Clinical Intramuscular Gene Transfer Trial of rAAVrh74.MCK.Micro-Dystrophin to Patients With Duchenne Muscular Dystrophy
Bu çalışma, rAAVrh74.MCK.micro-Dystrophin adlı bir gen terapisinin kasa içine (intramüsküler) uygulanarak Duchenne musküler distrofi (Duchenne muscular dystrophy) hastalarındaki güvenliğini incelemiştir. Çalışmaya, Dystrophin mutasyonu doğrulanmış, tekerlekli sandalyeye bağımlı, 7 yaş ve üzeri erkek hastalar dahil edilmiştir. Çalışma Faz 1 aşamasında yürütülmüş ve tamamlanmıştır.
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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.