A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)
Summary
The purpose of this study is to evaluate the safety and efficacy of exogenous gene transfer in DMD participants by measuring biological and clinical endpoints in three parts: two 48-week randomized, double-blinded, placebo-controlled periods (Part 1 and Part 2), and an open-label follow-up period (Part 3). Participants who are randomized to placebo in Part 1 will have the opportunity for treatment with delandistrogene moxeparvovec in Part 2. In order to provide a uniform approach to monitoring long-term safety and efficacy in participants who received SRP-9001 in a clinical trial, the Sponsor has amended Study Completion for this study to occur at Week 130. Therefore, participants have transitioned and will complete the remainder of the Part 3 follow up visits in a long-term extension study, SRP-9001-305 (NCT05967351).
Timeline
- Start
- 2018-12-05
- Primary completion
- 2020-12-08
- Completion
- 2023-08-16
Outcome
Mixed primary results
registry MEAN Delandistrogene Moxeparvovec Switched Over to Placebo: Change from Baseline: 23.82 (Standard Deviation 39.76) percent normal NCT03769116 ↗
registry analysis (other test); Delandistrogene Moxeparvovec Switched Over to Placebo vs Placebo Switched Over to Delandistrogene Moxeparvovec; p < 0.0001 NCT03769116 ↗
registry LEAST_SQUARES_MEAN Delandistrogene Moxeparvovec Switched Over to Placebo: 1.7 (Standard Error 0.6) score on a scale NCT03769116 ↗
registry analysis (other test); Delandistrogene Moxeparvovec Switched Over to Placebo vs Placebo Switched Over to Delandistrogene Moxeparvovec; p = 0.3730 NCT03769116 ↗
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | delandistrogene moxeparvovec | Gene therapy (AAV / viral vector) | — | Intravenous |