This open-label, dose-escalation study evaluated the safety of the gene transfer therapy SRP-9004 (patidistrogene bexoparvovec) given by isolated limb infusion (ILI). The Phase 1/2 trial enrolled about 6 participants with LGMD2D who had confirmed alpha-sarcoglycan deficiency or an identified SGCA gene mutation. The first cohort consisted of wheelchair-dependent adults, while the other cohorts enrolled participants aged 7 and older.
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.
Key Inclusion Criteria: * Cohort 1A must be adult and wheelchair-dependent; Cohorts 1B and 2 will be participants of age 7 or older. * Confirmed alpha-sarcoglycan deficiency or identified sarcoglycan alpha (SGCA) deoxyribonucleic acid (DNA) mutation. * Participants enrolled in Cohorts 1B or 2 must be able to walk independently, but must exhibit signs of lower extremity weakness (that is, a Gowers' sign, use a handrail for climbing stairs) and walk ≤80% of predicted distance on the 6 minute walk test (6MWT) based on normative data. Key Exclusion Criteria: * Active viral infection based on clinical observations. * The presence of SGCA mutations without weakness or loss of function. * Symptoms or signs of cardiomyopathy. * Serological evidence of human immunodeficiency virus (HIV), Hepatitis B, or C infection. * Diagnosis of (or ongoing treatment for) an autoimmune disease. * Participants with AAVrh74 or AAV8 binding antibody titers ≥ 1:50 as determined by enzyme-linked immunosorbent assay (ELISA) immunoassay. Other inclusion/exclusion criteria apply.