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:

• People who have a confirmed genetic diagnosis of Duchenne muscular dystrophy (DMD), specifically with a change in the dystrophin gene that could be addressed by a technique called "exon 45 skipping," as reviewed by a specialist genetic counsellor.
• People who were assigned male at birth and show clinical signs consistent with DMD, as determined by the trial doctor.
• People who are between 4 and 20 years of age (inclusive) — this applies to Part A of the trial.
• People who are able to walk and meet specific upper limb function test requirements set out in the trial protocol at the screening stage (Part A).
• People who have enough muscle tissue available for a biopsy sample, as assessed by the trial doctor.
• Other criteria defined in the trial protocol also apply (confirm with trial site).

Who may not be able to join:

• People who have another significant medical condition that could interfere with following the trial requirements.
• People who have had an acute illness (a sudden or short-term sickness) within 4 weeks before the first dose of the study drug that could affect study measurements or safety.
• People who have used certain medications, including: any exon skipping therapy in the past 12 months; any gene therapy at any point; blood-thinning medications (such as anticoagulants or antiplatelet agents) from 30 days before screening until the end of the study; immune-suppressing medications (other than corticosteroids taken for DMD) from 30 days before screening until the end of the study; or a type of drug called an HDAC inhibitor (for example, givinostat) from 30 days before screening until the end of the study.
• People with certain abnormal blood test results (confirm with trial site for specific details).
• People who need a breathing machine (ventilator) during the day, or who use invasive mechanical ventilation through a surgically placed airway (tracheostomy).
• People who have an abnormal heart electrical reading (ECG) that the trial doctor considers clinically significant, or a specific heart rhythm measurement (QTcF) above 450 milliseconds at screening or before the first dose.
• People who have taken another experimental or investigational drug within 3 months before the first dose, or within 5 half-lives of that drug — whichever period is longer.
• Other criteria defined in the trial protocol also apply (confirm with trial site).

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