NCT05881408

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

A Phase 3, Multinational, Randomized, Double-Blind, Placebo-Controlled Systemic Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of SRP- 9001 in Non-Ambulatory and Ambulatory Subjects With Duchenne Muscular Dystrophy (ENVISION)

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 148 (estimated)
• Sponsor: Sarepta Therapeutics, Inc. · Industry
• Sex: Male
• Age: —
• Healthy volunteers: Not accepted

Summary

The study will evaluate the safety and efficacy of delandistrogene moxeparvovec gene transfer therapy in non-ambulatory and ambulatory males with DMD. This is a randomized, double-blind, placebo-controlled 2-part study. Participants will be in the study for approximately 128 weeks. All participants will have the opportunity to receive intravenous (IV) delandistrogene moxeparvovec in either Part 1 or Part 2.

Conditions

• Duchenne Muscular Dystrophy

Interventions

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

Timeline

• Start date: 2023-05-31
• Primary completion: 2027-05-31
• Completion: 2028-06-30
• First posted: 2023-05-31
• Last updated: 2025-06-22

Locations

46 sites across 14 countries: United States, Australia, Belgium, Canada, Germany, Hong Kong, Israel, Italy, Japan, South Korea, Spain, Sweden, Taiwan, United Kingdom

Regulatory

• FDA-regulated drug study