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Long-term Follow-Up for RGX-121
Sponsor: REGENXBIO Inc.
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.
Official title: A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-121
Key Details
Gender
MALE
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
27
Start Date
2021-03-14
Completion Date
2028-10
Last Updated
2026-08-19
Healthy Volunteers
No
Conditions
Interventions
Long-term Follow-Up
No intervention
Locations (4)
University of California, San Francisco (UCSF)- Benioff Children's Hospital
San Francisco, California, United States
Saint Peter's Healthcare System
New Brunswich, New Jersey, United States
The Children's Hospital of Philadelphia (CHOP)
Philadelphia, Pennsylvania, United States
Hospital de Clinicas de Porto Alegre
Porto Alegre, Brazil