Shire Human Genetic Therapies, Inc. is sponsoring an observational clinical trial to evaluate the neurodevelopmental status of pediatric patients with MPS II who may show signs of central nervous system involvement. This is an observational study only, therefore no treatment for MPS II with an investigational drug will be offered.
This is a 24-month study during which 30 pediatric patients worldwide will be enrolled in an effort to identify, document and quantify neurodevelopmental deficiencies. The goal of the study is to help establish the onset of and natural rate of cognitive decline in MPS II patients.
To be eligible, your child must meet the following requirements, as verified by a doctor:
• documentation of a deficiency of the iduronate-2-sulfatase enzyme
• documentation of a normal enzyme activity in another enzyme or documented mutation in the iduronate-2-sulfatase gene
• male, age 2 to 12 years of age
• have sufficient hearing capacity, with or without hearing aids, tocomplete the required protocol tests
Contact one of the study centers listed below if you are interested inhaving your child participate in this study or if you have any questions.
Ann & Robert H. Lurie Children’s Hospital of Chicago, IL
Dr. Barbara Burton
Rachel Katz, MSW, clinical trial coordinator
Children’s Hospital Oakland, CA
Dr. Paul Harmatz
Jacqueline Madden, PNP, clinical trial coordinator
UNC Chapel Hill, NC
Dr. Joseph Muenzer
Heather Preiss, RN, clinical trial coordinator