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Long Term Follow-up for RGX-202
Sponsor: REGENXBIO Inc.
Summary
RGX-202-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-202 in participants who have received RGX-202 (an investigative gene therapy designed to deliver a transgene for novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain) in a separate parent study.
Official title: A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-202 Gene Therapy in Males With Duchenne Muscular Dystrophy (DMD)
Key Details
Gender
MALE
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
66
Start Date
2024-05-08
Completion Date
2029-12-01
Last Updated
2026-03-24
Healthy Volunteers
No
Conditions
Interventions
No Intervention
Observational study
Locations (5)
Arkansas Children's Hospital
Little Rock, Arkansas, United States
Stanford School of Medicine /Division of Neuromuscular Medicine
Palo Alto, California, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
The University of Texas Southwestern Medical Center
Dallas, Texas, United States
Virginia Commonwealth University
Richmond, Virginia, United States