Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis (NCT05203939)
A Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa Associated With NR2E3 and RHO Mutations and Leber Congenital Amaurosis With Mutation(s) in CEP290 Gene
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Associated Retina Consultants — Phoenix, Arizona
Age range: 6 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (7 Sites)
- 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
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Retinitis Pigmentosa
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- Prospective Natural History Study of Retinitis Pigmentosa — Pittsburgh, Pennsylvania
- Study to Evaluate Safety of RTx-015 Injection in Retinitis Pigmentosa or Choroideremia Patients (ENVISION) — Beverly Hills, California
- FIGHT-RP 1 Extension Study — Baltimore, Maryland
- Promising ROd-cone DYstrophy Gene therapY — Miami, Florida
Explore by condition: All Retinitis Pigmentosa clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.