G.O.L.D. trial of NR082 gene therapy for ND4 mutation-associated Leber hereditary optic neuropathy

Phase 3, randomised, double-blind trial of esonadogene mvoparvovec in Leber hereditary optic neuropathy (LHON) (4 arms), sponsored by Neurophth. Primary endpoints: Incidence of adverse events, serious adverse events, and dose-limiting toxicities within 12 weeks in Stage 1, Incidence of adverse events and serious adverse events within 6 weeks in Stage 2 safety lead-in and 1 more. Started Jun 2021; status: active.