Status: Recruiting
Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients
The goals of this clinical trial are assess the natural course of LCA5-IRD over 6 months and to evaluate the safety and preliminary efficacy of subretinal gene therapy with OPGx-001 in patients with inherited retinal degeneration due to biallelic mutations in the LCA5 gene. Funding Source- FDA Office of Orphan Products Development (OOPD).
Participants needed: 22
Trial details
Phase: Phase 1, Phase 2Age: 4+Biological sex: AllType: InterventionalSponsor: Opus Genetics, IncUpdated: Feb 19, 2026Locations: 2
Eligibility criteria
Are willing and able to provide written informed consent (ICF) and, where approp... [+7]
Women who are pregnant or individuals (women of childbearing potential and men)... [+8]