Sub-Retinal Administration of OCU400-301: Sub-Retinal Administration of OCU400-301
Study 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
Sex
ALL
Min age
3 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
1. Males or females ≥ 3 years of age
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:
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 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
Primary outcome measure(s)
Change in functional vision from baseline to week 52 in pooled analysis when study eyes in treatment group were compared to study eyes in untreated control — 52 weeks Change in functional vision from baseline to week 52 in pooled analysis from retinitis pigmentosa subjects when study eyes in treatment group were compared to study eyes in untreated control, as measured by the ability of a study participant to navigate through a maze in Luminance Dependent Navigation Assessment (LDNA)
Trial sites (17)
Facility
City
Region
Status
Associated Retina Consultants
Phoenix
Arizona
University of Southern Califormia
La Jolla
California
University of Southern California, Roski Eye Insitute
Los Angeles
California
Advanced Research, LLC.
Deerfield Beach
Florida
Bascom Palmer Eye Institute, University of Miami, Miller School of Medicine
Miami
Florida
Erie Retina Research LLC
Erie
Pennsylvania
Vanderbilt Eye Institute
Nashville
Tennessee
Retina Consultants of Texas
Bellaire
Texas
Retina Foundation of the Southwest
Dallas
Texas
Baylor College of Medicine
Houston
Texas
Valley Retina Institute
McAllen
Texas
Gundersen Health System
La Crosse
Wisconsin
Calgary Retina Consultants
Calgary
Alberta
University of Alberta
Edmonton
Alberta
The University of British Columbia
Vancouver
British Columbia
Sunnybrook Health Sciences Centre
Toronto
Ontario
Research Institute of the McGill University Health Centre
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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