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 must be a boy
- You must be between 4 and 9 years old (inclusive)
- Your weight or BMI must be at or below the 95th percentile for your age (not significantly above the healthy range for your size)
- You must have a confirmed diagnosis of Duchenne Muscular Dystrophy (DMD) proven through genetic testing
- You must be able to perform certain movement tests at the start of the trial, including a walking ability assessment scoring above a set level, a timed stand-up test of 7 seconds or less, and/or walking at least 350 metres in 6 minutes (confirm with trial site for exact requirements based on age)
- You must already be taking steroid (corticosteroid) medication, or be about to start it, as part of your normal care before the trial screening visit
- A parent or legal guardian must sign a consent form agreeing to participation
- You must be registered with or covered by a healthcare scheme (depending on your country's rules)
Who may not be able to join:
- Boys who have a heart muscle problem where the heart is not pumping strongly enough (below a specific level measured by an ultrasound scan)
- Boys who need any breathing support during the day or night
- Boys who have other health conditions or have had (or are planning) surgery that might affect how DMD progresses or how results are measured
- Boys who are unable to cooperate with muscle strength testing
- Boys who have metal implants in body areas that need to be scanned by MRI
- Boys or families who are unwilling or unable to follow all the study rules and attend all required visits
- Boys who have taken part in another clinical trial using an experimental medicine within the last 3 months (or within the required washout period, whichever is longer)
- Boys who have previously received a gene therapy treatment for DMD, including gene transfer or gene editing products, or who have recently taken an exon skipping drug within a specific timeframe before the trial
- Boys who are currently taking part in any other interventional clinical 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: Francesco MUNTONI, Pr, GOSH LONDON
Phone: +44 02079052602
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
NSAA scale; 10 Meter Walk/ Run test (10MW/RT); Raise from floor (RFF); 6 Minutes Walk Test (6 MWT); Myoset : Myo-grip, -pinch; Stride velocity 95th centile (SV95c); Muscle Imaging Nuclear Magnetic Resonance Imaging (NMRI); Pulmonary Function Test (PFT); ECG - Echocardiography; ACTIVLIM; EQ-5D
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.