Phase 3 Muscular Dystrophy Trial, Recruiting

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

Who might be able to join this trial:

• Boys aged 9 to 17 years old at the time of screening
• Those who are able to give written agreement to take part, either by the patient themselves and/or their parent or legal guardian, as required by local rules
• Those who have been diagnosed with Duchenne muscular dystrophy (DMD) confirmed by genetic testing
• Those who use a wheelchair full-time and are either unable to walk 10 metres, or cannot complete that walk within 30 seconds without support or devices
• Those whose arm and upper body function scores fall within a specific range (scores 3 to 6) on a standardised arm function test called the PUL 2.0
• Those taking heart medications related to DMD (such as ACE inhibitors, beta-blockers, or water tablets) where the dose has been stable for at least 1 month before starting the study
• Those who have been on corticosteroids (steroid tablets or similar) for at least 6 months before starting the study, with no major changes to the dose during that time (small adjustments for changes in body weight are acceptable)
• Those willing to use reliable contraception from the time of joining the trial until 3 months after the last dose — acceptable methods include complete abstinence from sexual activity (as a consistent lifestyle choice, not just periodic), or a condom with spermicide combined with an effective method used by the female partner

Who may not be able to join:

• Those who have taken another experimental drug within the 3 months before starting the study
• Those who have used a dystrophin-restoring treatment (such as ataluren or exon-skipping therapies) within 6 months before starting the study
• Those who have previously received any gene therapy for DMD (such as AAV micro-dystrophin treatments)
• Those who have used any medication or supplement (other than corticosteroids) that could affect muscle strength or function within the 3 months before starting — note that vitamin D, calcium, and general supplements are allowed
• Those using testosterone, unless it is being used as a medically prescribed replacement therapy for delayed puberty with a stable dose for at least 6 months and levels within the normal range for age
• Those with significant tightening (contracture) of more than 30 degrees in the elbow of the dominant arm
• Those whose arm function test scores are too inconsistent between repeated measurements at screening (confirm with trial site for exact scoring thresholds)
• Those whose lung capacity (forced vital capacity) is below 40% of what would be expected for their age and size
• Those who need ventilator support during the day (night-time ventilator use and bi-level airway pressure therapy at night are allowed)
• Those who have had a serious breathing failure episode in the 8 weeks before screening
• Those with significant heart failure or heart muscle disease, or a heart pumping function (left ventricular ejection fraction) below 45%
• Those with certain abnormal heart rhythm readings on an ECG, or a personal or family history of specific heart rhythm conditions (confirm with trial site for exact measurements)
• Those who have a major surgical procedure, including back (scoliosis) surgery, planned within 1 year of starting the study
• Those with poorly controlled asthma or ongoing lung conditions such as bronchitis, bronchiectasis, emphysema, or recurring pneumonia that the treating doctor believes could affect breathing
• Those whose blood test results at screening show low platelet counts, low white blood cell counts, or low haemoglobin (these may be repeated once before a final decision is made)
• Those with very high fasting triglyceride (blood fat) levels above 300 mg/dL at screening (this may be repeated once before a final decision is made)
• Those with a current or past history of liver disease or liver problems, including certain abnormal liver blood test results, unless the cause is a specific harmless condition called Gilbert's disease
• Those with poor kidney function, as measured by a specific blood test (serum Cystatin C), if the result is more than twice the upper limit of normal (this may be repeated once before a final decision is made)
• Those who test positive for hepatitis B, hepatitis C, or HIV at screening
• Those with a known allergy or sensitivity to any ingredient in the study medication
• Those with sorbitol intolerance, malabsorption issues, or a hereditary form of fructose intolerance
• Those with another uncontrolled neurological condition or a significant unrelated medical condition, as assessed by the treating doctor
• Those with a psychiatric illness or personal circumstances that the treating doctor believes would prevent reliable participation in the study tests and procedures
• Those for whom an MRI scan is not safe or suitable (for example, due to severe claustrophobia, metal implants in the body, or uncontrolled seizures)

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