AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
Running, not enrolling · Phase 2/Phase 3
Conditions studied: Duchenne Muscular Dystrophy
In brief
RGX-202 is a gene therapy designed to deliver a transgene for a novel microdystrophin that includes functional elements of naturally-occurring dystrophin including the C-Terminal (CT) domain. This is a multicenter, open-label dose evaluation clinical study to assess the safety, tolerability, and clinical efficacy of a one-time intravenous (IV) dose of RGX-202 in participants with Duchenne.
Key facts
- Study ID
- NCT05693142
- Run by
- REGENXBIO Inc.
- People needed
- 65
- Starts
- 2023-01-04
- Expected to finish
- 2028-06-01
- Last updated by the study team
- 2026-07-21
Who can join
Age: 1 and older. Sex: male. Healthy volunteers: not accepted.
Where it is running
- Arkansas Children's Hospital — Little Rock, Arkansas, United States
- Children's Hospital of Orange County — Orange, California, United States
- Stanford School of Medicine /Division of Neuromuscular Medicine — Palo Alto, California, United States
- Children's Hospital Colorado — Aurora, Colorado, United States
- University of Florida — Gainesville, Florida, United States
- Rare Disease Research — Atlanta, Georgia, United States
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
- University of Iowa — Iowa City, Iowa, United States
- University of Kansas Medical Center — Kansas City, Kansas, United States
- University of Massachusetts Chan Medical School — Worcester, Massachusetts, United States
- Helen DeVos Children's Hospital — Grand Rapids, Michigan, United States
- Columbia University Medical Center — New York, New York, United States
- Cincinnati Children's — Cincinnati, Ohio, United States
- Nationwide Children's Hospital — Columbus, Ohio, United States
- Oregon Health & Science University — Portland, Oregon, United States
- Monroe Carell Children's Hospital at Vanderbilt — Nashville, Tennessee, United States
- The University of Texas Southwestern Medical Center — Dallas, Texas, United States
- Children's Hospital of the King's Daughters — Norfolk, Virginia, United States
- Children's Hospital of Richmond at Virginia Commonwealth University — Richmond, Virginia, United States
- BC Children's Hospital — Vancouver, British Columbia, Canada
- Children's Hospital London Health Science Centre — London, Ontario, Canada
- Children's Hospital of Eastern Ontario — Ottawa, Ontario, Canada
- The Hospital for Sick Children — Toronto, Ontario, Canada
Full record on ClinicalTrials.gov
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