drugset / Trial / NCT05203939
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
Non-randomizedSequentialOpen-labelTreatment
Summary
This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 in patients with retinitis pigmentosa associated with NR2E3 and RHO mutations and in patients with LCA due to mutation(s) in CEP290 gene (OCU400-101). To document prospective eye pathology in the above subjects Investigators will also conduct a Natural History Study (OCU400-104)i This is a multicenter study, which will be conducted in two phases and will enroll up to a total of 24 subjects in the OCU400-101 and 100 subjects in the OCU400-104 study.
Timeline
- Start
- 2022-01-24
- Primary completion
- 2027-03
- Completion
- 2027-03
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | BALURETGENE PARVEC | Other / unclassified | 1.66e+10 vg | Other |
| Subject | BALURETGENE PARVEC | Other / unclassified | 3.33e+10 vg | Other |
| Subject | BALURETGENE PARVEC | Other / unclassified | 1e+11 vg | Other |
| Subject | BALURETGENE PARVEC | Other / unclassified | 1.66e+11 vg | Other |