NCT03769116

A Randomized, Double-blind, Placebo-controlled Study of Delandistrogene Moxeparvovec (SRP-9001) for Duchenne Muscular Dystrophy (DMD)

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial for Duchenne Muscular Dystrophy Using SRP-9001

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 41 (actual)
• Sponsor: Sarepta Therapeutics, Inc. · Industry
• Sex: Male
• Age: 4 Years – 7 Years
• Healthy volunteers: Not accepted

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).

Conditions

• Muscular Dystrophy, Duchenne

Interventions

Type	Name	Description
GENETIC	delandistrogene moxeparvovec	Single IV infusion of delandistrogene moxeparvovec
GENETIC	placebo	Single IV infusion of matching placebo

Timeline

• Start date: 2018-12-05
• Primary completion: 2020-12-08
• Completion: 2023-08-16
• First posted: 2018-12-07
• Last updated: 2024-11-14
• Results posted: 2023-09-15

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study