Gene therapy for treating 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
This study is testing a new gene therapy called OCU400 to see if it can improve vision in people with retinitis pigmentosa who have specific genetic mutations.
Quick facts
| Phase | Phase 3 |
|---|---|
| Study type | Interventional |
| Enrollment | 150 (estimated) |
| Ages | 8 Years and up |
| Sex | All |
| Sponsor | Ocugen Industry-sponsored |
| Locations | 15 sites (Phoenix, Arizona and 14 other locations) |
| Trial ID | NCT06388200 on ClinicalTrials.gov |
What this trial studies
This Phase 3 study evaluates the efficacy, safety, and tolerability of OCU400 gene therapy in patients with retinitis pigmentosa (RP), specifically those with RHO mutations and other RP-associated mutations. The study will enroll 150 participants, divided into two arms: one for those with confirmed RHO mutations and another for those with various RP mutations. Participants will be randomized to receive either the treatment or a control, with the treatment involving a bilateral sub-retinal injection of OCU400. The study aims to assess the impact of this gene therapy on visual function over a 12-month follow-up period.
Who should consider this trial
Good fit: Ideal candidates include individuals aged 8 and older with a clinical diagnosis of retinitis pigmentosa and a confirmed genetic mutation associated with the condition.
Not a fit: Patients who have previously received gene therapy or have significant retinal damage may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could significantly improve vision and quality of life for patients with retinitis pigmentosa.
How similar studies have performed: Previous studies on gene therapy for retinal diseases have shown promising results, indicating potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. Males or females ≥ 8 years of age 2. Clinical diagnosis of RP with confirmed genetic diagnosis of autosomal dominant RHO mutation or any other mutation associated with RP. 3. BCVA ≤ 75 letters and ≥25 letters as measured by an ETDRS chart. 4. Visual field of \>5° in any meridian as measured by a III4e isopter or equivalent. 5. Able to perform a Luminance LDNA at certain light intensity at the Screening visit. 6. Presence of photoreceptors as determined by SD-OCT Exclusion Criteria: 1. Subject lacks evidence of outer nuclear layer 2. Previous treatment with a gene-therapy or cell therapy product or treatment with any investigational drug or ocular device within one year. 3. History of corticosteroid related IOP spikes or uncontrolled glaucoma. 4. Absence of large regions of sensitivity in the pericentral and peripheral retinal regions 5. Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months. 6. Active ocular/intraocular infection, any history of rhegmatogenous retinal detachment or Current retinal detachment or retinal implant. 7. Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception
Where this trial is running
Phoenix, Arizona and 14 other locations
- Associated Retina Consultants — Phoenix, Arizona, United States (Recruiting)
- University of Southern Califormia — La Jolla, California, United States (Recruiting)
- University of Southern California, Roski Eye Insitute — Los Angeles, California, United States (Recruiting)
- Advanced Research, LLC. — Deerfield Beach, Florida, United States (Recruiting)
- Bascom Palmer Eye Institute, University of Miami, Miller School of Medicine — Miami, Florida, United States (Recruiting)
- Erie Retina Research LLC — Erie, Pennsylvania, United States (Recruiting)
- Vanderbilt Eye Institute — Nashville, Tennessee, United States (Recruiting)
- Retina Consultants of Texas — Bellaire, Texas, United States (Recruiting)
- Retina Foundation of the Southwest — Dallas, Texas, United States (Recruiting)
- Valley Retina Institute — McAllen, Texas, United States (Recruiting)
- Gundersen Health System — La Crosse, Wisconsin, United States (Recruiting)
- Calgary Retina Consultants — Calgary, Alberta, Canada (Recruiting)
- University of Alberta — Edmonton, Alberta, Canada (Not_yet_recruiting)
- The University of British Columbia — Vancouver, British Columbia, Canada (Not_yet_recruiting)
- Sunnybrook Health Sciences Centre — Toronto, Ontario, Canada (Not_yet_recruiting)
Study contacts
- Study coordinator: Umair Qazi, MD, MPH
- Email: umair.qazi@ocugen.com
- Phone: 484-237-3390
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.