Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
NCT05203939 · View on ClinicalTrials.gov ↗
Study 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.
Conditions Studied
Interventions
- DRUG OCU400 Low Dose
- DRUG OCU400 Med Dose
- DRUG OCU400 High Dose
- DRUG OCU400 Second Eye Dosing
Study Locations (7)
Arizona
- Associated Retina Consultants — Phoenix
California
- Ocugen Site 5 - University of California, San Diego (UCSD) - Shiley Eye Institute — La Jolla
Florida
- Ocugen Site 3 - Bascom Palmer Eye Institute — Miami
Georgia
- Ocugen Site 6 - Emory University — Atlanta
Oregon
- Ocugen Site 2 - Casey Eye Institute - OHSU — Portland
Pennsylvania
- Ocugen Site 8 - Mid Atlantic Retina - Wills Eye Hospital — Philadelphia
Texas
- Ocugen Site 1 - Retina Foundation of the Southwest — Dallas
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 22 participants |
| Start Date | 2022-01-24 |
| Est. Completion | 2027-03 |
| Phase | Phase 1 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05203939
The ClinicalTrials.gov registry entry for NCT05203939 describes a study currently listed as active not recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 22 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Ocugen, which has 5 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 2 conditions, with Retinitis Pigmentosa appearing as the primary indexed condition, and to 4 interventions — of which OCU400 Low Dose is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05203939 reports 7 study locations spanning 7 distinct geographic areas — top geographies include Arizona, California, Florida. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT05203939 about?
NCT05203939 is a clinical study titled "Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis". 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 ...
What is the current status of trial NCT05203939?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 22 participants. The study started on 2022-01-24. Estimated completion is 2027-03.
What conditions does trial NCT05203939 study?
This clinical trial studies the following conditions: Retinitis Pigmentosa, Leber Congenital Amaurosis. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05203939?
The interventions under investigation include: OCU400 Low Dose (DRUG), OCU400 Med Dose (DRUG), OCU400 High Dose (DRUG), OCU400 Second Eye Dosing (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05203939?
This trial is sponsored by Ocugen, which has 5 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT05203939 being conducted?
This trial has 7 study locations across Arizona, California, Florida, Georgia, Oregon. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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