This study examined the safety of a gene therapy called rAAVrh74.MCK.micro-Dystrophin delivered into the muscle (intramuscular) in patients with Duchenne muscular dystrophy. It included wheelchair-dependent male patients aged 7 and over with a confirmed Dystrophin mutation. It 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: * Age 7 or older; must be wheelchair-dependent * Confirmed Dystrophin mutations based on mutation compatibility with micro-dys cDNA based on previously published methods. * Males of any ethnic group will be eligible. * Ability to cooperate with muscle testing. * Willingness of sexually active subjects with reproductive capacity to practice reliable method of contraception (If appropriate). Exclusion Criteria: * Active viral infection based on clinical observations. * Symptoms or signs of cardiomyopathy, including: * Dyspnea on exertion, pedal edema, shortness of breath upon lying flat, or rales at the base of the lungs * Echocardiogram with ejection fraction below 40% * Serological evidence of HIV infection, or Hepatitis A, B or C infection * Diagnosis of (or ongoing treatment for) an autoimmune disease * Concomitant illness or requirement for chronic drug treatment that in the opinion of the PI creates unnecessary risks for gene transfer. * Subjects with AAVrh74 binding antibody titers ≥ 1:50 as determined by ELISA immunoassay. * Abnormal laboratory values in the clinically significant range as defined in protocol or based upon normal values in the Nationwide Children's Hospital Laboratory.