Trials / NCT05616793
Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients
Recruiting now Phase 1 / Phase 2 Gene therapy
- Sponsor
- Opus Genetics, Inc
- Treatment
- AAV8.hLCA5
- Gene
- LCA5
- Conditions
- LCA5
- Ages
- 4 Years to no upper limit
- Enrollment
- 22 participants
- Started
- 2023-06-15
- Main results expected
- 2028-06-15
- Results posted
- Not yet
- Record last updated
- 2026-02-19
What the study is testing
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).
Where it runs
United States (2)
- University of Pennsylvania Perelman School of Medicine, Philadelphia, Pennsylvania (recruiting now)
- Retina Foundation of the Southwest, Dallas, Texas (recruiting now)