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Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy
Bu çalışma, rAAV1.CMV.huFollistatin344 adlı bir gen terapisinin kasa içine (intramüsküler) uygulanarak Duchenne musküler distrofi (Duchenne muscular dystrophy) hastalarındaki güvenliğini ve etkisini incelemiştir. Çalışmaya, DMD gen mutasyonu doğrulanmış, 7 yaş ve üzeri, kas fonksiyonu bozulmuş erkek hastalar dahil edilmiştir. Çalışma Faz 1/Faz 2 aşamasında yürütülmüş ve tamamlanmıştır.
Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.
Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.
Inclusion Criteria: * Age 7 or older * Confirmed DMD gene mutations * Impaired muscle function based on clinical evidence including difficulty climbing stairs, getting from the floor (Gowers' sign), and weakness of individual muscles of extremities * Males of any ethnic group will be eligible * Ability to cooperate with study procedures including muscle testing. * Willingness of sexually active subjects with reproductive capacity to practice reliable method of contraception * Subjects must be on stable dose of prednisone for three months at time of enrollment or be started on oral dose of daily prednisone regimen for 30 days prior to gene transfer. Study participants will continue prednisone post gene transfer unless there is adverse event that warrants prednisone taper or withdrawal. Exclusion Criteria: * Active viral infection based on clinical observations. * The presence of a DMD gene mutation without weakness or loss of function * 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 rAAV1 binding antibody titers \> 1:50 as determined by ELISA immunoassay * Abnormal laboratory values for liver, kidney, CBC, in the clinically significant range, based upon normal values in the Nationwide Children's Hospital Laboratory