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This is a long-term follow-up study to MPS II Treatment with RGX-121. No new treatment is provided through this study, as its purpose is to provide up to 3 years of observation and monitoring of effects and side-effects following gene-transfer provided in the initial study.
This study is open to those who participated in the base study. It commences at the termination of their involvement in the earlier research.
Boys: Ages 28 months to 7 years
Participants will have an initial enrollment visit, followed by annual visits with the study doctor for medical examinations, tests, and assessments. Additionally, parents/guardians may be contacted by the study team at the 6-month interval between visits for updates on the child’s health. The 3 years of visits and contacts adds to the initial 2 years of follow-up provided by the initial study, for a total patient monitoring period of up to 5 years.
Visits: 7, including contact calls
Duration: 3 years
Study Description at National Institutes of Health
Hunter Syndrome MPS II
Neurodevelopment in Rare Disorders Research
Deepa Soundara Rajan, MD
For more information about the study or enrollment, please contact:
Dawn Kolar, Clinical Research Coordinator
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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