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
Status: Recruiting
Location: See all (17) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 3
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.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 5
Healthy Volunteers: f
View:
• Males or females ≥ 5 years of age
• Clinical diagnosis of RP with confirmed genetic diagnosis of autosomal dominant RHO mutation
• Clinical Diagnosis of Syndromic or Non-Syndromic RP with/without confirmed genetic diagnosis of any other RP associated mutation (except AD-NR2E3)
• 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
Locations
United States
Arizona
Associated Retina Consultants
RECRUITING
Phoenix
California
University of Southern Califormia
RECRUITING
La Jolla
University of Southern California, Roski Eye Insitute
RECRUITING
Los Angeles
Florida
Advanced Research, LLC.
RECRUITING
Deerfield Beach
Bascom Palmer Eye Institute, University of Miami, Miller School of Medicine
RECRUITING
Miami
Pennsylvania
Erie Retina Research LLC
RECRUITING
Erie
Tennessee
Vanderbilt Eye Institute
RECRUITING
Nashville
Texas
Retina Consultants of Texas
RECRUITING
Bellaire
Retina Foundation of the Southwest
RECRUITING
Dallas
Baylor College of Medicine
NOT_YET_RECRUITING
Houston
Valley Retina Institute
RECRUITING
Mcallen
Wisconsin
Gundersen Health System
RECRUITING
La Crosse
Other Locations
Canada
Calgary Retina Consultants
RECRUITING
Calgary
University of Alberta
RECRUITING
Edmonton
Research Institute of the McGill University Health Centre
NOT_YET_RECRUITING
Montreal
Sunnybrook Health Sciences Centre
RECRUITING
Toronto
The University of British Columbia
RECRUITING
Vancouver
Contact Information
Primary
Umair Qazi, MD, MPH
umair.qazi@ocugen.com
484-237-3390
Backup
Sahar Matloob, MD, ACRP-CP
sahar.matloob@ocugen.com
484-237-3390
Time Frame
Start Date:2024-06-18
Estimated Completion Date:2026-12-31
Participants
Target number of participants:150
Treatments
Experimental: RHO Arm
Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10E10 vg/eye
Experimental: Gene Agnostic Arm
Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10E10 vg/eye