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This study evaluates the drug Eteplirsen (an exon-skipping antisense therapy) in boys with Duchenne muscular dystrophy (DMD, a progressive muscle disease caused by mutations in the DMD gene). It included males aged 7-16 with a genetically confirmed DMD diagnosis who had been on corticosteroids for at least 24 weeks. It was a Phase 3 study and has been completed.
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Male 7-16 years old * Diagnosed with DMD, genotypically confirmed * Stable dose of corticosteroids for at least 24 weeks * Have intact right and left alternative upper muscle groups * Mean 6MWT greater than 300m (primary analysis on 300 to 450 meters) * Stable pulmonary and cardiac function: predicted FVC equal to or greater than 50% and LVEF of greater than 50% Exclusion Criteria: * Previous treatment with drisapersen or any other RNA antisense agent or any gene therapy within the last 6 months * Participation in any other DMD interventional clinical study within 12 weeks * Major surgery within 3 months * Presence of other clinically significant illness * Major change in the physical therapy regime within 3 months Other inclusion/exclusion criteria apply.