PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy

Phase 1, open-label, single-arm trial of PYC-001 in autosomal dominant optic atrophy (ADOA), sponsored by PYC Therapeutics. Primary endpoints: Incidence, type, severity and relationship of ocular treatment-emergent adverse events (TEAEs), and treatment-emergent serious adverse events (SAEs) in the study eye at 48 weeks, Incidence, type, severity and relationship of ocular TEAEs, and treatment-emergent SAEs in the fellow eye at 48 weeks and 4 more. Started Oct 2024; status: active, not recruiting.