A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa
- Conditions
- Retinitis Pigmentosa
- Interventions
- Genetic: Sub-Retinal Administration of OCU400-301
- Registration Number
- NCT06388200
- Lead Sponsor
- Ocugen
- Brief Summary
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 150 subjects.
- Detailed Description
A total of one hundred and fifty (150) RP participants will be enrolled in this study- 75 in each of either two arms, RHO arm (n=75) or Gene agnostic arm (n=75). RHO arm will only enroll participants with confirmed genetic diagnosis of mutation in RHO gene; whereas Gene Agnostic arm will enroll RP Participants based on clinical diagnosis of RP and a confirmed genetic diagnosis with a gene associated with RP.
Subjects in each arm will be randomized into treatment (N=50) and control groups (N=25). Subjects in the treatment group will receive a sequential, bilateral sub-retinal injection of OCU400 if both eyes meet inclusion criteria. Control or untreated group subjects will receive OCU400 subretinal injection after completion of 12-month follow-up.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 150
- Males or females ≥ 8 years of age
- Clinical diagnosis of RP with confirmed genetic diagnosis of autosomal dominant RHO mutation or any other mutation associated with RP.
- BCVA ≤ 75 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
- Subject lacks evidence of outer nuclear layer
- Previous treatment with a gene-therapy or cell therapy product or treatment with any investigational drug or ocular device within one year.
- History of corticosteroid related IOP spikes or uncontrolled glaucoma.
- Absence of large regions of sensitivity in the pericentral and peripheral retinal regions
- Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months.
- Active ocular/intraocular infection, any history of rhegmatogenous retinal detachment or Current retinal detachment or retinal implant.
- Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description RHO Arm Sub-Retinal Administration of OCU400-301 Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10E10 vg/eye Gene Agnostic Arm Sub-Retinal Administration of OCU400-301 Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10E10 vg/eye
- Primary Outcome Measures
Name Time Method Number of participants with change in functional vision in the study eye of RP subjects as assessed by LDNA 52 weeks Change in the LDNA assessment from screening in the study eye of RP subjects with RHO mutation will be compared to controls
Number of participants with change in functional vision in the study eye in Gene Agnostic group as assessed by LDNA 52 weeks Change in the LDNA assessment from screening in the study eye in Gene Agnostic Arm subjects will be compared to controls
- Secondary Outcome Measures
Name Time Method Number of participants with change in functional vision in the all treated eyes of subjects in Gene Agnostic Arm as assessed by LDNA 52 weeks Change in the LDNA assessment from screening in all treated eyes of subjects in Gene Agnostic Arm subjects will be compared to controls
Number of participants with change in visual function in Gene Agnostic Arm as assessed by LLVA letter scores 52 weeks Change in LLVA letter scores in Gene Agnostic Arm subjects will be compared to controls
Number of participants with change in visual function in subjects with RHO mutation as assessed by LLVA letter score 52 weeks Change in LLVA letter scores in RP subjects with mutations in RHO will be compared to controls
Ocular and non-ocular Events 52 weeks Incidence and severity of Study Drug-related adverse events (SDAE), Treatment-Emergent adverse events (TEAEs), Serious adverse events (SAEs) and Adverse Events of Special Interest (AESI).
Number of participants with change in functional vision in the all treated eyes of RP subjects as assessed by LDNA 52 weeks Change in the LDNA assessment from screening in all treated eyes of RP subjects with RHO mutation will be compared to controls
Trial Locations
- Locations (15)
Advanced Research, LLC.
🇺🇸Deerfield Beach, Florida, United States
Bascom Palmer Eye Institute, University of Miami, Miller School of Medicine
🇺🇸Miami, Florida, United States
Calgary Retina Consultants
🇨🇦Calgary, Alberta, Canada
University of Alberta
🇨🇦Edmonton, Alberta, Canada
The University of British Columbia
🇨🇦Vancouver, British Columbia, Canada
Sunnybrook Health Sciences Centre
🇨🇦Toronto, Ontario, Canada
Erie Retina Research LLC
🇺🇸Erie, Pennsylvania, United States
Gundersen Health System
🇺🇸La Crosse, Wisconsin, United States
University of Southern Califormia
🇺🇸La Jolla, California, United States
Retina Foundation of the Southwest
🇺🇸Dallas, Texas, United States
Valley Retina Institute
🇺🇸McAllen, Texas, United States
Associated Retina Consultants
🇺🇸Phoenix, Arizona, United States
University of Southern California, Roski Eye Insitute
🇺🇸Los Angeles, California, United States
Vanderbilt Eye Institute
🇺🇸Nashville, Tennessee, United States
Retina Consultants of Texas
🇺🇸Bellaire, Texas, United States