RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2024-06-18
Study Completion:2026-10-30
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT06388200

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.

Ages Eligible for Study:8 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. 1. Males or females ≥ 8 years of age
  2. 2. Clinical diagnosis of RP with confirmed genetic diagnosis of autosomal dominant RHO mutation or any other mutation associated with RP.
  3. 3. BCVA ≤ 75 letters and ≥25 letters as measured by an ETDRS chart.
  4. 4. Visual field of \>5° in any meridian as measured by a III4e isopter or equivalent.
  5. 5. Able to perform a Luminance LDNA at certain light intensity at the Screening visit.
  6. 6. Presence of photoreceptors as determined by SD-OCT
  1. 1. Subject lacks evidence of outer nuclear layer
  2. 2. Previous treatment with a gene-therapy or cell therapy product or treatment with any investigational drug or ocular device within one year.
  3. 3. History of corticosteroid related IOP spikes or uncontrolled glaucoma.
  4. 4. Absence of large regions of sensitivity in the pericentral and peripheral retinal regions
  5. 5. Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months.
  6. 6. Active ocular/intraocular infection, any history of rhegmatogenous retinal detachment or Current retinal detachment or retinal implant.
  7. 7. Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception

Contacts and Locations

Study Contact

Umair Qazi, MD, MPH
CONTACT
484-237-3390
umair.qazi@ocugen.com
Sahar Matloob, MD, ACRP-CP
CONTACT
484-237-3390
sahar.matloob@ocugen.com

Principal Investigator

Huma Qamar
STUDY_CHAIR
Ocugen

Study Locations (Sites)

Associated Retina Consultants
Phoenix, Arizona, 85020
United States
Advanced Research, LLC.
Deerfield Beach, Florida, 33064
United States
Bascom Palmer Eye Institute, University of Miami, Miller School of Medicine
Miami, Florida, 33136
United States
Retina Consultants of Texas
Bellaire, Texas, 77401
United States
Retina Foundation of the Southwest
Dallas, Texas, 75231
United States

Collaborators and Investigators

Sponsor: Ocugen

  • Huma Qamar, STUDY_CHAIR, Ocugen

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-06-18
Study Completion Date2026-10-30

Study Record Updates

Study Start Date2024-06-18
Study Completion Date2026-10-30

Terms related to this study

Keywords Provided by Researchers

  • Modified Gene therapy
  • Retinitis Pigmentosa
  • RHO
  • RP
  • NR2E3
  • Subretinal Injection

Additional Relevant MeSH Terms

  • Retinitis Pigmentosa