Phase 1 Muscular Dystrophy Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You have been formally diagnosed with Duchenne muscular dystrophy (DMD), confirmed through medical records and genetic testing.
- Depending on which group (cohort) you are placed in, you may need to be a certain age — ranging from 2 years old up to under 18 years old — and either still able to walk or no longer able to walk.
- If you are in certain groups, you must have been on a stable dose of oral steroid medication for at least 12 weeks before screening, with the dose expected to stay the same for the first year of the study.
- If you are in Cohort 8, you are no longer able to walk, and you must score within a specific range on an arm strength and movement test called the Performance of the Upper Limb (PUL) assessment (confirm with trial site).
- If you are in Cohorts 4 or 6, you are not yet taking regular steroids for DMD and are not on them at the time of screening.
- You are able to cooperate with physical movement and motor skill tests.
- Your blood does not show high levels of antibodies to a specific substance used in the gene therapy (rAAVrh74) (confirm with trial site).
- Your genetic mutation type matches the requirements for the specific group you are being considered for (confirm with trial site).
Who may not be able to join:
- If you are in Cohort 8, you have a known allergy or sensitivity to a medication called sirolimus, which is used as part of that group's treatment.
- You have another serious illness, an autoimmune condition, are on certain long-term medications, or have a significant cognitive impairment that the doctor believes would make gene therapy too risky for you.
- You have previously received gene therapy, or have taken an experimental medication or any treatment aimed at increasing dystrophin levels, within a timeframe specified by the study rules.
- Your results from certain medical tests or lab work fall outside the ranges considered acceptable for this trial.
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Medical Director, Sarepta Therapeutics, Inc.
Phone: 1-888-SAREPTA (1-888-727-3782)
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
Part 1 (Cohorts 1 to 5): Change from Baseline in Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12, as Measured by Western Blot; Part 1 (Cohorts 6 to 8): Quantity of Delandistrogene Moxeparvovec Dystrophin Expression at Week 12 as Measured by Western Blot; Cohort 8: Number of Participants with Acute Liver Injury (ALI)
Can't join this trial?
Data last synced from ClinicalTrials.gov: 27 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.