Phase 2 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:

• You have been diagnosed with Duchenne muscular dystrophy (DMD) through genetic testing, and your specific genetic change is the type that could be treated by a technique called "exon 44 skipping," as confirmed by a genetic counselor connected to the trial.
• You were assigned male at birth and show physical signs of Duchenne muscular dystrophy, as assessed by the study doctor.
• You are between 4 and 20 years old (inclusive) — this applies to Part A of the trial.
• You are able to walk, and your arm and upper body function meets a specific level measured by a standard test called the PUL 2.0 (confirm with trial site for exact requirements).
• You have enough muscle tissue available for a small tissue sample (biopsy), as determined by the study doctor.
• Other criteria set out in the study protocol also apply (confirm with trial site).

Who may not be able to join:

• You have another significant health condition that could make it difficult to follow the trial's requirements or affect the results.
• You have had an acute (sudden) illness in the 4 weeks before your first dose of the study drug that could affect safety or test results.
• You have previously received any exon skipping therapy or any gene therapy at any point in your life.
• You are currently taking or have recently taken blood-thinning medications (such as anticoagulants, antithrombotics, or antiplatelet drugs).
• You are taking immune-suppressing medications, other than standard oral corticosteroids used for DMD.
• You are currently taking or have previously taken a type of medication called an HDAC inhibitor, including a drug called givinostat.
• You have abnormal blood test results that fall outside acceptable ranges for this trial (confirm with trial site for specifics).
• You need a breathing machine (ventilator) during the day, or you use an invasive ventilator through a tube in your throat (tracheostomy).
• You have an abnormal heart electrical reading (ECG), including a specific measurement called QTcF that is greater than 450 milliseconds, as assessed by the study doctor.
• You have taken another experimental or investigational drug within the past 3 months (or within 5 half-lives of that drug, whichever is longer).
• Other criteria set out in the study protocol also apply (confirm with trial site).

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