Find Retinitis Pigmentosa Clinical Trials Near You
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: Active_not_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 140 subjects. Study has completed enrollment of all 140 subjects.
Eligibility
Participation Requirements
Sex: All
Minimum Age: 3
Healthy Volunteers: f
View:
• Males or females ≥ 3 years of age
• Confirmed genetic diagnosis of autosomal dominant RHO mutation with clinical diagnosis of RP
• Clinical Diagnosis of Syndromic or Non-Syndromic RP with/without confirmed genetic diagnosis of any other RP associated mutation (except AD-NR2E3)
• BCVA ≤ 80 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
Phoenix
California
University of Southern Califormia
La Jolla
University of Southern California, Roski Eye Insitute
Los Angeles
Florida
Advanced Research, LLC.
Deerfield Beach
Bascom Palmer Eye Institute, University of Miami, Miller School of Medicine
Miami
Pennsylvania
Erie Retina Research LLC
Erie
Tennessee
Vanderbilt Eye Institute
Nashville
Texas
Retina Consultants of Texas
Bellaire
Retina Foundation of the Southwest
Dallas
Baylor College of Medicine
Houston
Valley Retina Institute
Mcallen
Wisconsin
Gundersen Health System
La Crosse
Other Locations
Canada
Calgary Retina Consultants
Calgary
University of Alberta
Edmonton
Research Institute of the McGill University Health Centre
Montreal
Sunnybrook Health Sciences Centre
Toronto
The University of British Columbia
Vancouver
Time Frame
Start Date:2024-06-18
Completion Date:2027-02-12
Participants
Target number of participants:140
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 10\^10 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 10\^10 vg/eye