NCT06388200

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

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
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 140 (actual)
• Sponsor: Ocugen · Industry
• Sex: All
• Age: 3 Years
• Healthy volunteers: Not accepted

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.

Detailed description

A total of one hundred and forty (140) RP participants will be enrolled in this study into RHO arm or Gene agnostic arm. RHO arm will only enroll participants with confirmed genetic diagnosis of mutation in RHO gene; whereas Gene Agnostic arm will enroll RP Participants based on clinical diagnosis of RP and a confirmed genetic diagnosis with a gene associated with RP. Subjects in each arm will be randomized into treatment and control groups with a 2:1 ratio. Subjects in the treatment group will receive a sequential, bilateral sub-retinal injection of OCU400 if both eyes meet inclusion criteria. Control or untreated group subjects will receive OCU400 subretinal injection after completion of 12-month follow-up.

Conditions

• Retinitis Pigmentosa

Interventions

Type	Name	Description
GENETIC	Sub-Retinal Administration of OCU400-301	Sub-Retinal Administration of OCU400-301

Timeline

• Start date: 2024-06-18
• Primary completion: 2027-02-12
• Completion: 2027-02-12
• First posted: 2024-04-29
• Last updated: 2026-03-05

Locations

17 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study