OCU400
Study Overview
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.
Study 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
Medical Condition(s)
Retinal degeneration resulting from genetic mutations NR2E3, RHO
Product
AAV-NR2E3
Received orphan drug designation
OCUGEN PROTOCOL NUMBER
OCU400-301
NUMBER OF SUBJECTS
150
STUDY LENGTH
30 months
SEX
Male & Female
STUDY DATES
April 2024 – October 2026
AGE
18+
PHASE
3
COUNTRY
United States