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

Description

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.

Conditions

Retinitis Pigmentosa

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Study Overview

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Study Details

Study overview

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.

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

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

Condition
Retinitis Pigmentosa
Intervention / Treatment

-

Contacts and Locations

Phoenix

Associated Retina Consultants, Phoenix, Arizona, United States, 85020

Deerfield Beach

Advanced Research, LLC., Deerfield Beach, Florida, United States, 33064

Miami

Bascom Palmer Eye Institute, University of Miami, Miller School of Medicine, Miami, Florida, United States, 33136

Bellaire

Retina Consultants of Texas, Bellaire, Texas, United States, 77401

Dallas

Retina Foundation of the Southwest, Dallas, Texas, United States, 75231

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • 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

Ages Eligible for Study

8 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Ocugen,

Huma Qamar, STUDY_CHAIR, Ocugen

Study Record Dates

2026-10-30