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

Running, not enrolling · Phase 3

Conditions studied: Retinitis Pigmentosa

In brief

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.

Key facts

Study ID

NCT06388200

Run by

Ocugen

People needed

140

Starts

2024-06-18

Expected to finish

2027-02-12

Last updated by the study team

2026-07-31

Who can join

Age: 3 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• Subject lacks evidence of outer nuclear layer
• Previous treatment with a gene-therapy or cell therapy product or treatment with any investigational drug or ocular device within one year.
• History of any corticosteroid contraindication, corticosteroid related IOP spikes or uncontrolled glaucoma.
• Cataract surgery within 3 months. YAG capsulotomy within 1 month. Any other intraocular surgery within 6 months.
• Active ocular/intraocular infection, any history of rhegmatogenous retinal detachment or Current retinal detachment or retinal implant.
• Breast-feeding, pregnancy, sperm donation or inability to practice strict contraception

Where it is running

• Associated Retina Consultants — Phoenix, Arizona, United States
• University of Southern Califormia — La Jolla, California, United States
• University of Southern California, Roski Eye Insitute — Los Angeles, California, United States
• Advanced Research, LLC. — Deerfield Beach, Florida, United States
• Bascom Palmer Eye Institute, University of Miami, Miller School of Medicine — Miami, Florida, United States
• Erie Retina Research LLC — Erie, Pennsylvania, United States
• Vanderbilt Eye Institute — Nashville, Tennessee, United States
• Retina Consultants of Texas — Bellaire, Texas, United States
• Retina Foundation of the Southwest — Dallas, Texas, United States
• Baylor College of Medicine — Houston, Texas, United States
• Valley Retina Institute — McAllen, Texas, United States
• Gundersen Health System — La Crosse, Wisconsin, United States
• Calgary Retina Consultants — Calgary, Alberta, Canada
• University of Alberta — Edmonton, Alberta, Canada
• The University of British Columbia — Vancouver, British Columbia, Canada
• Sunnybrook Health Sciences Centre — Toronto, Ontario, Canada
• Research Institute of the McGill University Health Centre — Montreal, Quebec, Canada

Full record on ClinicalTrials.gov

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