drugset / Trial / NCT05693142
AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD)
Non-randomizedParallel-groupOpen-labelTreatment
Summary
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. For additional information on how to participate (or be considered for the study), please follow this link: https://mytomorro.ws/affinity-ct-gov
Timeline
- Start
- 2023-01-04
- Primary completion
- 2026-08
- Completion
- 2028-08
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Ompenaclid | Small molecule | 1e+14 unknown | Intravenous |
| Subject | Ompenaclid | Small molecule | 2e+14 unknown | Intravenous |