NCT06388200Active, not recruitingPhase 3
A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa
A Phase 3, Multi-Center, Randomized Study to Assess The Efficacy, Safety and Tolerability of Subretinal OCU400 Gene Therapy for the Treatment of Retinitis Pigmentosa
Source: ClinicalTrials.gov · Last updated 31 Jul 2026
Brief summary (registry, in English)
This is a Phase 3 study to Assess the Efficacy, Safety and Tolerability of OCU400 in patients with retinitis pigmentosa (RP) associated with RHO mutations and patients with any other RP associated mutation with a clinical phenotype of RP. This is a multicenter, assessor blinded and randomized study which will enroll 140 subjects. Study has completed enrollment of all 140 subjects.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 140 (actual)
- First submitted
- 9 Apr 2024
- Start date
- 18 Jun 2024
- Primary completion date
- 12 Feb 2027
- Lead sponsor
- Ocugen
- Diseases
- Retinitis pigmentosa
- NCT number
- NCT06388200
What you commit to
- Study type: Interventional · Phase 3
- Participants enrolled: 140
- From 18 Jun 2024 to the primary completion date, 12 Feb 2027
- 17 sites in 2 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Previous treatment with a gene-therapy or cell therapy product or treatment with any investigational drug or ocular device within one year.
- Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months.
Other illness
- History of any corticosteroid contraindication, corticosteroid related IOP spikes or uncontrolled glaucoma.
- Active ocular/intraocular infection, any history of rhegmatogenous retinal detachment or Current retinal detachment or retinal implant.
Other
- Subject lacks evidence of outer nuclear layer
- Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception
Who the study is looking for
- Males or females ≥ 3 years of age
- Confirmed genetic diagnosis of autosomal dominant RHO mutation with clinical diagnosis of RP
- Clinical Diagnosis of Syndromic or Non-Syndromic RP with/without confirmed genetic diagnosis of any other RP associated mutation (except AD-NR2E3)
- BCVA ≤ 80 letters and ≥25 letters as measured by an ETDRS chart
- Visual field of \>5° in any meridian as measured by a III4e isopter or equivalent
- Able to perform a Luminance LDNA at certain light intensity at the Screening visit
- Presence of photoreceptors as determined by SD-OCT
Age in the registry: 3 years to - · Sex: All · Does not accept healthy volunteers
Sites (17)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Calgary Retina Consultants Coordinator of this site? Confirm its status | Calgary | Canada | Unknown status |
| University of Alberta Coordinator of this site? Confirm its status | Edmonton | Canada | Unknown status |
| Research Institute of the McGill University Health Centre Coordinator of this site? Confirm its status | Montreal | Canada | Unknown status |
| Sunnybrook Health Sciences Centre Coordinator of this site? Confirm its status | Toronto | Canada | Unknown status |
| The University of British Columbia Coordinator of this site? Confirm its status | Vancouver | Canada | Unknown status |
| Retina Consultants of Texas Coordinator of this site? Confirm its status | Bellaire | United States | Unknown status |
| Retina Foundation of the Southwest Coordinator of this site? Confirm its status | Dallas | United States | Unknown status |
| Advanced Research LLC Coordinator of this site? Confirm its status | Deerfield Beach | United States | Unknown status |
| Erie Retina Research LLC Coordinator of this site? Confirm its status | Erie | United States | Unknown status |
| Baylor College of Medicine Coordinator of this site? Confirm its status | Houston | United States | Unknown status |
| Gundersen Health System Coordinator of this site? Confirm its status | La Crosse | United States | Unknown status |
| University of Southern Califormia Coordinator of this site? Confirm its status | La Jolla | United States | Unknown status |
| University of Southern California, Roski Eye Insitute Coordinator of this site? Confirm its status | Los Angeles | United States | Unknown status |
| Valley Retina Institute Coordinator of this site? Confirm its status | McAllen | United States | Unknown status |
| Bascom Palmer Eye Institute, University of Miami, Miller School of Medicine Coordinator of this site? Confirm its status | Miami | United States | Unknown status |
| Vanderbilt Eye Institute Coordinator of this site? Confirm its status | Nashville | United States | Unknown status |
| Associated Retina Consultants Coordinator of this site? Confirm its status | Phoenix | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.