drugset / Trial / NCT05616793
Safety and Tolerability Subretinal OPGx-001 for LCA5-Associated Inherited Retinal Degeneration (LCA5-IRD) and Non-interventional Arm With Untreated Patients
Non-randomizedParallel-groupOpen-labelOther
Summary
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).
Timeline
- Start
- 2023-06-15
- Primary completion
- 2028-06-15
- Completion
- 2028-06-15
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AAV8.hLCA5 | Gene therapy (AAV / viral vector) | 1e+10 vg | Other |
| Subject | AAV8.hLCA5 | Gene therapy (AAV / viral vector) | 3e+10 vg | Other |
| Subject | AAV8.hLCA5 | Gene therapy (AAV / viral vector) | 1e+11 vg | Other |