This study looks at the safety of a gene therapy (rAAV2.5-CMV-minidystrophin) that aims to provide a small form of the dystrophin protein (mini-dystrophin), which is important for muscles, in Duchenne muscular dystrophy. Male patients aged 5 and older with a known mutation in the dystrophin gene were able to take part. Sponsored by Nationwide Children's Hospital, the study was conducted in Phase 1 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: * Known null mutation of the Dystrophin gene * Male age of 5 years or older * If taking corticosteroids, must have dose unchanged for the past 3 months * Serum creatine kinase elevation greater than 10x normal value (established by Children's Hospital) * Progressive, symmetrical proximal muscle weakness of arms and legs Exclusion Criteria: * Unable to cooperate for muscle strength testing * Joint contractures that prohibit muscle strength testing * Concomitant illness * Individuals predisposed to excessive vagal responses (bradyarrhythmia or hypotension) * Controlled substance abuse