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This study examined the safety and effect of a gene therapy called rAAV1.CMV.huFollistatin344 delivered into the muscle (intramuscular) in patients with Duchenne muscular dystrophy. It included male patients aged 7 and over with a confirmed DMD gene mutation and impaired muscle function. It was conducted in Phase 1/Phase 2 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 * 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