | |
| 105 | Biomarker for Mucolipidosis Disorder Type I, II, III, IV (BioML) | Active, not recruiting | No Results Available | Skeletal Abnormalities|Psychomotor Retardation | | Children's Hospital, Faculty of Medicine, Ain Shams University, Cairo, Egypt|Centogene AG, Rostock, Germany|Amrita Institute of Medical Sciences & Research Centre, Cochin, Kerala, India|NIRMAN-University of Mumbai-Institute of Research in Mental and Neurological handicap, Mumbai, India|Lady Ridgeway Hospital for Children, Colombo 8, Sri Lanka | https://ClinicalTrials.gov/show/NCT02298673 | |
| 106 | Clinical Trial of Growth Hormone in MPS I, II, and VI | Terminated | Has Results | Mucopolysaccharidosis I|Mucopolysaccharidosis II|Mucopolysaccharidosis VI | Drug: Somatropin (DNA origin) | University of Minnesota, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT00748969 | |
| 107 | Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients With MPS VI | Completed | Has Results | Mucopolysaccharidosis VI | Drug: Placebo|Drug: N-acetylgalactosamine 4-sulfatase | BioMarin Pharmaceutical Inc., Novato, California, United States | https://ClinicalTrials.gov/show/NCT00067470 | |
| 108 | Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181 | Completed | No Results Available | Mucopolysaccharidosis I | Drug: AGT-181 | | https://ClinicalTrials.gov/show/NCT02597114 | |
| 109 | Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III | Completed | No Results Available | Mucopolysaccharidosis Type I|Mucopolysaccharidosis Type II|Mucopolysaccharidosis Type III|Behavior Disorders|Coping Behavior | Other: This is an observational study | University of Heidelberg, Heidelberg, Germany | https://ClinicalTrials.gov/show/NCT03161171 | |
| 110 | Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics | Terminated | No Results Available | Mucopolysaccharidoses|Mucopolysaccharidosis I|Mucopolysaccharidosis II|Mucopolysaccharidosis IV|Mucopolysaccharidosis VI | Other: Dried blood spot test for MPS | University of Medicine and Dentistry of New Jersey, New Brunswick, New Jersey, United States|Hospital for Special Surgery, New York, New York, United States | https://ClinicalTrials.gov/show/NCT01675674 | |
| 111 | A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI | Completed | Has Results | Mucopolysaccharidosis VI|Maroteaux-Lamy Syndrome | Drug: Naglazyme | Children's Hospital Los Angeles, Los Angeles, California, United States|Children's Hospital Oakland, Oakland, California, United States|Hospital Femme Mere Enfant Centre, Lyon, France|Hospital PediAtrico de Coimbra, Coimbra, Portugal | https://ClinicalTrials.gov/show/NCT00299000 | |
| 112 | A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old | Completed | Has Results | Mucopolysaccharidosis I|Hurler Syndrome|Hurler-Scheie Syndrome|Scheie Syndrome | Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) | Hôpital E. Herriot, Lyon, France|Johannes Gutenberg Universität, Kinderklinik, Mainz, Germany|Sophia Children's Hospital, Rotterdam, Netherlands|Willink Biochemical Genetics Unit Royal Hospital for Children, Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT00146757 | |
| 113 | New Imaging Technology to Assess Effect of Enzyme Replacment Therapy on Eye Disease Progession in Mucopolysacchardiosis | Unknown status | No Results Available | Mucopolysaccharidoses | | Manchester Royal Eye Hospital, Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT02583152 | |
| 114 | A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723 | Completed | No Results Available | Mucopolysaccharidosis II | Drug: Idursulfase|Drug: GSK2788723 | GSK Investigational Site, Tokyo, Japan | https://ClinicalTrials.gov/show/NCT01602601 | |
| 115 | Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome | Terminated | No Results Available | Mucopolysaccharidosis Type IH|MPS I|Hurler Syndrome | Drug: Laronidase | Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT01572636 | |
| 116 | Natural History of Atypical Morquio A Disease | Recruiting | No Results Available | Mucopolysaccharidosis IV A | Drug: Elosulfase Alfa 1 MG/ML Intravenous Solution [VIMIZIM] | Rheumatology department - Bordeaux University Hospital, Bordeaux, Aquitaine, France | https://ClinicalTrials.gov/show/NCT03204370 | |
| 117 | Expanded Access to Mepsevii | Available | No Results Available | MPS VII|Mucopolysaccharidosis VII|Sly Syndrome | Drug: Mepsevii | | https://ClinicalTrials.gov/show/NCT03775174 | |
| 118 | The Long-term Safety Study of Idursulfase-beta in Hunter Syndrome(Mucopolysaccharidosis II) Patients | Unknown status | No Results Available | Hunter Syndrome | | Samsung Medical Center, Seoul, Korea, Republic of | https://ClinicalTrials.gov/show/NCT02044692 | |
| 119 | Stem Cell Transplant w/Laronidase for Hurler | Completed | Has Results | Mucopolysaccharidosis I|Hurler Syndrome | Procedure: Stem Cell Transplant|Drug: Laronidase ERT | Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT00176891 | |
| 120 | A Study Investigating the Relationship Between the Development of Laronidase Antibody and Urinary GAG (Glycosaminoglycan) Levels in Aldurazyme® Treated Patients | Completed | No Results Available | Mucopolysaccharidosis I|Hurler's Syndrome|Hurler-Scheie Syndrome|Scheie's Syndrome | Drug: laronidase | Childrens Hospital Los Angeles, Los Angles, California, United States|Connecticut Children's Medical Center, Hartford, Connecticut, United States|Children's Hospital of Wisconsin, Milwaukee, Wisconsin, United States | https://ClinicalTrials.gov/show/NCT00144768 | |
| 121 | Stem Cell Transplantation for Hurler | Completed | Has Results | Mucopolysaccharidosis I|Mucopolysaccharidosis VI|Mannosidosis|Mucolipidosis Type II (I-cell Disease) | Procedure: Stem Cell Transplant|Drug: Busulfan, Cyclophosphamide, ATG | Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT00176917 | |
| 122 | Longitudinal Studies of Brain Structure and Function in MPS Disorders | Completed | No Results Available | Mucopolysaccharidosis Type I|Mucopolysaccharidosis Type II|Mucopolysaccharidosis Type VI|Mucopolysaccharidosis Type IV|Mucopolysaccharidosis Type VII | | Oakland Children's Hospital, Oakland, California, United States|University of Minnesota, Minneapolis, Minnesota, United States|New York University, New York, New York, United States|Hospital for Sick Children, Toronto, Ontario, Canada | https://ClinicalTrials.gov/show/NCT01870375 | |
| 123 | Psychological Concomitants of Morquio Syndrome (The MAP Study) | Completed | No Results Available | Morquio Disease|Mucopolysaccharidosis IV | | Emory University, Decatur, Georgia, United States | https://ClinicalTrials.gov/show/NCT01752296 | |
| 124 | Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy | Completed | Has Results | Hunter Syndrome|Mucopolysaccharidosis II|MPS II | Biological: Idursulfase | Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica, Porto Alegre, RS, Brazil|Instytut Pomnik Centrum Zdrowia Dziecka, Klinika Chorob Metaboliczynch, Endokrynologii i Diabetologii, Warsaw, Poland|National Taiwan University Hospital, Dept. of Pediatrics and Medical Genetics, Taipei, Taiwan | https://ClinicalTrials.gov/show/NCT00607386 | |
| 125 | A Multicenter, Multinational, Observational Morquio A Registry Study (MARS) | Recruiting | No Results Available | Mucopolysaccharidosis IV Type A|Morquio A Syndrome|MPS IVA | Drug: Vimizim® (elosulfase alfa) | Phoenix Children's Hospital, Phoenix, Arizona, United States|Arkansas Children's Hospital, Little Rock, Arkansas, United States|Children's Hospital Los Angeles, Los Angeles, California, United States|University of Southern California, Los Angeles, California, United States|Children's Hospital of Orange County, Orange, California, United States|A.I. Dupont Hospital for Children, Wilmington, Delaware, United States|Children's National Health System, Washington, District of Columbia, United States|Miami Children's Hospital, Miami, Florida, United States|Emory University, Decatur, Georgia, United States|Kapiolani Medical Center for Women and Children, Honolulu, Hawaii, United States|Ann & Robert H. Lurie Children's Hospital, Chicago, Illinois, United States|University Of Iowa, Iowa City, Iowa, United States|University of Louisville Research Foundation Inc. - Kosair Charities Pediatric Clinical Research Unit, Louisville, Kentucky, United States|Children's Hospital of Boston, Boston, Massachusetts, United States|University of Michigan, Ann Arbor, Michigan, United States|Childrens Hospitals and Clinics of Minnesota, Minneapolis, Minnesota, United States|Children's Specialty Center of Nevada, Las Vegas, Nevada, United States|Atlantic Health System, Morristown, New Jersey, United States|St. Joseph's Regional Medical Center, Paterson, New Jersey, United States|NYU-Langone Medical Center School of Medicine, New York, New York, United States|Duke University Medical Center, Durham, North Carolina, United States|Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States|Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, United States|UTHealth McGovern Medical School, Houston, Texas, United States|Children's Hospital of the King's Daughters, Norfolk, Virginia, United States|Seattle Children's Hospital, Seattle, Washington, United States|Children's Hospital of Wisconsin, Milwaukee, Wisconsin, United States|Government Of Western Australia - Princess Margaret Hospital For Children (PMH), Perth, Nedlands, Australia|Children's Hospital of Westmead, Westmead, New South Wales, Australia|Westmead Hospital, Westmead, New South Wales, Australia|Chidren's Heatlh Queensland Hospital and Health Service, South Brisbane, Queensland, Australia|Royal Adelaide Hospital, Adelaide, South Australia, Australia|Royal Children's Hospital - Victoria, Parkville, Victoria, Australia|Royal Perth Hospital, Perth, Western Australia, Australia|Medical University of Vienna, Vienna, Austria|Antwerp University Hospital, Edegem, Belgium|Children's & Women's Centre of British Columbia, Vancouver, British Columbia, Canada|Sainte-Justine Hospital, Montreal, Quebec, Canada|McGill University Health Centre, Montreal, Quebec, Canada|Université de Sherbrooke, Sherbrooke, Quebec, Canada|Klinika Dětského a Dorostového Lékařstvi, Prague, Czechia|Copenhagen University Hospital, Klinik For Sjaeldne Handicap, Copenhagen, Dk-2100, Denmark|Hôpital Femme Mère Enfant, Bron Cedex, France|Hopital Necker, Paris Cedex 15, France|Hopital d'enfants - Hopitaux de Brabois, Vandœuvre-lès-Nancy, France|Clinical Study Center, University Pediatric Clinic, Bochum, Germany|Universitätsklinikum Hamburg-Eppendorf, Hamburg, Germany|University Medical Center, Johannes Gutenberg-University Mainz, Mainz, Germany|Zentrum für seltene Erkrankungen, Horst-Schmidt-Kliniken, Wiesbaden, Germany|Temple Street Children's University Hospital, Dublin, Ireland|Azienda Policlinico Vittorio Emanuele, presidio G Rodolico, Università di Catania, Catania, Italy|Azienda Ospedaliero Meyer, Firenze, Italy|Azienda Ospedaliero San Gerardo di Monza, Monza, Italy|Federico II University Hospital, Naples, Italy|Hospital Kuala Lumpur, Kuala Lumpur, Malaysia|Academic Medical Center - University of Amsterdam, Amsterdam, Netherlands|Rotterdam University Hospital Medical Centre, Rotterdam, Netherlands|The Children Memorial Health Institute, Warszawa, Poland|Hospital Pediátrico de Coimbra (Centro Hospitalar de Coimbra), Coimbra, Portugal|Centro Hospitalar Lisboa Norte, Lisboa, Portugal|Centro Hospitalar Lisboa Centro - Hospital de Dona Estefânia, Lisbon, Portugal|San Jorge Children's Hospital, Centro de Investigaciones Clinicas, San Juan, Puerto Rico|Changhua Christian Hospital (CCH), Changhua City, Changhua County, Taiwan|Kaohsiung Medical University Chung-Ho Memorial Hospital, Kaohsiung, San-Min District, Taiwan|Chang Gung Memorial Hospital, Taoyuan City, Taoyuan County, Taiwan|Chang Gung Memorial Hospital Kaohsiung Branch, Kaohsiung City, Taiwan|Kaohsiung Veterans General Hospital, Kaohsiung, Taiwan|China Medical University and Hospital, Taichung, Taiwan|Taichung Veterans General Hospital, Taichung, Taiwan|National Cheng Kung University (NCKU) Hospital, Tainan, Taiwan|National Taiwan University Hospital, Taipei, Taiwan|MacKay Memorial Hospital, Taipei, Taiwan|Belfast City Hospital, Belfast, United Kingdom|University Hospital Birmingham - Selly Oaks, Birmingham, United Kingdom|Birmingham Children's Hospital, Birmingham, United Kingdom|Royal Free NHS Foundation Trust, London, United Kingdom|National Hospital for Neurology and Neurosurgery, London, United Kingdom|Great Ormond Street Hospital NHS Foundation Trust, London, United Kingdom|Royal Manchester Children's Hospital - Manchester University NHS Foundation Trust, Manchester, United Kingdom|Salford Royal NHS Foundation Trust, Salford, United Kingdom | https://ClinicalTrials.gov/show/NCT02294877 | |
| 126 | A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers | Completed | No Results Available | Mucopolysaccharidosis Type II|Metabolic Diseases | Biological: GNR-055 | FSAEI HE "I.M. Sechenov First Moscow State Medical University" of the Ministry of Health of Russian Federation, Moscow, Russian Federation | https://ClinicalTrials.gov/show/NCT04539340 | |
| 127 | A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I | Completed | No Results Available | Cognitive Decline|Mucopolysaccharidosis I|Hurler-Scheie Syndrome|Scheie Syndrome|Lysosomal Storage Disease | Drug: laronidase | Children's Hospital & Research Center Oakland, Oakland, California, United States|Los Angeles Biomedical Institute at Harbor-UCLA, Torrance, California, United States|University of Minnesota, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT00852358 | |
| 128 | Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I | Terminated | No Results Available | Spinal Cord Compression|Mucopolysaccharidosis I|Hurler-Scheie Syndrome|Scheie Syndrome|Lysosomal Storage Disease | Drug: laronidase | Los Angeles Biomedical Research Institute at Harbor-UCLA, Torrance, California, United States|Helsinki University Central Hospital, Helsinki, Finland | https://ClinicalTrials.gov/show/NCT00786968 | |
| 129 | Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy | Completed | No Results Available | Mucopolysaccharidosis Type III A|Sanfilippo Disease Type A | Genetic: SAF-301 | Hôpitaux Universitaires Paris Sud (Bicêtre), Le Kremlin-Bicêtre, France | https://ClinicalTrials.gov/show/NCT02053064 | |
| 130 | Intracerebral Gene Therapy for Sanfilippo Type A Syndrome | Completed | No Results Available | Mucopolysaccharidosis Type III A|Sanfilippo Disease Type A | Genetic: SAF-301 | Hôpital Bicêtre - Assistance Publique des Hôpitaux de Paris, Le Kremlin Bicêtre, France|Hôpital Necker, Assistance Publique des Hôpitaux de Paris, Paris, France | https://ClinicalTrials.gov/show/NCT01474343 | |
| 131 | Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA) | Terminated | Has Results | Sanfilippo Syndrome | Biological: rhHNS-10 mg|Biological: rhHNS-45 mg|Biological: rhHNS-90 mg | Emma Children's Hospital, Academic Medical Center, Amsterdam, Netherlands|St. Mary's Hospital, Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT01299727 | |
| 132 | Psychological Concomitants of Morquio A Syndrome - Longitudinal Effects of Enzyme Replacement Therapy (The MAPLE Study) | Completed | No Results Available | Morquio A Syndrome|Mucopolysaccharidosis IV A | | Emory University, Decatur, Georgia, United States | https://ClinicalTrials.gov/show/NCT02208661 | |
| 133 | Hunter Outcome Survey (HOS) | Recruiting | No Results Available | Hunter Syndrome | | Shire, Lexington, Massachusetts, United States | https://ClinicalTrials.gov/show/NCT03292887 | |
| 134 | Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease | Completed | Has Results | Sanfilippo Syndrome | Drug: Recombinant human heparan N-sulfatase [rhHNS] | Los Angeles Biomedical Research, Torrance, California, United States|University of Minnesota Department of Pediatrics, Minneapolis, Minnesota, United States|University of North Carolina, Chapel Hill, North Carolina, United States|Hospital Universitario Austral A Unidad de Investigacion, Buenos Aires, Argentina|Chu Bicetre, Le Kremlin-Bicêtre, Paris, France|Universitätsklinikum Hamburg Eppendorf, Hamburg, Germany|U.O.S Malattie Metaboliche Rare Clinical Pediatrica, Monza, Italy|Academisch Medisch Centrum, Amsterdam, Netherlands|Hospital Vall D'Hebron, Barcelona, Spain|Great Ormond Street Hospital, London, United Kingdom | https://ClinicalTrials.gov/show/NCT02060526 | |
| 135 | Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age | Active, not recruiting | No Results Available | Hunter Syndrome | Drug: Elaprase for intravenous (IV) infusion | Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States|Hospital Infantil Dr Robert Reid Cabral, Santo Domingo, Dominican Republic|Universitätsmedizin der Johannes Gutenberg-Universität Mainz, Mainz, Germany|Hospital Kuala Lumpur, Kuala Lumpur, Malaysia|Philippine General Hospital, Manila, Philippines|Mother and Child Health Care Institute of Serbia Dr Vukan Cupic, Beograd, Serbia|Chulalongkorn University, Bangkok, Thailand|National Pediatrics Hospital, Hanoi, Vietnam | https://ClinicalTrials.gov/show/NCT02455622 | |
| 136 | Neurobehavioral Phenotypes in MPS III | Completed | No Results Available | Sanfilippo Syndrome Type A|Sanfilippo Syndrome Type B|Hurler Syndrome | | University of Minnesota, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT01873911 | |
| 137 | Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX | Enrolling by invitation | No Results Available | Hemophilia B|Mucopolysaccharidosis I|Mucopolysaccharidosis II | Biological: SB-318|Biological: SB-913|Biological: SB-FIX | Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States | https://ClinicalTrials.gov/show/NCT04628871 | |
| 138 | A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA) | Completed | Has Results | Sanfilippo Syndrome Type A | Other: assessment | University of Minnesota, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT01047306 | |
| 139 | A Study of GC1111 in Hunter Syndrom Patients | Recruiting | No Results Available | Hunter Syndrome | Combination Product: GC1111|Combination Product: Comparator | Samsug Medical Center, Seoul, Korea, Republic of | https://ClinicalTrials.gov/show/NCT03920540 | |
| 140 | Biomarker for Hurler Disease (BioHurler) | Active, not recruiting | No Results Available | Mucopolysaccharidosis Type I|Gargoylism|Metabolism, Inborn Errors | | Children's Hospital, Faculty of Medicine, Ain Shams University, Cairo, Egypt|Centogene AG, Rostock, Germany|Amrita Institute of Medical Sciences & Research Centre, Cochin, Kerala, India|Navi Mumbai Institute of Research In Mental And Neurological Handicap (NIRMAN), Mumbai, India|Lady Ridgeway Hospital for Children, Colombo 8, Sri Lanka | https://ClinicalTrials.gov/show/NCT02298712 | |
| 141 | Intrathecal Enzyme Replacement for Hurler Syndrome | Completed | No Results Available | Hurler Syndrome | Drug: IRT Laronidase | University of Minnesota, Fairview, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT00638547 | |
| 142 | Efficacy and Safety of Enteric-coated Mycophenolate Sodium in Heart Transplant Recipients | Completed | No Results Available | Patients Successfully Completing the 12-month Treatment Period of the Core Study (de Novo Heart Recipients) Who Were Interested of Being Treated With EC-MPS | Drug: EC-MPS | | https://ClinicalTrials.gov/show/NCT00238446 | |
| 143 | Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children | Completed | No Results Available | Mucopolysaccharidosis Type I (MPS I)|Mucopolysaccharidosis Type II (MPS II)|Mucopolysaccharidosis Type III (MPS III)|Mucopolysaccharidosis Type VI (MPS VI)|Krabbe Disease | | University of Chicago, Chicago, Illinois, United States|University of Minnesota, Minneapolis, Minnesota, United States|Hunter James Kelly Institute, Buffalo, New York, United States | https://ClinicalTrials.gov/show/NCT01938014 | |
| 144 | Efficacy and Safety of Enteric-Coated Mycophenolate Sodium (EC-MPS) in Renal Transplant Patients | Completed | No Results Available | Kidney Transplantation | Drug: Enteric-Coated Mycophenolate Sodium (EC-MPS) | Novartis, Basel, Switzerland | https://ClinicalTrials.gov/show/NCT00239083 | |
| 145 | Safety and Efficacy of Enteric-coated Mycophenolate Sodium (EC-MPS) Plus Valsartan in Patients With Kidney Transplants (MYTHOS | Completed | No Results Available | De Novo Renal Transplantation | Drug: Enteric-coated Mycophenolate sodium (EC-MPS), valsartan | | https://ClinicalTrials.gov/show/NCT00308425 | |
| 146 | Study of Enteric-coated Mycophenolate Sodium (EC-MPS) With Steroid Withdrawal vs EC-MPS With Standard Steroid Regimen in de Novo Renal Transplant Recipients. | Completed | No Results Available | Kidney de Novo Transplant | Drug: Enteric-coated Mycophenolate sodium (EC-MPS) | | https://ClinicalTrials.gov/show/NCT00238992 | |
| 147 | An Open Label Study to Evaluate the Tolerability and Safety of Enteric-coated Mycophenolate Sodium (EC-MPS) in Combination With Cyclosporine Microemulsion (CsA-ME) in Maintenance Renal Transplant Recipients | Completed | No Results Available | Kidney Transplant Recipients | Drug: Enteric-coated Mycophenolate sodium (EC-MPS) | | https://ClinicalTrials.gov/show/NCT00239044 | |
| 148 | Study of Enteric-Coated Mycophenolate Sodium (EC-MPS) Plus Reduced-dose Cyclosporine Microemulsion (CsA-ME) Compared to EC-MPS Plus Standard Dose CsA-ME in Eldery de Novo Renal Transplant Recipients Treated With Basiliximab and Short-term Steroids | Completed | No Results Available | Denovo Renal Transplantation | Drug: Enteric-Coated Mycophenolate Sodium | | https://ClinicalTrials.gov/show/NCT00239031 | |
| 149 | Biomarkers for Hunter Syndrome | Recruiting | No Results Available | Hunter Syndrome|Mucopolysaccharidosis II|Hunter's Syndrome, Mild Form|Hunter's Canal Syndrome | | Private Practice, Cancun, Quintana Roo, Mexico|Hospital Pediatrico de Sinaloa, Culiacán, Sinaloa, Mexico|Centenario Hospital Miguel Hidalgo, Aguascalientes, Mexico|Hospital Infantil de Tampaulipas, Ciudad Victoria, Mexico | https://ClinicalTrials.gov/show/NCT01330277 | |
| 150 | Study to Evaluate the Combination of Enteric-coated Mycophenolate Sodium (EC-MPS), Basiliximab, and C2-monitored Cyclosporine in de Novo Renal Transplant Recipients at Potential High Risk of Delayed Graft Function (DGF) | Completed | No Results Available | Renal Transplantation | Drug: Enteric-Coated Mycophenolate Sodium (EC-MPS) | | https://ClinicalTrials.gov/show/NCT00154232 | |
| 151 | Investigation of the Effectiveness of Muscle Inhibition and Space Correction Techniques of Kinesiotaping Method in Female Patients With Myofascial Pain Syndrome Related to Upper Trapezius Active Trigger Points | Completed | No Results Available | Female Patients With Myofascial Pain Syndrome (MPS) Related to Upper Trapezius Active Trigger Points (TP) | Other: Kinesiotaping with space correction technique|Other: Kinesiotaping with muscle inhibition technique|Other: Home exercise program | | https://ClinicalTrials.gov/show/NCT03949010 | |
| 152 | A Study of Potential Treatment-Responsive Biomarkers in Hunter Syndrome | Recruiting | No Results Available | Hunter Syndrome | Other: No Intervention | UCSF Benioff Children's Hospital, Oakland, California, United States|NYU School of Medicine, New York, New York, United States|UNC Children's Research Institue, Chapel Hill, North Carolina, United States|UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States|UKGM Zentrum fur Kinderheilkune/Kinderneurologie, Giessen, Germany|Manchester Centre for Genomic Medicine, Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT04007536 | |
| 153 | Biomarker for Sly Disease (MPS VII) (BioSly) | Active, not recruiting | No Results Available | Developmental Delay|Skeletal Abnormalities|Hepatomegaly|Splenomegaly | | Children's Hospital, Faculty of Medicine, Ain Shams University, Cairo, Egypt|Centogene AG, Rostock, Germany|Amrita Institute of Medical Sciences & Research Centre, Cochin, Kerala, India|Navi Mumbai Institute of Research In Mental And Neurological Handicap (NIRMAN), Mumbai, India|Lady Ridgeway Hospital for Children, Colombo 8, Sri Lanka | https://ClinicalTrials.gov/show/NCT02298699 | |
| 154 | Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome | Completed | No Results Available | Morquio Syndrome A|Maroteaux Lamy Syndrome|MPS IVA|MPS VI | | Greenwood Genetic Center, Greenville, South Carolina, United States | https://ClinicalTrials.gov/show/NCT01961518 | |
| 155 | BIOCLEAN MPS (Multi-Purpose Solution) VII in Combination With Biofinity Lens: A Two Week Crossover Study | Completed | Has Results | Myopia | Device: Bioclean MPS VII|Device: Aosept Clearcare|Device: comfilcon A | Kodama Eye Clinic, Joyo-shi, Kyoto-fu, Japan|Higashihara Eye Clinic, Kameoka, Kyoto-fu, Japan|Iwasaki Eye Clinic, Osaka-shi, Osaka-fu, Japan | https://ClinicalTrials.gov/show/NCT02537730 | |
| 156 | Enteric-coated Mycophenolate Sodium (EC-MPS) and Mycophenolate Mofetil (MMF) in Renal Transplant Patients With Gastrointestinal (GI) Intolerance | Completed | Has Results | Renal Transplantation | Drug: Enteric-coated mycophenolate sodium (EC-MPS)|Drug: Mycophenolate mofetil|Drug: Placebo to mycophenolate sodium|Drug: Placebo to mycophenolate mofetil | AKDHC Medical Research Services, LLC, Phoenix, Arizona, United States|University of Southern California, Los Angeles, California, United States|Cedars-Sinai Medical Center, Los Angeles, California, United States|National Institute of Transplantation, Los Angeles, California, United States|David Geffen School of Medicine at UCLA, Los Angeles, California, United States|UC Davis Medical Center, Sacramento, California, United States|University of California San Diego, San Diego, California, United States|University of California, San Diego, San Diego, California, United States|University of California, San Francisco, California, United States|Denver Nephrology, Denver, Colorado, United States|University of Colorado Health Science Center, Denver, Colorado, United States|Yale University Transplantation, New Haven, Connecticut, United States|Washington Hospital Transplant Research, Washington, District of Columbia, United States|University of Florida College of Medicine, Gainesville, Florida, United States|Piedmont Hospital, Atlanta, Georgia, United States|Medical College of Georgia, Augusta, Georgia, United States|University of Chicago Medical Center, Chicago, Illinois, United States|Loyola University Medical Center, Maywood, Illinois, United States|Clarian Health Partners, Indianapolis, Indiana, United States|Univ of KS Medical Ctr, Kansas City, Kansas, United States|University of Kentucky Medical Center, Lexington, Kentucky, United States|Northwest Louisiana Nephrology Research, Shreveport, Louisiana, United States|WKHS/LSUHSC Regional Transplant Center, Shreveport, Louisiana, United States|University of Maryland, Baltimore, Maryland, United States|The Johns Hopkins Hospital, Baltimore, Maryland, United States|Mid-Atlantic Nephrology Associates, Baltimore, Maryland, United States|Brigham and Women's Hospital, Boston, Massachusetts, United States|Henry Ford Health System, Detroit, Michigan, United States|St. John Hospital Medical Center, Detroit, Michigan, United States|University of Minnesota, Minneapolis, Minnesota, United States|St. Luke's Hospital of Kansas City, Kansas City, Missouri, United States|Washington University School of Medicine, St. Louis, Missouri, United States|University of Nebraska Medical Center, Omaha, Nebraska, United States|St. Baranabas Medical Center, Livingston, New Jersey, United States|Montefiore Medical Center, Bronx, New York, United States|UNC Chapel Hill, Chapel Hill, North Carolina, United States|East Carolina University, Greenville, North Carolina, United States|Wake Forest, Winston-Salem, North Carolina, United States|University Hospitals of Cleveland, Cleveland, Ohio, United States|Cleveland Clinic, Cleveland, Ohio, United States|Oregon Health & Science University, Portland, Oregon, United States|Pinnacle Health at Harrisburg Hospital, Harrisburg, Pennsylvania, United States|Thomas Jefferson University Hospital, Philadelphia, Pennsylvania, United States|Rhode Island Hospital, Providence, Rhode Island, United States|Medical University of South Carolina, Charleston, South Carolina, United States|The University of Tennessee Health Science Center, Memphis, Tennessee, United States|Vanderbilt University Medical Center, Nashville, Tennessee, United States|CRSTI, Dallas, Texas, United States|Baylor All Saints, Fort Worth, Texas, United States|University of Texas Medical Branch, Galveston, Texas, United States|University of Utah Hospitals and Clinics, Salt Lake City, Utah, United States|Virginia Commonwealth University, Richmond, Virginia, United States|University of Washington, Seattle, Washington, United States | https://ClinicalTrials.gov/show/NCT00400400 | |
| 157 | Evaluation of an Investigational Multi-Purpose Solution (MPS) | Completed | Has Results | Study Focus is Healthy Contact Lens Wearers | Device: Investigational MPS|Device: Opti-Free | Complete Family Vision Center, San Diego, California, United States|Eye Care Associates of Hawaii, Waipahu, Hawaii, United States|Insight Eyecare, Warrensburg, Missouri, United States|Drs. Quinn, Quinn & Associates, Athens, Ohio, United States|Western Reserve Vision Care, Beachwood, Ohio, United States|Central Ohio Eyecare, Columbus, Ohio, United States|Northeastern Eye Institute, Scranton, Pennsylvania, United States|West Bay Eye Associates, Warwick, Rhode Island, United States|Ziegler Leffingwell Eyecare, West Allis, Wisconsin, United States | https://ClinicalTrials.gov/show/NCT00961051 | |
| 158 | Efficacy of Heat-shock Protein (HSP) Inhibitors in Myeloproliferative Syndromes (MPS) | Completed | No Results Available | Myeloproliferative Syndrome | Biological: Blood sample|Other: Flow cytometry|Other: western blot | CHU Dijon Bourgogne, Dijon, France | https://ClinicalTrials.gov/show/NCT02873832 | |
| 159 | Acute Clinical Evaluation of Multi-purpose Solutions (MPS) in Soft Contact Lens Wearers | Completed | No Results Available | Myopia | Device: Contact lens multi-purpose solution|Other: Saline/blister pack solution|Device: Contact lenses | Visioncare Research, Ltd., Farnham, Surrey, United Kingdom | https://ClinicalTrials.gov/show/NCT00929487 | |
| 160 | Comparison of a New Abbott Medical Optics (AMO) Multi-Purpose Solution (MPS) to Existing Contact Lens Regimens | Completed | Has Results | Normal Contact Lens Wearers | Device: Multi-purpose disinfecting solution|Device: Clear Care|Device: Opti-Free RepleniSH | Centre for Contact Lens Research, Waterloo, Ontario, Canada | https://ClinicalTrials.gov/show/NCT01294917 | |
| 161 | Efficacy and Safety of Enteric-Coated Mycophenolate Sodium (EC-MPS) in Kidney Transplant Recipients | Completed | No Results Available | Kidney Transplantation | Drug: EC-MPS, Tacrolimus | Novartis, East Hanover, New Jersey, United States | https://ClinicalTrials.gov/show/NCT00229138 | |
| 162 | Patient Reported Outcomes in Renal Transplant Patients Tolerating Gastrointestinal (GI) Symptoms Converted to Myfortic (EC-MPS) | Unknown status | No Results Available | Kidney Transplant | Drug: Myfortic | Foothills Medical Centre, Calgary, Alberta, Canada | https://ClinicalTrials.gov/show/NCT00676221 | |
| 163 | The Efficacy of Enteric-coated Mycophenolate Sodium (EC-MPS) (Myfortic) in The Treatment of Relapse or Resistant Proliferative Lupus Nephritis | Terminated | No Results Available | Lupus Nephritis | Drug: mycophenolate sodium|Drug: cyclophosphamide | Nopparat Rajathani, Bangkok, Thailand|Khon Kaen University, Khon Kaen, Thailand|Thammasart University, Pathumthani, Thailand | https://ClinicalTrials.gov/show/NCT01015456 | |
| 164 | Study of the Presence and Extent of Corneal Disturbance Associated With B+L Biotrue MPS Used With B+L PureVision Lenses | Completed | Has Results | Keratitis | Device: B+L Biotrue MPS and B+L PureVision | Joel J. Ackerman, OD, PC, Phoenix, Arizona, United States | https://ClinicalTrials.gov/show/NCT01268306 | |
| 165 | A Study of CellCept (Mycophenolate Mofetil) in Kidney Transplant Patients Switched From EC-MPS. | Terminated | No Results Available | Kidney Transplantation | Drug: mycophenolate mofetil [CellCept] | Berlin, Germany|Berlin, Germany|Dresden, Germany|Essen, Germany|Hannover, Germany|Köln, Germany|Münster, Germany | https://ClinicalTrials.gov/show/NCT00420472 | |
| 166 | Efficacy and Safety of Everolimus+EC-MPS After Early CNI Elimination vs EC-MPS +Tacrolimus in Renal Transplant Recipients | Completed | Has Results | Chronic Renal Failure | Drug: Everolimus|Drug: Tacrolimus (FK506)|Drug: Basiliximab|Drug: Enteric Coated Mycophenolate Sodium (EC-MPS)|Drug: Corticosteroids | Novartis Investigative Site, Haifa, Israel|Novartis Investigative Site, Petach Tikva, Israel|Novartis Investigative Site, Tel-Aviv, Israel | https://ClinicalTrials.gov/show/NCT00965094 | |
| 167 | Efficacy and Safety of Enteric-coated Mycophenolate Sodium (EC-MPS) in de Novo Kidney Transplant Recipients | Completed | No Results Available | De Novo Kidney Transplant Recipients | Drug: Enteric-coated Mycophenolate sodium (EC-MPS) | | https://ClinicalTrials.gov/show/NCT00312143 | |
| 168 | Efficacy and Safety of Enteric-coated Mycophenolate Sodium (EC-MPS) in de Novo Kidney Transplant Recipients | Completed | No Results Available | Renal Transplantation | Drug: Enteric-Coated Mycophenolate Sodium (EC-MPS) | Novartis, Basel, Switzerland | https://ClinicalTrials.gov/show/NCT00238953 | |
| 169 | A Prospective Study to Investigate Mycophenolic Acid (MPA) Exposure Through Area Under the Curve (AUC) in Renal Transplants Recipients Treated With Mycophenolate Mofetil (MMF) and After Conversion to Mycophenolate Sodium (EC-MPS) | Completed | No Results Available | Renal Transplantation | Drug: Mycophenolate sodium|Drug: Mycophenolate mofetil | Irmandade Da Santa Casa de Misericórdia de Porto Alegre, Porto Alegre, Rio Grande do Sul, Brazil | https://ClinicalTrials.gov/show/NCT01822483 | |
| 170 | Gastrointestinal Tolerability of MMF vs EC-MPS in Maintenance Transplant Patients Treated With Calcineurin Inhibitors | Unknown status | No Results Available | Organ Transplantation | Drug: MMF|Drug: EC-MPS | Toronto General Hospital - Multi Organ Transplant Program, Toronto, Ontario, Canada | https://ClinicalTrials.gov/show/NCT00611494 | |
| 171 | Efficacy and Safety of Enteric-coated Mycophenolate Sodium (EC-MPS) in a Cyclosporine Microemulsion Based Regimen in de Novo Living Donor Kidney Transplant Recipients | Completed | No Results Available | Kidney Transplant | Drug: Enteric-Coated Mycophenolate Sodium (EC-MPS) | Novartis Investigative Site, Busan, Korea, Republic of|Novartis Investigative Site, Daegu, Korea, Republic of|Novartis Investigative Site, Daegu, Korea, Republic of|Novartis Investigative Site, Jeollanam-do, Korea, Republic of|Novartis Investigative Site, Seoul, Korea, Republic of|Novartis Investigative Site, Seoul, Korea, Republic of|Novartis Investigative Site, Seoul, Korea, Republic of|Novartis Investigative Site, Seoul, Korea, Republic of | https://ClinicalTrials.gov/show/NCT00537862 | |
| 172 | Study of Enteric-coated Mycophenolate Sodium (EC-MPS) With Cyclosporine Microemulsion and Steroids in Pediatric de Novo Renal Transplant Patients | Completed | No Results Available | Kidney Transplantation | Drug: Enteric-Coated Mycophenolate Sodium (EC-MPS) | Novartis, Basel, Switzerland | https://ClinicalTrials.gov/show/NCT00154206 | |
| 173 | Conversion Trial From Mycophenolate Mofetil (MMF) to Enteric-coated Mycophenolate Sodium (EC-MPS) in Stable Transplanted Patients Suffering From Gastrointestinal (GI) Adverse Events While on Mycophenolate Mofetil Therapy (MMF) Therapy | Completed | No Results Available | Kidney Transplantation|Liver Transplantation | Drug: Enteric-coated Mycophenolate sodium (EC-MPS) | Novartis, Basel, Switzerland | https://ClinicalTrials.gov/show/NCT00149942 | |
| 174 | Efficacy and Safety of Everolimus in Combination With Cyclosporine Microemulsion Versus Everolimus in Combination With Enteric-coated Mycophenolate Sodium (EC-MPS), in Adult Renal Transplant Patients in Maintenance. | Completed | Has Results | Renal Transplantation | Drug: Everolimus + Cyclosporine|Drug: Everolimus + Enteric-coated Mycophenolate Sodium (EC-MPS)|Drug: Steroids | Novartis Investigative Site, Paris, France | https://ClinicalTrials.gov/show/NCT00425308 | |
| 175 | Efficacy and Safety of Everolimus With Enteric-Coated Mycophenolate Sodium (EC-MPS) in a Cyclosporine Microemulsion-free Regimen Compared to Standard Therapy in de Novo Renal Transplant Patients | Completed | Has Results | Renal Transplantation | Drug: Everolimus|Drug: Cyclosporine|Drug: Enteric-coated mycophenolate sodium|Drug: Corticosteroids | Novartis Investigational Sites, Nurnberg, Germany|Novartis Pharma AG, Basel, Switzerland|Novartis Investigational Sites, Bern, Switzerland | https://ClinicalTrials.gov/show/NCT00154310 | |
| 176 | A Study to Investigate the Tolerability and Safety of Converting Stable Renal Transplant Recipients Who Receive Tacrolimus With or Without Corticosteroids From Mycophenolate Mofetil (MMF) to Enteric-coated Mycophenolate Sodium (EC-MPS) | Completed | No Results Available | Renal Transplantation | Drug: Enteric-Coated Mycophenolate Sodium (EC-MPS) | | https://ClinicalTrials.gov/show/NCT00238979 | |
| 177 | Administration of IV Laronidase Post Bone Marrow Transplant in Hurler | Completed | Has Results | Hurler Syndrome | Drug: Laronidase | Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT01173016 | |
| 178 | ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases | Terminated | No Results Available | Inherited Metabolic Diseases|Lysosomal Storage Disorders|Peroxisomal Storage Diseases|Inborn Errors of Metabolism|Mucopolysaccharidosis | Biological: ALD-101 | Mattel Children's Hospital at UCLA, Los Angeles, California, United States|Mt. Sinai Medical Center, New York, New York, United States|Duke University, Durham, North Carolina, United States | https://ClinicalTrials.gov/show/NCT00654433 | |
| 179 | Multicenter, Randomized, Open-label Study to Assess Whether Treatment With Mycophenolate Sodium (MPS) Allows Higher Dose Optimization Versus Mycophenolate Mofetil (MMF) Leading to a Dose Reduction of Tacrolimus. Maximiza Study. | Completed | Has Results | Prophilaxis of Acute Rejection in Patients Receiving a Renal Allograft | Drug: 1|Drug: 2 | Novartis Investigative Site, Granada, Andalucia, Spain|Novartis Investigative Site, Oviedo, Asturias, Spain|Novartis Investigative Site, Santander, Cantabria, Spain|Novartis Investigative Site, Toledo, Castilla la Mancha, Spain|Novartis Investigative Site, Valladolid, Castilla y Leon, Spain|Novartis Investigative Site, Barcelona, Catalunya, Spain|Novartis Investigative Site, Barcelona, Cataluña, Spain|Novartis Investigative Site, Barcelona, Cataluña, Spain|Novartis Investigative Site, Alicante, Comunidad Valenciana, Spain|Novartis Investigative Site, Valencia, Comunidad Valenciana, Spain|Novartis Investigative Site, La Coruna, Galicia, Spain|Novartis Investigative Site, Palma de Mallorca, Islas Baleares, Spain|Novartis Investigative Site, La Laguna, Las Palmas de Gran Canaria, Spain|Novartis Investigative Site, Pamplona, Navarra, Spain|Novartis Investigative Site, Barakaldo, Pais Vasco, Spain|Novartis Investigative Site, Madrid, Spain|Novartis Investigative Site, Madrid, Spain|Novartis Investigative Site, Madrid, Spain|Novartis Investigative Site, Zaragoza, Spain | https://ClinicalTrials.gov/show/NCT01056822 | |
| 180 | Combined Use of a Novel Cardioplegic Formula With Myocardial Protection System (MPS)® Versus Cardioplexol ® in Isolated Coronary Artery Bypass Grafting (CABG) Using MiECC; | Completed | No Results Available | Complication of Extracorporeal Circulation|Complication of Coronary Artery Bypass Graft | Procedure: use of a cardioplegic formula in isolated CABG (coronary artery bypass grafting) using MiECC (Minimal extracorporeal circulation | Klinik für Herzchirurgie, Universitätsspital Basel, Basel, Switzerland | https://ClinicalTrials.gov/show/NCT03612388 | |
| 181 | MYPROMS-ES02: Safety and Efficacy of Basiliximab, Cyclosporine Microemulsion and Enteric-coated Mycophenolate Sodium (EC-MPS) Versus EC-MPS and Steroid Therapy in Kidney Transplant Recipients Who Are Hepatitis C Positive | Terminated | No Results Available | De Novo Kidney Transplant | Drug: Enteric-coated Mycophenolate sodium (EC-MPS) | | https://ClinicalTrials.gov/show/NCT00284921 | |
| 182 | Intensive Pharmacovigilance Program for Elaprase (SHP ELA-701) | Completed | No Results Available | Hunter Syndrome | | Hospital Pediátrico de Sinaloa, Culiacán Rosales, México, Mexico|IMSS UMAE 25 (INSTITUTO MEXICANO DEL SEGURO SOCIAL UNIDAD MEDICA DE ALTA ESPECIALIDAD No 25), Monterrey, Nuevo León, Mexico|Hospital del Niño Tabasqueño, Villahermosa, Tabasco, Mexico|Hospital Centenario Miguel Hidalgo, Aguascalientes, Mexico|Hospital Infantil Federico Gomez, Ciudad de México, Mexico|Hospital Universitario de Colima, Colima, Mexico | https://ClinicalTrials.gov/show/NCT03582449 | |
| 183 | A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase® | Completed | Has Results | Hunter Syndrome | Other: Control|Drug: Idursulfase IT (1 mg)|Drug: Idursulfase IT (10 mg)|Drug: Idursulfase IT (30 mg) | University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States|Birmingham Children's Hospital, Birmingham, United Kingdom|Birmingham Children's Hospital NHS Foundation Trust, Birmingham, United Kingdom | https://ClinicalTrials.gov/show/NCT00920647 | |
| 184 | Naglazyme After Allo Transplant for Maroteaux-Lamy Syndrome | Active, not recruiting | No Results Available | Maroteaux-Lamy Syndrome | Drug: Naglazyme® | University of Minnesota Medical Center, Fairview, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT02156674 | |
| 185 | Combined Use of a Novel Cardioplegic Formula With MPS® Versus Cardioplexol ® in Urgent Isolated CABG Using MiECC in Patients With Recent Acute Myocardial Infarction | Completed | No Results Available | Heart Attack|Cardioplegia Solution Adverse Reaction|Coronary Artery Bypass Grafting (CABG) | Other: Myocardial protection system (MPS)|Other: Cardioplexol | Cardiac Surgery Clinic University Hospital Basel, Basel, Switzerland | https://ClinicalTrials.gov/show/NCT04309994 | |
| 186 | PMCF Study of MPS-Flex® Knee System in TKA | Not yet recruiting | No Results Available | Total Knee Arthroplasty | | | https://ClinicalTrials.gov/show/NCT04427722 | |
| 187 | Combined Use of a Novel Cardioplegic Formula With MPS ® Using the MiECC in Isolated CABG Versus OPCABG | Completed | No Results Available | Complication of Coronary Artery Bypass Graft|Complication of Extracorporeal Circulation | Procedure: CABG | Herzchirurgie University Hospital Basel, Basel, Switzerland | https://ClinicalTrials.gov/show/NCT03609723 | |
| 188 | Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome | Completed | Has Results | Hunter Syndrome | Other: No treatment | Emory University, Decatur, Georgia, United States|Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States|Children's Hospitals and Clinics of Minnesota, Minneapolis, Minnesota, United States|University of North Carolina, Division of Genetics and Metabolism, Chapel Hill, North Carolina, United States|University of Utah School of Medicine, Salt Lake City, Utah, United States|Central Manchester University Hospitals NHS Foundation Trust, St. Mary's Hospital, Manchester, United Kingdom|Salford Royal NHS Foundation Trust, Salford, United Kingdom | https://ClinicalTrials.gov/show/NCT01449240 | |
| 189 | Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation | Unknown status | No Results Available | I Cell Disease|Fucosidosis|Globoid Cell Leukodystrophy|Adrenoleukodystrophy|Mannosidosis|Niemann-Pick Disease|Pulmonary Complications|Mucopolysaccharidosis I|Mucopolysaccharidosis VI|Metachromatic Leukodystrophy|Gaucher's Disease|Wolman Disease | | Fairview University Medical Center, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT00005900 | |
| 190 | An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients | Completed | Has Results | Hunter Syndrome | Biological: Idursulfase | Children's Hospital & Research Center Oakland, Oakland, California, United States|Children's Hospitals and Clinics of Minnesota, Division of Genetics, Minneapolis, Minnesota, United States|Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica, Porto Alegre, RS, Brazil|Birmingham Children's Hospital, Birmingham, United Kingdom|Great Ormond Street Hospital, London, United Kingdom|Central Manchester University Hospitals, St. Mary's Hospital, Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT00882921 | |
| 191 | Efficacy and Safety of Enteric-coated Mycophenolate Sodium (EC-MPS) in Comparison to Mycophenolate Mofetil (MMF) in de Nove Heart Patients | Completed | No Results Available | Heart Transplantation | Drug: Enteric-coated Mycophenolate Sodium | Novartis Investigative site, Los Angeles, California, United States|University of Minnesota, Minneapolis, Minnesota, United States|Novartis Pharmaceuticals, Philadelphia, Pennsylvania, United States|Argentina, Buenos Aires, Argentina|Site 1: X5000BJH, Cordoba, Argentina|Novartis Investigative site, Sydney, Australia, Sydney, Australia|Novartis Investigative, Edmonton, Alberta, Canada|Novartis, Toronto, Ontario, Canada|Novarits, Ottawa, Canada | https://ClinicalTrials.gov/show/NCT00574743 | |
| 192 | Ultrasonographic Assessment of Carpal Tunnel Syndrome | Completed | No Results Available | Carpal Tunnel Syndrome | | University of Utah/Primary Children's Medical Center, Salt Lake City, Utah, United States | https://ClinicalTrials.gov/show/NCT02196220 | |
| 193 | Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome | Completed | No Results Available | Sanfilippo Syndrome B | Drug: rAAV2/5-hNAGLU | Hopitaux Universitaires Paris-Sud, Paris, Le Kremlin-Bicetre Cedex, France | https://ClinicalTrials.gov/show/NCT03300453 | |
| 194 | Establishment of Human Cellular Disease Models for Morquio Disease | Completed | No Results Available | Morquio Disease | | Childrens Hospital and Institute of Child Health, Ferozepur Road, Lahore, Pakistan | https://ClinicalTrials.gov/show/NCT03872713 | |
| 195 | Extension Study on the Safety and Efficacy of Enteric-coated Mycophenolate Sodium in Kidney Transplant Recipients | Completed | No Results Available | Renal Transplantation | Drug: Enteric-Coated Mycophenolate Sodium (EC-MPS) | | https://ClinicalTrials.gov/show/NCT00239070 | |
| 196 | Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094 | Active, not recruiting | No Results Available | Hunter Syndrome | Drug: Idursulfase-IT|Drug: Elaprase | Children's Hospital and Research Center at Oakland, Oakland, California, United States|Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, Illinois, United States|University of North Carolina, Chapel Hill, North Carolina, United States|Women's and Children's Hospital, Adelaide, Australia|The Hospital for Sick Children, Toronto, Ontario, Canada|Hôpital Femme Mère Enfants, Bron, France|Instituto Nacional de PediatrÃa, Coyoacan, Ciudad De México, Mexico|Hospital Infantil Universitario Niño Jesus, Madrid, Spain|Royal Manchester Children's Hospital, Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT02412787 | |
| 197 | Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products | Recruiting | No Results Available | Severe Combined Immunodeficiency|Malignancy, Hematologic|Neuroblastoma|Neoplasm|Mucopolysaccharidosis I | Procedure: Venipuncture | St. Jude Children's Research Hospital, Memphis, Tennessee, United States | https://ClinicalTrials.gov/show/NCT00695279 | |
| 198 | Study of Enteric-coated Mycophenolate Sodium Maintenance Therapy in Patients With Renal Transplant Receiving Cyclosporine Microemulsion and Steroids, | Completed | No Results Available | Maintenance Renal Transplantation | Drug: Enteric-Coated Mycophenolate Sodium | | https://ClinicalTrials.gov/show/NCT00239057 | |
| 199 | Safety and Efficacy of Hunterase | Completed | No Results Available | Hunter Syndrome | Biological: Hunterase | Samsug Medical Center, Seoul, Korea, Republic of | https://ClinicalTrials.gov/show/NCT01645189 | |
| 200 | Longitudinal Studies of the Glycoproteinoses | Unknown status | No Results Available | Aspartylglucosaminuria|Fucosidosis|Galactosialidosis|Alpha Mannosidosis|Beta Mannosidosis|Mucolipidosis II|Mucolipidosis III|Schindler Disease|Sialidosis | | Greenwood Genetic Center, North Charleston, South Carolina, United States | https://ClinicalTrials.gov/show/NCT01891422 | |
| 201 | Extension Study of the Safety and Efficacy of Enteric-coated Mycophenolate Sodium in Kidney Transplant Recipients | Completed | No Results Available | Renal Transplantation | Drug: Enteric-Coated Mycophenolate Sodium (EC-MPS) | | https://ClinicalTrials.gov/show/NCT00241059 | |
| 202 | Extension Study of Enteric-coated Mycophenolate Sodium With Short-term or no Steroid Use Compared With Enteric-coated Mycophenolate Sodium With Standard Steroid Therapy in de Novo Kidney Recipients | Completed | No Results Available | Renal Transplantation | Drug: Enteric-coated Mycophenolate sodium (EC-MPS) | | https://ClinicalTrials.gov/show/NCT00240955 | |
| 203 | Extension Study to Assess Tolerability and Safety of Enteric-coated Mycophenolate Sodium in Renal Transplant Recipients Who Received Mycophenolate Mofetil | Completed | No Results Available | Renal Transplantation | Drug: Enteric-Coated Mycophenolate Sodium (EC-MPS) | | https://ClinicalTrials.gov/show/NCT00238966 | |
| 204 | Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase® | Completed | Has Results | Hunter Syndrome | Behavioral: Neurobehavioral testing|Other: Visual and auditory assessments | University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States|Birmingham Children's Hospital, Birmingham, United Kingdom | https://ClinicalTrials.gov/show/NCT00937794 | |
| 205 | Morquio's Syndrome: a Case Study | Terminated | No Results Available | Morquio's Disease | Other: physiotherapeutic treatment | | https://ClinicalTrials.gov/show/NCT00609440 | |
| 206 | Extension Study of Enteric-coated Mycophenolate Sodium in Combination With Full Dose or Reduced Dose Cyclosporine Microemulsion in Patients With de Novo Kidney Transplants | Completed | No Results Available | Renal Transplantation | Drug: Enteric-Coated Mycophenolate Sodium (EC-MPS) | | https://ClinicalTrials.gov/show/NCT00238940 | |
| 207 | Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment | Completed | Has Results | Hunter Syndrome | Biological: idursulfase-IT|Other: No IT treatment | Children's Hospital and Research Center at Oakland, Oakland, California, United States|Ann & Robert H Lurie Children's Hospital of Chicago, Chicago, Illinois, United States|University of North Carolina, Chapel Hill, North Carolina, United States|Women's and Children's Hospital, 72 King William Road, North Adelaide, Australia|The Hospital for Sick Children, Toronto, Ontario, Canada|Hôpital Femme Mère Enfant, Bron, France|Instituto Nacional de PediatrÃa, Coyoacan, Ciudad De México, Mexico|Hospital Infantil Universitario Niño Jesus, Madrid, Spain|Royal Manchester Children's Hospital, Manchester, United Kingdom | https://ClinicalTrials.gov/show/NCT02055118 | |
| 208 | Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment | Active, not recruiting | No Results Available | Hunter Syndrome | Drug: Idursulfase-IT|Drug: Elaprase | Ann & Robert H Lurie Childrens Hospital of Chicago, Chicago, Illinois, United States|University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States|Legacy Emanuel Hospital, Portland, Oregon, United States|Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, United States|Vanderbilt Children's Hospital, Nashville, Tennessee, United States|University of Utah Hospital, Salt Lake City, Utah, United States|Seattle Children's Hospital, Seattle, Washington, United States|British Columbia Children's Hospital, Vancouver, British Columbia, Canada|Birmingham Children's Hospital, Birmingham, United Kingdom | https://ClinicalTrials.gov/show/NCT01506141 | |
| 209 | Safety and Tolerability of Enteric-Coated Mycophenolate Sodium (EC-MPS) in Renal Transplant Patients With GI Intolerance | Completed | No Results Available | Renal Transplantation | Drug: Enteric-coated mycophenolate sodium (EC-MPS) | Novartis, East Hanover, New Jersey, United States | https://ClinicalTrials.gov/show/NCT00150020 | |
| 210 | Paraspinal Stimulation in Treating MPS | Unknown status | No Results Available | Clinical Study - RCT | Procedure: DIMMST | Hospital de ClÃnicas de Porto Alegre, Porto Alegre, RS, Brazil|Hospital de ClÃnicas de Porto Alegre, Porto Alegre, RS, Brazil|Hospital de ClÃnicas E Porto Alegre, Porto Alegre, RS, Brazil | https://ClinicalTrials.gov/show/NCT01708343 | |
| 211 | Efficacy and Safety of Enteric-coated Mycophenolate Sodium and Cyclosporine in Combination With and Without Steroids, in Adult Renal Transplant Recipients | Completed | Has Results | Renal Transplantation | Drug: Enteric-coated mycophenolate sodium (EC-MPS)|Drug: Prednisone | C.H.U. La Milétrie, Poitiers, France | https://ClinicalTrials.gov/show/NCT00413920 | |
| 212 | The Effects of Whey vs. Collagen on MPS | Completed | No Results Available | Aging | Dietary Supplement: Collagen peptide|Dietary Supplement: Whey protein isolate | Exercise Metabolism Research Laboratory, McMaster Univeristy, Hamilton, Ontario, Canada|Ivor Wynne Center, Hamilton, Ontario, Canada | https://ClinicalTrials.gov/show/NCT03281434 | |
| 213 | Daily Protein Intake Distribution and MPS (ARCIII) | Completed | No Results Available | Overweight | Other: whey protein|Other: placebo | | https://ClinicalTrials.gov/show/NCT02315014 | |
| 214 | Enteric-coated Mycophenolate Sodium (EC-MPS) With Reduced-dose Tacrolimus Versus EC-MPS With Standard-dose Tacrolimus in Stable Kidney Transplant Recipients | Completed | Has Results | Kidney Diseases | Drug: Enteric-coated mycophenolate sodium (EC-MPS)|Drug: Tacrolimus|Drug: Corticosteroids | Novartis, Basel, Switzerland | https://ClinicalTrials.gov/show/NCT00284934 | |
| 215 | Human Placental-Derived Stem Cell Transplantation | Active, not recruiting | No Results Available | Mucopolysaccharidosis I|Mucopolysaccharidosis VI|Adrenoleukodystrophy|Niemann-Pick Disease|Metachromatic Leukodystrophy|Wolman Disease|Krabbe's Disease|Gaucher's Disease|Fucosidosis|Batten Disease|Severe Aplastic Anemia|Diamond-Blackfan Anemia|Amegakaryocytic Thrombocytopenia|Myelodysplastic Syndrome|Acute Myelogenous Leukemia|Acute Lymphocytic Leukemia | Drug: Human Placental Derived Stem Cell | Children's Hospital Colorado, Denver, Colorado, United States|New York Medical College, Valhalla, New York, United States|University of Utah, Salt Lake City, Utah, United States | https://ClinicalTrials.gov/show/NCT01586455 | |
| 216 | HSCT for High Risk Inherited Inborn Errors | Completed | Has Results | Adrenoleukodystrophy|Metachromatic Leukodystrophy|Globoid Cell Leukodystrophy|Tay Sachs Disease|Sandhoffs Disease|Wolman Disease|I-Cell Disease|Sanfilippo Syndrome|GM1 Gangliosidosis | Drug: Clofarabine|Procedure: Total body Irradiation|Drug: Melphalan|Biological: Hematopoietic Stem Cell Transplantation|Drug: Alemtuzumab|Drug: mycophenylate mofetil|Device: Cyclosporine A|Drug: Hydroxyurea | Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT00383448 | |
| 217 | Macular Photocoagulation Study (MPS) | Unknown status | No Results Available | Choroidal Neovascularization|Macular Degeneration|Histoplasmosis | Procedure: Argon Blue-Green Laser Photocoagulation | | https://ClinicalTrials.gov/show/NCT00000158 | |
| 218 | Influence of Pantoprazole on the Bioavailability of MMF and EC-MPS | Completed | Has Results | Transplantation|Immunosuppression|Drug Interaction | Drug: Mycophenolate sodium|Drug: Pantoprazole|Drug: Mycophenolate mofetil | Charité Hospital Campus Mitte, Berlin, Germany | https://ClinicalTrials.gov/show/NCT01801280 | |
| 219 | A Comparative Evaluation of a MPS Dressing and a CMC Dressing on Subjects With Lower Extremity Venous Ulcers | Recruiting | No Results Available | Venous Leg Ulcer | Device: Marine polysaccharide dressing|Device: Carboxymethylcellulose dressing | Valley Vascular Surgery Associates, Fresno, California, United States|Central Valley Vein and Wound Center, Selma, California, United States|Covenant Hospital, Saginaw, Michigan, United States|Wake Forest Baptist Health, Winston-Salem, North Carolina, United States | https://ClinicalTrials.gov/show/NCT03903692 | |
| 220 | Comparative Efficacy of PRP and Dry Needling in Management of Trigger Points in Masseter Muscle in MPS Patients | Recruiting | No Results Available | Myofascial Pain Syndrome | Procedure: PRP injection|Procedure: Dry needling | PGIDS, Rohtak, Haryana, India | https://ClinicalTrials.gov/show/NCT04286880 | |
| 221 | Comfort Evaluation of a New MPS vs. Replenish in Contact Lens Patients | Completed | No Results Available | Corneal Staining | Other: new MPS|Other: RepleniSH | Private Practice, Azusa, California, United States | https://ClinicalTrials.gov/show/NCT00518700 | |
| 222 | Evaluation of Daily Disposable Lenses With Multi-Purpose Solution (MPS) | Completed | Has Results | Myopia | Device: Nelfilcon A contact lens|Device: Etafilcon A contact lens|Device: FID 107027 solution | Contact Alcon Call Center for Trial Locations, Fort Worth, Texas, United States | https://ClinicalTrials.gov/show/NCT00733291 | |
| 223 | Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders | Active, not recruiting | No Results Available | Hurler Syndrome (MPS I)|Hurler-Scheie Syndrome|Hunter Syndrome (MPS II)|Sanfilippo Syndrome (MPS III)|Krabbe Disease (Globoid Leukodystrophy)|Metachromatic Leukodystrophy (MLD)|Adrenoleukodystrophy (ALD and AMN)|Sandhoff Disease|Tay Sachs Disease|Pelizaeus Merzbacher (PMD)|Niemann-Pick Disease|Alpha-mannosidosis | Biological: hematopoietic stem cell infusion | Duke University Medical Center, Durham, North Carolina, United States | https://ClinicalTrials.gov/show/NCT01372228 | |
| 224 | UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells | Recruiting | No Results Available | Adrenoleukodystrophy|Batten Disease|Mucopolysaccharidosis II|Leukodystrophy, Globoid Cell|Leukodystrophy, Metachromatic|Neimann Pick Disease|Pelizaeus-Merzbacher Disease|Sandhoff Disease|Tay-Sachs Disease|Brain Diseases, Metabolic, Inborn|Alpha-Mannosidosis|Sanfilippo Mucopolysaccharidoses | Biological: DUOC-01 | Duke University Medical Center, Durham, North Carolina, United States | https://ClinicalTrials.gov/show/NCT02254863 | |
| 225 | Natural History of Morquio B and Late-Onset of GM1 Gangliosidosis | Not yet recruiting | No Results Available | Morquio B Disease|GM1 Gangliosidosis Type III | | BC Children's Hospital, Vancouver, British Columbia, Canada | https://ClinicalTrials.gov/show/NCT04320329 | |
| 226 | Biomarker for Morquio Disease (BioMorquio) | Active, not recruiting | No Results Available | Morquio Syndrome|Accumulation of Mucopolysaccharides|Morquio Syndrome A|Morquio B Disease | | Children's Hospital, Faculty of Medicine, Ain Shams University, Cairo, Egypt|Centogene AG, Rostock, Germany|Amrita Institute of Medical Sciences & Research Centre, Cochin, Kerala, India|Navi Mumbai Institute of Research In Mental And Neurological Handicap (NIRMAN), Mumbai, India|Lady Ridgeway Hospital for Children, Colombo 8, Sri Lanka | https://ClinicalTrials.gov/show/NCT01457456 | |
| 227 | A National Multi-center Randomized, Open Label Study to Evaluate Efficacy and Safety of Everolimus With EC-MPS Compared to Standard Treatment Combination Tacrolimus and EC-MPS in de Novo Liver Transplant Recipients | Completed | Has Results | Liver Transplantation | Drug: tacrolimus|Drug: everolimus|Drug: Basiliximab|Drug: Mycophenolic Acid|Drug: Corticosteroids | Novartis Investigative Site, Besancon cedex, France|Novartis Investigative Site, Bordeaux Cedex, France|Novartis Investigative Site, Chambray les Tours, France|Novartis Investigative Site, Clichy, France|Novartis Investigative Site, Creteil, France|Novartis Investigative Site, Grenoble, France|Novartis Investigative Site, Lille Cedex, France|Novartis Investigative Site, Marseille, France|Novartis Investigative Site, Montpellier, France|Novartis Investigative Site, Nice, France|Novartis Investigative Site, Paris Cedex 13, France|Novartis Investigative Site, Rennes Cedex, France|Novartis Investigative Site, Toulouse Cedex 4, France|Novartis Investigative Site, Villejuif, France | https://ClinicalTrials.gov/show/NCT01625377 | |
| 228 | Longitudinal Study of Neurodegenerative Disorders | Recruiting | No Results Available | MLD|Krabbe Disease|ALD|MPS I|MPS II|MPS III|Vanishing White Matter Disease|GM3 Gangliosidosis|PKAN|Tay-Sachs Disease|NP Deficiency|Osteopetrosis|Alpha-Mannosidosis|Sandhoff Disease|Niemann-Pick Diseases|MPS IV|Gaucher Disease|GAN|GM1 Gangliosidoses|Morquio Disease|S-Adenosylhomocysteine Hydrolase Deficiency|Batten Disease|Pelizaeus-Merzbacher Disease|Leukodystrophy|Lysosomal Storage Diseases|Purine Nucleoside Phosphorylase Deficiency|Multiple Sulfatase Deficiency Disease | Other: Palliative Care|Biological: Hematopoetic Stem Cell Transplantation | The Program for the Study of Neurodevelopment in Rare Disorders, Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, United States | https://ClinicalTrials.gov/show/NCT03333200 | |
| 229 | A One-year, Open Label Study to Investigate the Safety and the Effect of Enteric-coated Mycophenolate Sodium (EC-MPS) in Combination With Cyclosporine Microemulsion in de Novo Kidney Transplant Recipients. | Completed | No Results Available | Kidney Transplantation | Drug: Enteric-Coated Mycophenolate sodium (EC-MPS) | | https://ClinicalTrials.gov/show/NCT00154245 | |
| 230 | Mindfulness and Paralympic Sport: a Mindfulness Based Intervention for Paralympic Boccia Brazilian Team | Active, not recruiting | No Results Available | Stress | Other: A mindfulness based intervention | Centro Mente Aberta em Mindfulness, São Paulo, Brazil | https://ClinicalTrials.gov/show/NCT04334642 | |
| 231 | Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage Disorders | Recruiting | No Results Available | Lysosomal Storage Diseases | | LDRTC, Fairfax, Virginia, United States | https://ClinicalTrials.gov/show/NCT03812055 | |
| 232 | Alcon Multi-Purpose Disinfecting Solution Versus Renu Fresh Multi-Purpose Solution in Symptomatic Contact Lens Wearers | Completed | Has Results | Symptomatic Contact Lens Wearers | Device: FID 114675A Multi-Purpose Disinfecting Solution (MPDS)|Device: renu fresh Multi-Purpose Solution (MPS)|Device: Contact lenses | Contact Alcon Call Center for Trial Locations, Fort Worth, Texas, United States | https://ClinicalTrials.gov/show/NCT01187355 | |
| 233 | Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders | Completed | Has Results | Mucopolysaccharidosis|Hurler Syndrome|Hunter Syndrome|Maroteaux-Lamy Syndrome|Sly Syndrome|Alpha Mannosidosis|Fucosidosis|Aspartylglucosaminuria|Adrenoleukodystrophy (ALD)|Krabbe Disease|Metachromatic Leukodystrophy (MLD)|Sphingolipidoses|Peroxisomal Disorders | Drug: Campath-1H|Drug: Cyclophosphamide|Drug: Busulfan|Procedure: Allogeneic stem cell transplantation|Drug: Cyclosporine A|Drug: Mycophenolate Mofetil | Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT01043640 | |
| 234 | MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis | Recruiting | No Results Available | Mucopolysaccharidosis Disorders|Hurler Syndrome|Hunter Syndrome|Maroteaux Lamy Syndrome|Sly Syndrome|Alpha-Mannosidosis|Fucosidosis|Aspartylglucosaminuria|Glycoprotein Metabolic Disorders|Sphingolipidoses|Recessive Leukodystrophies|Globoid Cell Leukodystrophy|Metachromatic Leukodystrophy|Niemann-Pick B|Niemann-Pick C Subtype 2|Sphingomyelin Deficiency|Peroxisomal Disorders|Adrenoleukodystrophy With Cerebral Involvement|Zellweger Syndrome|Neonatal Adrenoleukodystrophy|Infantile Refsum Disease|Acyl-CoA Oxidase Deficiency|D-Bifunctional Enzyme Deficiency|Multifunctional Enzyme Deficiency|Alpha-methylacyl-CoA Racmase Deficiency|Mitochondrial Neurogastrointestingal Encephalopathy|Severe Osteopetrosis|Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation)|Inherited Metabolic Disorders | Biological: Stem Cell Transplantation|Drug: IMD Preparative Regimen|Drug: Osteopetrosis Only Preparative Regimen|Drug: Osteopetrosis Haploidentical Only Preparative Regimen|Drug: cALD SR-A (Standard-Risk, Regimen A)|Drug: cALD SR-B (Standard-Risk, Regimen B)|Drug: cALD HR-D (High-Risk, Regimen C)|Drug: cALD HR-D (High-Risk, Regimen D) | Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT02171104 | |
| 235 | Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford | Recruiting | No Results Available | Rare Disorders|Undiagnosed Disorders|Disorders of Unknown Prevalence|Cornelia De Lange Syndrome|Prenatal Benign Hypophosphatasia|Perinatal Lethal Hypophosphatasia|Odontohypophosphatasia|Adult Hypophosphatasia|Childhood-onset Hypophosphatasia|Infantile Hypophosphatasia|Hypophosphatasia|Kabuki Syndrome|Bohring-Opitz Syndrome|Narcolepsy Without Cataplexy|Narcolepsy-cataplexy|Hypersomnolence Disorder|Idiopathic Hypersomnia Without Long Sleep Time|Idiopathic Hypersomnia With Long Sleep Time|Idiopathic Hypersomnia|Kleine-Levin Syndrome|Kawasaki Disease|Leiomyosarcoma|Leiomyosarcoma of the Corpus Uteri|Leiomyosarcoma of the Cervix Uteri|Leiomyosarcoma of Small Intestine|Acquired Myasthenia Gravis|Addison Disease|Hyperacusis (Hyperacousis)|Juvenile Myasthenia Gravis|Transient Neonatal Myasthenia Gravis|Williams Syndrome|Lyme Disease|Myasthenia Gravis|Marinesco Sjogren Syndrome(Marinesco-Sjogren Syndrome)|Isolated Klippel-Feil Syndrome|Frasier Syndrome|Denys-Drash Syndrome|Beckwith-Wiedemann Syndrome|Emanuel Syndrome|Isolated Aniridia|Axenfeld-Rieger Syndrome|Aniridia-intellectual Disability Syndrome|Aniridia - Renal Agenesis - Psychomotor Retardation|Aniridia - Ptosis - Intellectual Disability - Familial Obesity|Aniridia - Cerebellar Ataxia - Intellectual Disability|Aniridia - Absent Patella|Aniridia|Peters Anomaly - Cataract|Peters Anomaly|Potocki-Shaffer Syndrome|Silver-Russell Syndrome Due to Maternal Uniparental Disomy of Chromosome 11|Silver-Russell Syndrome Due to Imprinting Defect of 11p15|Silver-Russell Syndrome Due to 11p15 Microduplication|Syndromic Aniridia|WAGR Syndrome|Wolf-Hirschhorn Syndrome|4p16.3 Microduplication Syndrome|4p Deletion Syndrome, Non-Wolf-Hirschhorn Syndrome|Autosomal Recessive Stickler Syndrome|Stickler Syndrome Type 2|Stickler Syndrome Type 1|Stickler Syndrome|Mucolipidosis Type 4|X-linked Spinocerebellar Ataxia Type 4|X-linked Spinocerebellar Ataxia Type 3|X-linked Intellectual Disability - Ataxia - Apraxia|X-linked Progressive Cerebellar Ataxia|X-linked Non Progressive Cerebellar Ataxia|X-linked Cerebellar Ataxia|Vitamin B12 Deficiency Ataxia|Toxic Exposure Ataxia|Unclassified Autosomal Dominant Spinocerebellar Ataxia|Thyroid Antibody Ataxia|Sporadic Adult-onset Ataxia of Unknown Etiology|Spinocerebellar Ataxia With Oculomotor Anomaly|Spinocerebellar Ataxia With Epilepsy|Spinocerebellar Ataxia With Axonal Neuropathy Type 2|Spinocerebellar Ataxia Type 8|Spinocerebellar Ataxia Type 7|Spinocerebellar Ataxia Type 6|Spinocerebellar Ataxia Type 5|Spinocerebellar Ataxia Type 4|Spinocerebellar Ataxia Type 37|Spinocerebellar Ataxia Type 36|Spinocerebellar Ataxia Type 35|Spinocerebellar Ataxia Type 34|Spinocerebellar Ataxia Type 32|Spinocerebellar Ataxia Type 31|Spinocerebellar Ataxia Type 30|Spinocerebellar Ataxia Type 3|Spinocerebellar Ataxia Type 29|Spinocerebellar Ataxia Type 28|Spinocerebellar Ataxia Type 27|Spinocerebellar Ataxia Type 26|Spinocerebellar Ataxia Type 25|Spinocerebellar Ataxia Type 23|Spinocerebellar Ataxia Type 22|Spinocerebellar Ataxia Type 21|Spinocerebellar Ataxia Type 20|Spinocerebellar Ataxia Type 2|Spinocerebellar Ataxia Type 19/22|Spinocerebellar Ataxia Type 18|Spinocerebellar Ataxia Type 17|Spinocerebellar Ataxia Type 16|Spinocerebellar Ataxia Type 15/16|Spinocerebellar Ataxia Type 14|Spinocerebellar Ataxia Type 13|Spinocerebellar Ataxia Type 12|Spinocerebellar Ataxia Type 11|Spinocerebellar Ataxia Type 10|Spinocerebellar Ataxia Type 1 With Axonal Neuropathy|Spinocerebellar Ataxia Type 1|Spinocerebellar Ataxia - Unknown|Spinocerebellar Ataxia - Dysmorphism|Non Progressive Epilepsy and/or Ataxia With Myoclonus as a Major Feature|Spasticity-ataxia-gait Anomalies Syndrome|Spastic Ataxia With Congenital Miosis|Spastic Ataxia - Corneal Dystrophy|Spastic Ataxia|Rare Hereditary Ataxia|Rare Ataxia|Recessive Mitochondrial Ataxia Syndrome|Progressive Epilepsy and/or Ataxia With Myoclonus as a Major Feature|Posterior Column Ataxia - Retinitis Pigmentosa|Post-Stroke Ataxia|Post-Head Injury Ataxia|Post Vaccination Ataxia|Polyneuropathy - Hearing Loss - Ataxia - Retinitis Pigmentosa - Cataract|Muscular Atrophy - Ataxia - Retinitis Pigmentosa - Diabetes Mellitus|Non-hereditary Degenerative Ataxia|Paroxysmal Dystonic Choreathetosis With Episodic Ataxia and Spasticity|Olivopontocerebellar Atrophy - Deafness|NARP Syndrome|Myoclonus - Cerebellar Ataxia - Deafness|Multiple System Atrophy, Parkinsonian Type|Multiple System Atrophy, Cerebellar Type|Multiple System Atrophy|Maternally-inherited Leigh Syndrome|Machado-Joseph Disease Type 3|Machado-Joseph Disease Type 2|Machado-Joseph Disease Type 1|Leigh Syndrome|Late-onset Ataxia With Dementia|Infection or Post Infection Ataxia|GAD Ataxia|Hereditary Episodic Ataxia|Gliadin/Gluten Ataxia|Friedreich Ataxia|Fragile X-associated Tremor/Ataxia Syndrome|Familial Paroxysmal Ataxia|Exposure to Medications Ataxia|Episodic Ataxia With Slurred Speech|Episodic Ataxia Unknown Type|Episodic Ataxia Type 7|Episodic Ataxia Type 6|Episodic Ataxia Type 5|Episodic Ataxia Type 4|Episodic Ataxia Type 3|Episodic Ataxia Type 1|Epilepsy and/or Ataxia With Myoclonus as Major Feature|Early-onset Spastic Ataxia-neuropathy Syndrome|Early-onset Progressive Neurodegeneration - Blindness - Ataxia - Spasticity|Early-onset Cerebellar Ataxia With Retained Tendon Reflexes|Early-onset Ataxia With Dementia|Childhood-onset Autosomal Recessive Slowly Progressive Spinocerebellar Ataxia|Dilated Cardiomyopathy With Ataxia|Cataract - Ataxia - Deafness|Cerebellar Ataxia, Cayman Type|Cerebellar Ataxia With Peripheral Neuropathy|Cerebellar Ataxia - Hypogonadism|Cerebellar Ataxia - Ectodermal Dysplasia|Cerebellar Ataxia - Areflexia - Pes Cavus - Optic Atrophy - Sensorineural Hearing Loss|Brain Tumor Ataxia|Brachydactyly - Nystagmus - Cerebellar Ataxia|Benign Paroxysmal Tonic Upgaze of Childhood With Ataxia|Autosomal Recessive Syndromic Cerebellar Ataxia|Autosomal Recessive Spastic Ataxia With Leukoencephalopathy|Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay|Autosomal Recessive Spastic Ataxia - Optic Atrophy - Dysarthria|Autosomal Recessive Spastic Ataxia|Autosomal Recessive Metabolic Cerebellar Ataxia|Autosomal Dominant Spinocerebellar Ataxia Due to Repeat Expansions That do Not Encode Polyglutamine|Autosomal Recessive Ataxia, Beauce Type|Autosomal Recessive Ataxia Due to Ubiquinone Deficiency|Autosomal Recessive Ataxia Due to PEX10 Deficiency|Autosomal Recessive Degenerative and Progressive Cerebellar Ataxia|Autosomal Recessive Congenital Cerebellar Ataxia Due to MGLUR1 Deficiency|Autosomal Recessive Congenital Cerebellar Ataxia Due to GRID2 Deficiency|Autosomal Recessive Congenital Cerebellar Ataxia|Autosomal Recessive Cerebellar Ataxia-pyramidal Signs-nystagmus-oculomotor Apraxia Syndrome|Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to WWOX Deficiency|Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to TUD Deficiency|Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to KIAA0226 Deficiency|Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome|Autosomal Recessive Cerebellar Ataxia With Late-onset Spasticity|Autosomal Recessive Cerebellar Ataxia Due to STUB1 Deficiency|Autosomal Recessive Cerebellar Ataxia Due to a DNA Repair Defect|Autosomal Recessive Cerebellar Ataxia - Saccadic Intrusion|Autosomal Recessive Cerebellar Ataxia - Psychomotor Retardation|Autosomal Recessive Cerebellar Ataxia - Blindness - Deafness|Autosomal Recessive Cerebellar Ataxia|Autosomal Dominant Spinocerebellar Ataxia Due to a Polyglutamine Anomaly|Autosomal Dominant Spinocerebellar Ataxia Due to a Point Mutation|Autosomal Dominant Spinocerebellar Ataxia Due to a Channelopathy|Autosomal Dominant Spastic Ataxia Type 1|Autosomal Dominant Spastic Ataxia|Autosomal Dominant Optic Atrophy|Ataxia-telangiectasia Variant|Ataxia-telangiectasia|Autosomal Dominant Cerebellar Ataxia, Deafness and Narcolepsy|Autosomal Dominant Cerebellar Ataxia Type 4|Autosomal Dominant Cerebellar Ataxia Type 3|Autosomal Dominant Cerebellar Ataxia Type 2|Autosomal Dominant Cerebellar Ataxia Type 1|Autosomal Dominant Cerebellar Ataxia|Ataxia-telangiectasia-like Disorder|Ataxia With Vitamin E Deficiency|Ataxia With Dementia|Ataxia - Oculomotor Apraxia Type 1|Ataxia - Other|Ataxia - Genetic Diagnosis - Unknown|Acquired Ataxia|Adult-onset Autosomal Recessive Cerebellar Ataxia|Alcohol Related Ataxia|Multiple Endocrine Neoplasia|Multiple Endocrine Neoplasia Type II|Multiple Endocrine Neoplasia Type 1|Multiple Endocrine Neoplasia Type 2|Multiple Endocrine Neoplasia, Type IV|Multiple Endocrine Neoplasia, Type 3|Multiple Endocrine Neoplasia (MEN) Syndrome|Multiple Endocrine Neoplasia Type 2B|Multiple Endocrine Neoplasia Type 2A|Atypical Hemolytic Uremic Syndrome|Atypical HUS|Wiedemann-Steiner Syndrome|Breast Implant-Associated Anaplastic Large Cell Lymphoma|Autoimmune/Inflammatory Syndrome Induced by Adjuvants (ASIA)|Hemophagocytic Lymphohistiocytosis|Behcet's Disease|Alagille Syndrome|Inclusion Body Myopathy With Early-onset Paget Disease and Frontotemporal Dementia (IBMPFD)|Lowe Syndrome|Pitt Hopkins Syndrome|1p36 Deletion Syndrome|Jansen Type Metaphyseal Chondrodysplasia|Cockayne Syndrome|Chronic Recurrent Multifocal Osteomyelitis|CRMO|Malan Syndrome|Hereditary Sensory and Autonomic Neuropathy Type Ie|VCP Disease|Hypnic Jerking|Sleep Myoclonus|Mollaret Meningitis|Recurrent Viral Meningitis|CRB1|Leber Congenital Amaurosis|Retinitis Pigmentosa|Rare Retinal Disorder|KCNMA1-Channelopathy|Primary Biliary Cirrhosis|ZMYND11|Transient Global Amnesia|Glycogen Storage Disease|Alstrom Syndrome|White Sutton Syndrome|DNM1|EIEE31|Myhre Syndrome|Recurrent Respiratory Papillomatosis|Laryngeal Papillomatosis|Tracheal Papillomatosis|Refsum Disease|Nicolaides Baraitser Syndrome|Leukodystrophy | | Sanford Health, Sioux Falls, South Dakota, United States|Online Patient Enrollment System, Sydney, Australia | https://ClinicalTrials.gov/show/NCT01793168 | |
| 236 | The Effect of Very Low Calorie Diet With and Without Exercise on Muscle Synthesis in Middle-aged Overweight Male. | Recruiting | No Results Available | Overweight and Obesity | Dietary Supplement: Very low calorie diet (VLCD)|Other: High intensity interval training (HIIT)|Other: Resistance exercise training (RET) | MRC-ARUK Centre of Excellence for Musculoskeletal Ageing, School of Medicine, University of Nottingham,, Derby, Derbyshire, United Kingdom | https://ClinicalTrials.gov/show/NCT03116256 | |
| 237 | Evaluation of Marketed Contact Lens Disinfectant Systems on Eyelid Tissues | Completed | Has Results | Contact Lens Care | Device: Hydrogen peroxide-based contact lens care system|Device: PHMB-containing contact lens solution|Device: Soft contact lenses | | https://ClinicalTrials.gov/show/NCT01494818 | |
| 238 | Evaluation of Early Use of Everolimus (EVE) on Cytomegalovirus (CMV) Infection in Renal Transplant Recipients | Unknown status | No Results Available | Cytomegalovirus Infections | Drug: Everolimus+Tacrolimus+Prednisone|Drug: Mycophenolate+Tacrolimus+Prednisone | | https://ClinicalTrials.gov/show/NCT01927588 | |
| 239 | SM-88 as Maintenance Therapy for Advanced Ewing's Sarcoma Patients and as Salvage Therapy for Sarcoma Patients | Recruiting | No Results Available | Sarcoma, Ewing|Sarcoma | Drug: Combination metyrosine-derivative, low-dose methoxsalen, phenytoin and sirolimus (MPS) | Sarcoma Oncology Research Center, Santa Monica, California, United States | https://ClinicalTrials.gov/show/NCT03778996 | |
| 240 | Myfortic in Heart Transplant Patients With Gastrointestinal (GI) Symptoms | Unknown status | No Results Available | Heart Transplant Patients | Drug: Myfortic | McGill University Health Centre, Montreal, Quebec, Canada | https://ClinicalTrials.gov/show/NCT00468936 | |
| 241 | A Study of the Combination of Electrical Stimulation and Dysport® in Myofascial Pain Syndrome. | Completed | No Results Available | Myofascial Pain Syndromes | Biological: Botulinum toxin type A | Seoul National University Hospital, Seoul, Korea, Republic of | https://ClinicalTrials.gov/show/NCT00246142 | |
| 242 | An Evaluation of Marketed Contact Lens Disinfectant Systems on Eyelid Tissues | Completed | Has Results | Myopia | Device: AOSEPT® Plus contact lens solution|Device: ReNu MultiPlus® contact lens solution|Device: Etafilcon A contact lenses|Device: Lotrafilcon B contact lenses|Device: COMPLETE® MPS Easy Rub® Formula contact lens solution | | https://ClinicalTrials.gov/show/NCT01594294 | |
| 243 | Reducing Donor Specific Antibody (DSA) Strength in Maintenance Kidney Transplant Recipients (DSA Study) | Completed | Has Results | Transplant; Failure, Kidney | Drug: Myfortic Escalation | East Carolina University, Greenville, North Carolina, United States | https://ClinicalTrials.gov/show/NCT01044303 | |
| 244 | Clinical Study to Evaluate the Tolerability, Safety and Efficacy of Enteric-coated Mycophenolate Sodium After Equimolar Conversion From Mycophenolate Mofetil (MMF) in Patients With Renal Transplant | Completed | No Results Available | Prevention of Acute Rejection After Kidney Transplantation | Drug: Enteric-Coated Mycophenolate Sodium | | https://ClinicalTrials.gov/show/NCT00171379 | |
| 245 | Enteric-Coated Mycophenolate Sodium on Quality of Life in Patients With Gastrointestinal Symptoms Related to Mycophenolate Mofetil Therapy After Kidney Transplantation | Completed | Has Results | Renal Transplant | Drug: Enteric-Coated Mycophenolate Sodium (EC-MPS)|Drug: Mycophenolate Mofetil (MMF) | Novartis Investigative Site, Nottingham, United Kingdom | https://ClinicalTrials.gov/show/NCT00239005 | |
| 246 | An Open, Single Centre, Randomised, Parallel Group Study to Investigate Three Different Immunosuppressive Regimens | Completed | No Results Available | Comparative Study|Immunosuppressive Agents | Drug: Rapamycin|Drug: Everolimus|Drug: Prednisone | Clinic for transplantation immunology and nephrology, Basel, Switzerland | https://ClinicalTrials.gov/show/NCT01183247 | |
| 247 | Gastrointestinal and Health-related Quality of Life in Kidney Transplant Patients Treated With Mycophenolate Mofetil | Completed | No Results Available | Maintenance Kidney Transplant | Drug: Enteric-coated Mycophenolate sodium (EC-MPS)|Drug: Mycophenolate mofetil | Novartis, Basel, Switzerland | https://ClinicalTrials.gov/show/NCT00400647 | |
| 248 | Registry Gangliosidoses | Recruiting | No Results Available | Gangliosidoses|GM1 Gangliosidosis|Morquio B Disease|Sialidosis|Galactosialidosis|Gm2-Gangliosidosis, Variant B1|Tay-Sachs Disease|Sandhoff Disease|GM2 Activator Deficiency | | SphinCS Lyso gemeinnützige UG (haftungsbeschränkt), Hochheim, HE, Germany | https://ClinicalTrials.gov/show/NCT04624789 | |
| 249 | Intensified vs. Standard Dose Therapy With Mycophenolate Sodium Plus Cyclosporin Microemulsion and Corticosteroid Combination in Patients With de Novo Renal Transplant Patients | Completed | Has Results | Renal Transplantation | Drug: Enteric-coated mycophenolate sodium (EC-MPS) | Novartis Investigational Site, Various Cities, Germany | https://ClinicalTrials.gov/show/NCT00369278 | |
| 250 | SOCRATES: Steroid or Cyclosporine Removal After Transplantation Using Everolimus | Completed | Has Results | Renal Transplanted Recipients | Drug: Everolimus (RAD001)|Drug: Cyclosporine (Calcineurin Inhibitor (CNI))|Drug: Methylprednisone/prednisone|Drug: Mycophenolate sodium (MPA) | Royal Prince Alfred Hospital, NSW, Australia|Westmead Hospital, NSW, Australia|Princess Alexandra Hospital, QLD, Australia|Monash Medical Centre, Sale, Australia|Queen Elizabeth Hospital, Sale, Australia|Royal Melbourne Hospital, VIC, Australia|Sir Charles Gairdner Hospital, WA, Australia | https://ClinicalTrials.gov/show/NCT00371826 | |
| 251 | Clinical Study of Autologous Erythrocytes Derived MPs Packaging MTX Peritoneal Perfusion to Treat Malignant Ascites | Unknown status | No Results Available | Malignant Ascites | Other: Erythrocytes derived MPs containing MTX|Drug: convention drugs | Hui ting Xu, Wuhan, Hubei, China | https://ClinicalTrials.gov/show/NCT03230708 | |
| 252 | Effects of Growth Hormone in Chronically Ill Children | Withdrawn | No Results Available | Hurler Syndrome|Cerebral Palsy|Juvenile Rheumatoid Arthritis|Crohn Disease|HIV Infections | Drug: Growth Hormone|Procedure: Whole body Protein turnover|Procedure: DEXA scan | Children's Medical Center of Dallas, Dallas, Texas, United States | https://ClinicalTrials.gov/show/NCT00286689 | |
| 253 | A Gastrointestinal Quality of Life Study in Lung Transplant Recipients Converted From Mycophenolate Mofetil to Myfortic | Terminated | No Results Available | Lung Transplantation | Behavioral: GI and Quality of Life Assessment | Vanderbilt University Medical Center, Nashville, Tennessee, United States | https://ClinicalTrials.gov/show/NCT01044667 | |
| 254 | Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism | Terminated | Has Results | Hurler's Syndrome|Maroteaux-Lamy Syndrome|Sly Syndrome|Alpha Mannosidosis|Fucosidosis|Aspartylglucosaminuria|Sphingolipidoses|Krabbe Disease|Wolman's Disease|Niemann-Pick Disease Type B|Niemann-Pick Disease, Type C | Procedure: Stem Cell Transplantation|Drug: Cyclophosphamide|Drug: Campath-1H|Drug: Busulfan | University of Minnesota, Fairview, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT00668564 | |
| 255 | In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases | Not yet recruiting | No Results Available | MPS I|MPS II|MPS IVA|MPS VI|Mps VII|Gaucher Disease, Type 2|Gaucher Disease, Type 3|Pompe Disease Infantile-Onset|Wolman Disease | Drug: Aldurazyme (laronidase) | University of California, San Francisco, California, United States | https://ClinicalTrials.gov/show/NCT04532047 | |
| 256 | Providing Access to Cord Blood Units for Transplants | Withdrawn | No Results Available | Chronic Myelogous Leukemia/Other Leukemia|Acute Leukemias|MDS/MPS|Multiple Myeloma|Non-Hodgkin Lymphoma | Drug: Access to unlicensed cord blood units | | https://ClinicalTrials.gov/show/NCT01479582 | |
| 257 | Biomarker for Glycogen Storage Diseases (BioGlycogen) | Active, not recruiting | No Results Available | Fructose Metabolism, Inborn Errors|Glycogen Storage Disease|Glycogen Storage Disease Type I|Glycogen Storage Disease Type II|Glycogen Storage Disease Type III|Glycogen Storage Disease Type IV|Glycogen Storage Disease Type V|Glycogen Storage Disease Type VI|Glycogen Storage Disease Type VII|Glycogen Storage Disease Type VIII | | Centogene AG, Rostock, Germany|Amrita Institute of Medical Sciences & Research Centre, Cochin, Kerala, India|Navi Mumbai Institute of Research In Mental And Neurological Handicap (NIRMAN), Mumbai, India|Lady Ridgeway Hospital for Children, Colombo 8, Sri Lanka | https://ClinicalTrials.gov/show/NCT02385162 | |
| 258 | BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study | No longer available | No Results Available | Hurler Syndrome|Inherited Metabolic Disorder|Lysosomal Storage Disorder|Metachromatic Leukodystrophy|Inborn Errors of Metabolism | Biological: rivogenlecleucel|Drug: rimiducid | Children's Hospital Los Angeles, Los Angeles, California, United States|Stanford University; Division of Pediatric Stem Cell Transplant & Regenerative Medicine, Palo Alto, California, United States | https://ClinicalTrials.gov/show/NCT03639844 | |
| 259 | Biomarker for Maroteaux-Lamy Disease (BioMaroteaux) | Active, not recruiting | No Results Available | Lysosomal Storage Disease|Lung Diseases|Obstructive Sleep Apnoea|Macroglossia|Eye Abnormalities | | Children's Hospital, Faculty of Medicine, Ain Shams University, Cairo, Egypt|Centogene AG, Rostock, Germany|Amrita Institute of Medical Sciences & Research Centre, Cochin, Kerala, India|Navi Mumbai Institute of Research In Mental And Neurological Handicap (NIRMAN), Mumbai, India|Lady Ridgeway Hospital for Children, Colombo 8, Sri Lanka | https://ClinicalTrials.gov/show/NCT01458613 | |
| 260 | Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation | Recruiting | No Results Available | Bone Marrow Failure Syndrome|Thalassemia|Sickle Cell Disease|Diamond Blackfan Anemia|Acquired Neutropenia in Newborn|Acquired Anemia Hemolytic|Acquired Thrombocytopenia|Hemophagocytic Lymphohistiocytoses|Wiskott-Aldrich Syndrome|Chronic Granulomatous Disease|Common Variable Immunodeficiency|X-linked Lymphoproliferative Disease|Severe Combined Immunodeficiency|Hurler Syndrome|Mannosidosis|Adrenoleukodystrophy | Drug: Thiotepa--single daily dose|Drug: Thiotepa--escalated dose | UF Health Shands Children's Hospital, Gainesville, Florida, United States | https://ClinicalTrials.gov/show/NCT03513328 | |
| 261 | Bone Marrow Transplant With Abatacept for Non-Malignant Diseases | Completed | No Results Available | Hurler Syndrome|Fanconi Anemia|Glanzmann Thrombasthenia|Wiskott-Aldrich Syndrome|Chronic Granulomatous Disease|Severe Congenital Neutropenia|Leukocyte Adhesion Deficiency|Shwachman-Diamond Syndrome|Diamond-Blackfan Anemia|Dyskeratosis-congenita|Chediak-Higashi Syndrome|Severe Aplastic Anemia|Thalassemia Major|Hemophagocytic Lymphohistiocytosis|Sickle Cell Disease | Drug: Abatacept | Children's Healthcare of Atlanta, Atlanta, Georgia, United States | https://ClinicalTrials.gov/show/NCT01917708 | |
| 262 | Extension Study on the Efficacy and Safety of Enteric-coated Mycophenolate Sodium Administered in de Novo Kidney Transplant Patients | Completed | No Results Available | De Novo Kidney Transplantation | Drug: Enteric-Coated Mycophenolate Sodium (EC-MPS) | | https://ClinicalTrials.gov/show/NCT00239018 | |
| 263 | Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells | Completed | No Results Available | Sickle Cell Disease|Thalassemia|Anemia|Granuloma|Wiskott-Aldrich Syndrome|Chediak Higashi Syndrome|Osteopetrosis|Neutropenia|Thrombocytopenia|Hurler Disease|Niemann-Pick Disease|Fucosidosis | Procedure: Hematopoietic stem cell transplantation | Children Hospital Los Angeles, Los Angeles, California, United States | https://ClinicalTrials.gov/show/NCT00730314 | |
| 264 | Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT | Recruiting | No Results Available | Primary Immunodeficiency (PID)|Congenital Bone Marrow Failure Syndromes|Inherited Metabolic Disorders (IMD)|Hereditary Anemias|Inflammatory Conditions | Drug: Hydroxyurea|Drug: Alemtuzumab|Drug: Fludarabine|Drug: Melphalan|Drug: Thiotepa | UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States | https://ClinicalTrials.gov/show/NCT01962415 | |
| 265 | Efficacy and Safety of Enteric-coated Mycophenolate Sodium in Combination With Two Corticosteroid Regimens for the Treatment of Lupus Nephritis Flare | Completed | Has Results | Lupus Nephritis | Drug: Mycophenolate sodium|Drug: Prednisone|Drug: Methylprednisolone | Novartis, Bogota, Colombia|Novartis, Creteil, France|Novartis, Nantes, France|Novartis Investigative Site, Paris, France|Novartis, Berlin, Germany|Novartis, Tubingen, Germany|Novartis, Athens, Greece|Novartis, Budapest, Hungary|Novartis, Debrecen, Hungary|Novartis, Brescia, Italy|Novartis, Ferrara, Italy|Novartis, Milano, Italy|Novartis, Padova, Italy|Novartis, Barcelona, Spain|Novartis, Madrid, Spain|Novartis, Taichung, Taiwan|Novartis, Cambridge, United Kingdom | https://ClinicalTrials.gov/show/NCT00423098 | |
| 266 | Measurement of Patient Reported Gastrointestinal (GI) and Health-related Quality of Life (HRQL) Outcomes in Renal Transplant Recipients (MyLife) | Completed | No Results Available | Renal Transplantation|Gastrointestinal Problems | Drug: Enteric-Coated Mycophenolate Sodium (EC-MPS) | Bonn, Germany|Novartis, Basel, Switzerland | https://ClinicalTrials.gov/show/NCT00149968 | |
| 267 | Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases | Active, not recruiting | No Results Available | Primary Immune Deficiency Disorders|Hemophagocytic Lymphohistiocytosis|Inherited Bone Marrow Failure Syndrome|Hemoglobinopathies|Metabolic Disorders | Biological: BPX-501 and Rimiducid | Fred Hutchinson Cancer ResearchCenter, Seattle, Washington, United States | https://ClinicalTrials.gov/show/NCT02231710 | |
| 268 | Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders | Completed | Has Results | Lysosomal Storage Disease|Peroxisomal Disorder | Drug: Campath-1H|Drug: Clofarabine|Drug: Melphalan|Radiation: Total Body Irradiation with Marrow Boosting|Biological: Hematopoietic stem cell transplantation|Drug: Cyclosporine A|Drug: Mycophenolate mofetil | Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota, United States | https://ClinicalTrials.gov/show/NCT01626092 | |
| 269 | MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT) | Active, not recruiting | No Results Available | Inherited Metabolic Disorders (IMD) | Drug: MGTA-456 | University of Minnesota, Minneapolis, Minnesota, United States|Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States | https://ClinicalTrials.gov/show/NCT03406962 | |
| 270 | Efficacy and Tolerability of Hirudoid Cream in Prophylaxis and Treatment Infusion Phlebitis | Completed | No Results Available | Thrombophlebitis|Phlebitis | Drug: Hirudoid cream 0.3 % Mucopolysaccharide polysulfate|Drug: Placebo cream without active substance | Siriraj Hospital, Bangkok, Thailand|Chulalongkorn Hospital, Bangkok, Thailand|Rajvithi Hospital, Bangkok, Thailand|Bamrasnaradua Infectious Diseases Institute, Nonthaburi, Thailand | https://ClinicalTrials.gov/show/NCT01943006 | |
| 271 | Nervous System Degeneration in Glycosphingolipid Storage Disorders | Recruiting | No Results Available | Neurological Regression|Myoclonus|Cherry Red Spot|Brain Atrophy | | National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, United States | https://ClinicalTrials.gov/show/NCT00029965 | |
| 272 | Observational Registry Study of Renal Transplant Patients | Withdrawn | No Results Available | Kidney Transplant | | San Francisco investigational site, San Francisco, California, United States|Denver Investigational site, Denver, Colorado, United States|Springfield investigational site, Springfield, Massachusetts, United States|Detroit investigational site, Detroit, Michigan, United States|New York investigational site, New York, New York, United States|PHILADELPHIA investigational site, Philadelphia, Pennsylvania, United States|BURLINGTON investigational site, Burlington, Vermont, United States|Seattle investigational site, Seattle, Washington, United States | https://ClinicalTrials.gov/show/NCT01284257 | |
| 273 | Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC | Recruiting | No Results Available | Primary Immunodeficiency (PID)|Congenital Bone Marrow Failure Syndromes|Inherited Metabolic Disorders (IMD)|Hereditary Anemias|Inflammatory Conditions | Drug: data collection | UPMC Children's Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States | https://ClinicalTrials.gov/show/NCT04528355 | |
| 274 | S 81694 Plus Paclitaxel in Metastatic Breast Cancer | Completed | No Results Available | Metastatic Breast Cancer|Metastatic Triple Negative Breast Cancer | Drug: Combination therapy (S81694 + paclitaxel) phase I|Drug: Paclitaxel|Drug: Combination therapy (S81694 + paclitaxel) phase II | Institut Jules Bordet Clinique Oncologie Médicale, Bruxelles, Belgium|UZ Leuven Campus Gasthuisberg Dept. of General Medical, Leuven, Belgium|Institut de Cancérologie de l'Ouest site Saint Herblain, Saint Herblain, France|Chiba cancer center Breast surgery, Chiba, Japan|Osaka International Cancer Institute, Osaka, Japan|Erasmus MC Section Clinical Pharmacology, Rotterdam, Netherlands | https://ClinicalTrials.gov/show/NCT03411161 | |
| 275 | A Randomized Phase 2/3 Multi-Center Study of SM-88 in Patients With Metastatic Pancreatic Cancer | Recruiting | No Results Available | Pancreatic Cancer | Drug: SM-88 used with MPS (methoxsalen, phenytoin, sirolimus)|Drug: Capecitabine, Gemcitabine, and 5-FU | University of Alabama at Birmingham, Birmingham, Alabama, United States|City Of Hope, Duarte, California, United States|Sarcoma Oncology Research Center, Santa Monica, California, United States|Hartford Healthcare Cancer, New Britain, Connecticut, United States|H. Lee Moffitt Cancer Center & Research Institute, Tampa, Florida, United States|Advent Health Florida Hospital Tampa, Tampa, Florida, United States|June E. Nylen Cancer Center, Sioux City, Iowa, United States|University Medical Center, New Orleans, Louisiana, United States|Karmanos Cancer Institute, Detroit, Michigan, United States|Washington University, Saint Louis, Missouri, United States|New York Cancer and Blood Specialist, Bronx, New York, United States|North Shore Hematology Oncology, East Setauket, New York, United States|NY Cancer and Blood Specialist, East Setauket, New York, United States|NYU Langone Health, New York, New York, United States|Central Park Hematology & Oncology, New York, New York, United States|Weill Cornell, New York, New York, United States|The Ohio State University, Columbus, Ohio, United States|Texas Oncology-Baylor, Dallas, Texas, United States|MD Anderson, Houston, Texas, United States|Virginia Mason Medical Center, Seattle, Washington, United States | https://ClinicalTrials.gov/show/NCT03512756 | |
| 276 | Effects of Heart Imaging Radiation on DNA Double-Stranded Breaks in Blood Lymphocytes and Hair Follicle Cells | Completed | Has Results | Coronary Artery Disease|Chest Pain | Radiation: CCTA ~ BREAST SHIELDS | | https://ClinicalTrials.gov/show/NCT02617888 | |
| 277 | Reduced Intensity Conditioning Transplant Using Haploidentical Donors | Active, not recruiting | No Results Available | Chronic Myelogenous Leukemia|Acute Myelogenous Leukemia|Myelodysplastic Syndrome|Acute Lymphocytic Leukemia|Chronic Lymphocytic Leukemia|Hodgkin's Lymphoma|Non-Hodgkin's Lymphoma|Myelofibrosis|CMML|Multiple Myeloma | Drug: Fludarabine|Drug: Melphalan|Drug: Cyclophosphamide|Procedure: peripheral blood stem cell transplant | Northside Hospital, Atlanta, Georgia, United States | https://ClinicalTrials.gov/show/NCT02581007 | |
| 278 | Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders | Completed | Has Results | Non Malignant Disorders|Immunodeficiencies|Congenital Marrow Failures|Hemoglobinopathies|Inborn Errors of Metabolism|Sickle Cell|Thalassemia|Lysosomal Storage Disease | Biological: Unrelated Umbilical Cord Blood Transplant|Drug: Reduced Intensity Conditioning | Duke University Medical Center Pediatric Blood and Marrow Transplant Program, Durham, North Carolina, United States | https://ClinicalTrials.gov/show/NCT00744692 | |
| 279 | Measurement of Patient Reported Outcomes in Renal Transplant Patients With And Without Gastrointestinal (GI) Symptoms | Completed | No Results Available | Kidney Transplantation|GI Symptoms Severity | | Hospital Universiti Kebangsaan Malaysia, Cheras, Malaysia|Hospital Raja Perempuan Zainab II, Kota Bharu, Malaysia|Hospital Queen Elizabeth, Kota Kinabalu, Malaysia|Hospital Kuala Lumpur, Kuala Lumpur, Malaysia|Hospital Tengku Ampuan Afzan, Kuantan, Malaysia|Hospital Umum Sarawak, Kuching, Malaysia|Hospital Pulau Pinang, Pulau Pinang, Malaysia|Hospital Selayang, Selayang, Malaysia|Hospital Serdang, Serdang, Malaysia | https://ClinicalTrials.gov/show/NCT01300416 | |
| 280 | Phase I Study of Oral BAY 1217389 in Combination With Intravenous Paclitaxel | Completed | No Results Available | Medical Oncology | Drug: BAY1217389|Drug: Paclitaxel | Santa Monica, California, United States|Denver, Colorado, United States|New Haven, Connecticut, United States|Hackensack, New Jersey, United States|Houston, Texas, United States|San Antonio, Texas, United States|Rotterdam, Netherlands | https://ClinicalTrials.gov/show/NCT02366949 | |
| 281 | Phase I Dose Escalation of Oral BAY1161909 in Combination With Intravenous Paclitaxel | Terminated | No Results Available | Medical Oncology | Drug: BAY1161909|Drug: Paclitaxel | Santa Monica, California, United States|New Haven, Connecticut, United States|Boston, Massachusetts, United States|Detroit, Michigan, United States|Nashville, Tennessee, United States|San Antonio, Texas, United States | https://ClinicalTrials.gov/show/NCT02138812 | |
