A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa
NCT ID: NCT06388200
Last Updated: 2026-01-08
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
150 participants
INTERVENTIONAL
2024-06-18
2026-12-31
Brief Summary
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This is a multicenter, assessor blinded and randomized study which will enroll 150 subjects.
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Detailed Description
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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.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
SINGLE
Study Groups
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RHO Arm
Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10\^10 vg/eye
Sub-Retinal Administration of OCU400-301
Sub-Retinal Administration of OCU400-301
Gene Agnostic Arm
Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10\^10 vg/eye
Sub-Retinal Administration of OCU400-301
Sub-Retinal Administration of OCU400-301
Control for RHO Arm
Will not receive any active study intervention
No interventions assigned to this group
Control for Gene Agnostic Arm
Will not receive any active study intervention
No interventions assigned to this group
Interventions
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Sub-Retinal Administration of OCU400-301
Sub-Retinal Administration of OCU400-301
Eligibility Criteria
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Inclusion Criteria
2. Confirmed genetic diagnosis of autosomal dominant RHO mutation with clinical diagnosis of RP
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 ≤ 80 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
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
3 Years
ALL
No
Sponsors
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Ocugen
INDUSTRY
Responsible Party
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Principal Investigators
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Huma Qamar
Role: STUDY_CHAIR
Ocugen
Locations
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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
Baylor College of Medicine
Houston, Texas, United States
Valley Retina Institute
McAllen, Texas, United States
Gundersen Health System
La Crosse, Wisconsin, 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
Research Institute of the McGill University Health Centre
Montreal, Quebec, Canada
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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OCU400-301
Identifier Type: -
Identifier Source: org_study_id
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