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

NCT05096221 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 126

Last updated 2025-07-08

Study results available
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Summary

The study will evaluate the safety and efficacy of gene transfer therapy in boys with DMD. It is a randomized, double-blind, placebo-controlled study. The participants who are randomized to the placebo arm will have an opportunity for treatment with gene transfer therapy at the beginning of the second year.

Conditions

Interventions

GENETIC

delandistrogene moxeparvovec

Single IV infusion of delandistrogene moxeparvovec.

GENETIC

placebo

Single IV infusion of matching placebo.

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Sarepta Therapeutics, Inc.

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
4 Years
Max Age
7 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-10-27
Primary Completion
2023-10-04
Completion
2024-10-25
FDA Drug
Yes

Countries

  • United States
  • Belgium
  • Germany
  • Hong Kong
  • Italy
  • Japan
  • Spain
  • Taiwan
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05096221 on ClinicalTrials.gov