Phase 2 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:
- Boys between 2 and 7 years old (inclusive) at the time of consent
- Boys who have a confirmed DMD diagnosis through genetic testing, where the specific gene change falls within a region known as exons 45 to 55
- Boys whose symptoms match what doctors typically see in DMD, as assessed by the trial doctor
- Boys aged 2 to under 4 years who are able to walk at least 10 metres on their own (without walking aids) and can get up from the floor without someone physically helping them (using the hands-on-knees climbing-up method is fine)
- Boys aged 4 to 7 years who are able to walk at least 100 metres on their own (without walking aids) and whose score on a standard movement ability test (called the NSAA) falls between 16 and 29
- Boys who have received the standard childhood vaccinations recommended in their country
- Boys whose parent(s) or legal guardian(s) are willing and able to give written permission before the trial begins, and where required by local rules, the child also gives agreement
- Boys and their parent(s) or legal guardian(s) who are willing to take part in a long-term follow-up study after this trial ends
Who may not be able to join:
- Boys who have previously received any gene therapy, gene editing therapy, or cell-based therapy at any point in their life
- Boys who have taken part in another experimental treatment or investigational drug study within the 6 months before the trial start date
- Boys who have used any treatment aimed at increasing dystrophin (a key muscle protein) — including exon-skipping therapies — within 6 months before the scheduled trial start, or who are unwilling to avoid starting or restarting such therapies for at least 5 years after receiving the gene therapy in this trial
- Boys who are currently enrolled in another clinical trial that involves an active treatment or intervention (observational studies with no treatment are generally acceptable — confirm with trial site)
- Boys whose blood test shows antibodies against a substance called AAV9 (a component used to deliver the therapy)
- Boys who have any medical condition that would make it unsafe to receive immune-suppressing medications
- Boys whose genetic change falls in a different region of the gene (exons 1–44 or exons 56–79) rather than the required region
- Boys who show signs of heart muscle disease or reduced heart pumping function, specifically a heart measurement called LVEF below 50% on a heart ultrasound scan
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
Phone: (800) 593-0346
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
Incidence, severity, and causality of treatment-emergent adverse events and serious adverse events
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.