A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa

Phase 3

Recruiting

• Conditions: Retinitis Pigmentosa

• 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.

Study Timeline

• Study First Submitted: 2024-04-09
• Study First Submitted QC: 2024-04-24
• Study First Posted: 2024-04-29
• Study Start: 2024-06-18
• Status Verified: 2025-07
• Last Update Submitted: 2025-07-31
• Last Update Posted: 2025-08-03
• Primary Completion: 2026-12-31
• Study Completion: 2026-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 150

Inclusion Criteria

1. Males or females ≥ 5 years of age
2. Clinical diagnosis of RP with confirmed genetic diagnosis of autosomal dominant RHO mutation
3. Clinical Diagnosis of Syndromic or Non-Syndromic RP with/without confirmed genetic diagnosis of any other RP associated mutation (except AD-NR2E3)
4. BCVA ≤ 75 letters and ≥25 letters as measured by an ETDRS chart
5. Visual field of >5° in any meridian as measured by a III4e isopter or equivalent
6. Able to perform a Luminance LDNA at certain light intensity at the Screening visit
7. 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 any corticosteroid contraindication, corticosteroid related IOP spikes or uncontrolled glaucoma.
4. Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months.
5. Active ocular/intraocular infection, any history of rhegmatogenous retinal detachment or Current retinal detachment or retinal implant.
6. Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

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 (17)

Research Institute of the McGill University Health Centre; 🇨🇦 Montréal, Quebec, Canada

Associated Retina Consultants; 🇺🇸 Phoenix, Arizona, United States

University of Southern Califormia; 🇺🇸 La Jolla, California, United States

University of Southern California, Roski Eye Insitute; 🇺🇸 Los Angeles, California, United States

Advanced Research, LLC.; 🇺🇸 Deerfield Beach, Florida, United States

Bascom Palmer Eye Institute, University of Miami, Miller School of Medicine; 🇺🇸 Miami, Florida, United States

Erie Retina Research LLC; 🇺🇸 Erie, Pennsylvania, United States

Vanderbilt Eye Institute; 🇺🇸 Nashville, Tennessee, United States

Retina Consultants of Texas; 🇺🇸 Bellaire, Texas, United States

Retina Foundation of the Southwest; 🇺🇸 Dallas, Texas, United States

Scroll for more (7 remaining)