Trials / NCT05203939
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
Underway, not enrolling Phase 1 / Phase 2 Gene therapy
- Sponsor
- Ocugen
- Treatment
- OCU400 Low Dose; OCU400 Med Dose; OCU400 High Dose; OCU400 Second Eye Dosing
- Gene
- Not gene-specific (mentions CEP290, NR2E3, RHO)
- Conditions
- Retinitis Pigmentosa, Leber Congenital Amaurosis
- Ages
- 6 Years to no upper limit
- Enrollment
- 22 participants
- Started
- 2022-01-24
- Main results expected
- 2027-03
- Results posted
- Not yet
- Record last updated
- 2026-07-31
What the study is testing
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.
Where it runs
United States (7)
- Associated Retina Consultants, Phoenix, Arizona
- Ocugen Site 5 - University of California, San Diego (UCSD) - Shiley Eye Institute, La Jolla, California
- Ocugen Site 3 - Bascom Palmer Eye Institute, Miami, Florida
- Ocugen Site 6 - Emory University, Atlanta, Georgia
- Ocugen Site 2 - Casey Eye Institute - OHSU, Portland, Oregon
- Ocugen Site 8 - Mid Atlantic Retina - Wills Eye Hospital, Philadelphia, Pennsylvania
- Ocugen Site 1 - Retina Foundation of the Southwest, Dallas, Texas