Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis
Public ClinicalTrials.gov record NCT05203939. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT05203939
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 22 participants
Conditions and interventions
Conditions
Interventions
- OCU400 Low Dose Drug
- OCU400 Med Dose Drug
- OCU400 High Dose Drug
- OCU400 Second Eye Dosing Drug
Drug
Eligibility (public fields only)
- Age range
- 6 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jan 23, 2022
- Primary completion
- Feb 28, 2027
- Completion
- Feb 28, 2027
- Last update posted
- Jul 30, 2026
2022 – 2027
United States locations
- U.S. sites
- 7
- U.S. states
- 7
- U.S. cities
- 7
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Associated Retina Consultants | Phoenix | Arizona | 85020 | — |
| Ocugen Site 5 - University of California, San Diego (UCSD) - Shiley Eye Institute | La Jolla | California | 92093 | — |
| Ocugen Site 3 - Bascom Palmer Eye Institute | Miami | Florida | 33136 | — |
| Ocugen Site 6 - Emory University | Atlanta | Georgia | 30322 | — |
| Ocugen Site 2 - Casey Eye Institute - OHSU | Portland | Oregon | 97239 | — |
| Ocugen Site 8 - Mid Atlantic Retina - Wills Eye Hospital | Philadelphia | Pennsylvania | 19107 | — |
| Ocugen Site 1 - Retina Foundation of the Southwest | Dallas | Texas | 75231 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05203939, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jul 30, 2026 · Synced Sep 6, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT05203939 live on ClinicalTrials.gov.