Long-term Follow-Up for RGX-121
NCT04597385 · Status: ACTIVE_NOT_RECRUITING · Type: OBSERVATIONAL · Enrollment: 27
Last updated 2026-08-19
Summary
RGX-121-5101 is an observational long-term follow-up study to the RGX-121-101 or RGX-121-1102 studies where participants received RGX-121, a gene therapy intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study will evaluate the long-term safety and efficacy of RGX-121.
Conditions
- Mucopolysaccharidosis II
Interventions
- OTHER
-
Long-term Follow-Up
No intervention
Sponsors & Collaborators
- lead INDUSTRY
Eligibility
- Sex
- MALE
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2021-03-14
- Primary Completion
- 2028-10-31
- Completion
- 2028-10-31
Countries
- United States
- Brazil
Study Locations
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