MPS II Treatment with RGX-121 – Long-Term Observation

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-121

Protocol Description

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.

Eligibility Criteria

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

Requirements

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

Status: Enrolling by Invitation

Source(s) of Support

Regenxbio Inc.

Primary Investigator

Maria L. Escolar, MD, MS

Contact Information

For more information about the study or enrollment, please contact:
Jodi Martin, Clinical Research Coordinator
412-692-6351