United States Clinical Trial News

The National MPS Society recognizes research and clinical trials for MPS and ML exist around the world.  We are pleased to share some ongoing clinical trials, with current enrollment, listed below.  For more information about ongoing clinical trials, please visit: www.clinicaltrials.gov. Please contact Terri Klein for any additional questions.

 

Hurler Syndrome ( MPS I ) Clinical Trial Monthly News:

Regenxbio – RGX-111 Gene Therapy in Patients with MPS I

Clinical Trial, interventional, open label, multicenter study

https://clinicaltrials.gov/ct2/show/NCT03580083?term=regenxbio&cond=MPS+I&rank=1

 

Sangamo – Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-318 in Subjects with MPS I

Clinical Trial

https://clinicaltrials.gov/ct2/show/NCT02702115?term=sangamo&rank=3

 

Hunter Syndrome (MPS II) Clinical Trial Monthly News:

Regenxbio RGX 121 Gene Therapy in Patients with MPS II

https://clinicaltrials.gov/ct2/show/NCT03566043?cond=Hunter+Syndrome&draw=2&rank=11

 

Sangamo – Ascending Dose Study of Genome Editin g by the Zinc Finger Nuclease (SFN() Therapeutic SB-913 in Subjects with MPS II)

Clinical Trial

https://clinicaltrials.gov/ct2/show/NCT03041324?term=sangamo&rank=2

 

UCSF Benioff Children’s Hospital Oakland

Ascending Dose Study of Fenome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB -913 in Subjects with MPS II

https://clinicaltrials.gov/ct2/show/NCT03041324?cond=Hunter+Syndrome&draw=4&rank=22

 

Sanfilippo Syndrome (MPS IIIA) Clinical Trial Monthly News:

Abeona Phase ½ Clinical Trial of ABO-102

Clinical Trial

https://clinicaltrials.gov/ct2/show/NCT02716246?term=abeona&cond=MPS+IIIA&rank=1

 

SOBI  A Study to Assess the Safety, Tolerability, and Efficacy of Long -term SOBI003 Treatment in Pediatric MPS IIIA Patients

Clinical Trial – Single Arm, multi-center study for MPS IIIA – Open Enrollment

https://clinicaltrials.gov/ct2/show/NCT03811028?term=sobi&cond=Sanfilippo+Syndrome&rank=1

 

Lysogene Study of AAVrh10-h.SGSH Gene Therapy in Patients with MPS IIIA.

Clinical Trial – the study is interventional, single arm and multi center.

https://clinicaltrials.gov/ct2/show/NCT03612869?term=lysogene&cond=Sanfilippo+Syndrome&rank=3

 

Sanfilippo Syndrome ( MPS IIIB) Clinical Trial Monthly News:

Abeona – Phase ½ Clinical Trial of ABO-101

Clinical Trial

https://www.abeonatherapeutics.com/clinical-trials/mps-iiib

 

BioMarin – A Treatment Study fo MPS IIIB

Clinical Trial – Interventional, Phase 1/.2 Open Label Dose Escalation Study

https://clinicaltrials.gov/ct2/show/NCT02754076?cond=MPS+III+B&draw=2&rank=2