The purpose of the ATA-003-GSAR Phase 1 study is to evaluate the safety and tolerability of a single intravenous infusion of ATA-200 in pediatric patients with limb-girdle muscular dystrophy type 2C/R5 (LGMD R5). Eligible participants are ambulant boys and girls aged 6 to under 12 at screening with a confirmed LGMDR5 diagnosis made before age 10 based on clinical presentation and genotyping. They must also be able to complete the 10-meter walk test in less than 15 seconds and to rise from a chair with or without support.
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: * Confirmed diagnosis of LGMDR5 before age of 10, based on clinical presentation and genotyping * Ambulant male or female patients aged 6 to less than 12 years of age at screening * Able to perform the 10-meter walk test (10MWT) in less than 15 sec and to rise from chair with or without arm support Exclusion Criteria: * Detectable neutralizing antibodies against AAV8 * Cardiomyopathy with left ventricular ejection fraction (LVEF) \< 50% * Respiratory assistance * Concomitant medical condition that might interfere with LGMDR5 evolution * Acute illness within 4 weeks of anticipated IMP administration * Current participation in another clinical trial with investigational medicinal product * Previous participation in gene and cell therapy trials * Any condition that would contraindicate immunosuppressant treatment * Presence of any permanent items (e.g., metal braces) precluding undergoing MRI * Any vaccination 1 month prior to planned IMP administration * Serology consistent with HIV exposure or active hepatitis B or C infection * Grade 2 or higher lab abnormalities for liver function tests, creatinine, hemogram and coagulation