A Phase 1-2 multicenter study to evaluate the safety and tolerability of intravenous ATA-100, adeno-associated viral vector carrying the FKRP gene, in patients with FKRP-related limb-girdle muscular dystrophy (LGMDR9, formerly LGMD2I)

FKRP-related limb-girdle muscular dystrophy (LGMD R9)

Safety and tolerability of ATA-100, as measured by incidence of treatment emergent adverse events, incidence of serious adverse events, and incidence of clinically significant laboratory changes.

Change from baseline in muscular function tests (NSAD score, 10MWT, etc.) and pulmonary function test (FVC) at one year post-IMP administration, Change from baseline in muscle MRI parameters (fat fraction, T2 water content) at one year post-IMP administration, Change from baseline in other respiratory assessments (IC, MIP/MEP, SNIP) at one year post-IMP administration, Change from baseline in muscle biomarkers (histological features, biodistribution, transgene expression) at one year post-IMP administration, Change from baseline in patient reported outcome and quality-of-life assessment (gNMD, Activlim) at one year post-IMP administration, Change from baseline in biomarkers (creatine kinase, myomesin-3, circulating microRNA) at one year post-IMP administration

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Denmark, France