Phase 2 Muscular Dystrophy Trial, Recruiting

Written by an AI model from the ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• People who have a confirmed diagnosis of Duchenne muscular dystrophy (DMD), backed by genetic testing that found a mutation in the DMD gene.
• Boys with DMD who are able to walk and are aged 7 years or older but younger than 10 years at the time of screening.
• Boys who have been on a steady dose of a steroid medication (such as prednisolone, deflazacort, or vamorolone) for at least 3 months before screening, and are expected to continue that dose throughout the trial — or boys who stopped steroids at least 3 months before screening.
• Boys whose other regular medicines (such as heart medications like ACE inhibitors or beta-blockers) and any supplements have been at a stable dose for at least 1 month before screening and are expected to stay stable during the trial.
• Boys who previously received the gene therapy delandistrogene moxeparvovec (Elevidys) — either in another trial or as a prescribed treatment — more than 18 months before screening, where their muscle function tests have since stabilised or declined, as confirmed by the treating doctor.
• Boys who previously received an "exon skipping" treatment more than 6 months before screening, where their muscle function has since stabilised or declined, as confirmed by the treating doctor.
• Boys who have been taking givinostat (Duvyzat) at a stable dose for at least 18 months before screening — or boys who had to stop givinostat due to side effects before 18 months, provided their last dose was at least 30 days before screening.
• Boys who received a different investigational gene therapy (other than Elevidys) more than 24 months before screening.
• Boys who are doing a regular physiotherapy or strength training programme, provided the programme has been unchanged for at least 2 months before screening and will stay consistent during the trial.

Who may not be able to join:

• Boys who are expected to lose the ability to walk within 12 months.
• Boys for whom an MRI scan or open muscle biopsy would not be safe or is not recommended.
• Boys showing signs of significant liver problems, based on a specific blood test result at screening (confirm with trial site).
• Boys with reduced heart pumping function — specifically a left ventricular ejection fraction below 50% on a heart scan — or who have symptoms of heart muscle disease.
• Boys whose lung capacity (forced vital capacity) is below 60% of what would be expected for their age and size.
• Boys who are currently taking part in another clinical trial involving a treatment, or are in a follow-up study for a previous treatment.
• Boys who regularly consume grapefruit juice or products containing grapefruit.
• Boys who have significant behavioural or cognitive difficulties that the trial doctor determines would prevent safe participation in the study.

Important: Always verify eligibility with the trial site directly before applying.