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Gene therapy for boys with Duchenne Muscular Dystrophy

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)

Phase 3 Interventional Sarepta Therapeutics, Inc. · NCT05881408

This study is testing a new gene therapy to see if it can help boys with Duchenne Muscular Dystrophy improve their movement and muscle function.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment148 (estimated)
SexMale
SponsorSarepta Therapeutics, Inc. Industry-sponsored
Locations43 sites (Little Rock, Arkansas and 42 other locations)
Trial IDNCT05881408 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety and efficacy of delandistrogene moxeparvovec, a gene transfer therapy, in both non-ambulatory and ambulatory males diagnosed with Duchenne Muscular Dystrophy (DMD). It is a randomized, double-blind, placebo-controlled trial that will last approximately 128 weeks, allowing all participants to receive the treatment in either of the two study parts. The study aims to assess improvements in motor function through standardized assessments.

Who should consider this trial

Good fit: Ideal candidates are males aged 8 to 18 years with a confirmed diagnosis of DMD, either ambulatory or non-ambulatory.

Not a fit: Patients who have previously received gene therapy or treatments aimed at increasing dystrophin expression may not benefit from this study.

Why it matters

Potential benefit: If successful, this therapy could significantly improve motor function and quality of life for patients with Duchenne Muscular Dystrophy.

How similar studies have performed: Other studies involving gene therapy for DMD have shown promising results, indicating potential for success in this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Definitive diagnosis of DMD based on documented clinical findings and prior genetic testing.
* Cohort 1 only: Non-ambulatory per protocol-specified criteria.
* Cohort 2 only: Ambulatory per protocol-specified criteria and ≥8 to \<18 years of age at the time of Screening.
* Ability to cooperate with motor assessment testing.
* Stable daily dose of oral corticosteroids for at least 12 weeks prior to Screening, and the dose is expected to remain constant throughout the study (except for modifications to accommodate changes in weight).
* Recombinant Adeno-Associated Virus Serotype rh74 (rAAVrh74) antibody titers are not elevated as per protocol-specified requirements.
* A pathogenic frameshift mutation or premature stop codon in the DMD gene, except for any deletion mutations in exon 8 and/or 9.

Exclusion Criteria:

* Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol specified time limits.
* Abnormality in protocol-specified diagnostic evaluations or laboratory tests.
* Presence of any other clinically significant illness, medical condition, or requirement for chronic drug treatment that in the opinion of the Investigator creates unnecessary risk for gene transfer.

Other inclusion or exclusion criteria could apply.

Where this trial is running

Little Rock, Arkansas and 42 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Duchenne Muscular DystrophyDMDGene-DeliveryPediatricNorth Star Ambulatory AssessmentPerformance of Upper LimbDuchenne
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.