OCU400
Study Overview
The Phase 1/2 clinical study to evaluate the safety and efficacy of OCU400 for Retinitis Pigmentosa associated with NR2E3 and RHO mutations, utilizing unilateral sub-retinal injection (one eye per study subject) in up to 18 patients, with an extension for contralateral eye and long-term safety follow up.
Study title
A Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa associated with NR2E3 and RHO mutations
Medical Condition(s)
Retinal degeneration resulting from genetic mutations NR2E3, RHO
Product
AAV-NR2E3
Received orphan drug designation
OCUGEN PROTOCOL NUMBER
OCU400-101
NUMBER OF SUBJECTS
Up to 18 adults who meet eligibility criteria
STUDY LENGTH
12 months
SEX
Male & Female
STUDY DATES
January 2022 – April 2023
AGE
18+
PHASE
1/2
COUNTRY
United States