NCT06270719

An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy

A Long-term Multicenter Prospective Observational Study Evaluating the Comparative Effectiveness and Safety of Sarepta Gene Transfer Therapy vs. Standard of Care in Participants With Duchenne Muscular Dystrophy Under Conditions of Routine Clinical Practice

• Status: Enrolling By Invitation
• Phase: —
• Study type: Observational
• Enrollment: 500 (estimated)
• Sponsor: Sarepta Therapeutics, Inc. · Industry
• Sex: Male
• Age: 4 Years
• Healthy volunteers: Not accepted

Summary

This is a multicenter, prospective, observational Phase 4 study in the United States. The study is designed to collect both medical history and prospective data on Duchenne muscular dystrophy (DMD) treatment outcomes in participants receiving delandistrogene moxeparvovec as part of clinical care, compared to participants with DMD receiving or prescribed to start chronic glucocorticoid treatment at baseline in routine clinical practice.

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
GENETIC	Delandistrogene Moxeparvovec	No study medication will be provided by the sponsor during this study.
DRUG	Standard of Care	No study medication will be provided by the sponsor during this study.

Timeline

• Start date: 2024-02-07
• Primary completion: 2029-12-31
• Completion: 2038-12-31
• First posted: 2024-02-21
• Last updated: 2025-12-02

Locations

25 sites across 1 country: United States

Regulatory

• FDA-regulated drug study