NCT05203939

Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa and Leber Congenital Amaurosis

A Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa Associated With NR2E3 and RHO Mutations and Leber Congenital Amaurosis With Mutation(s) in CEP290 Gene

• Status: Active Not Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 22 (actual)
• Sponsor: Ocugen · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 in patients with retinitis pigmentosa associated with NR2E3 and RHO mutations and in patients with LCA due to mutation(s) in CEP290 gene (OCU400-101). To document prospective eye pathology in the above subjects Investigators will also conduct a Natural History Study (OCU400-104)i This is a multicenter study, which will be conducted in two phases and will enroll up to a total of 24 subjects in the OCU400-101 and 100 subjects in the OCU400-104 study.

Detailed description

This study will be conducted in two phases enrolling up to 24 subjects. Treated subjects will receive a single subretinal injection of OCU400 in the study eye. This is a multicenter, open-label, dose-ranging study in two subgroups of subjects with three consecutive cohorts. A total of 18 adult RP subjects from each of the following subgroups with Biallelic autosomal recessive NR2E3 mutations, autosomal dominant NR2E3 mutations or Autosomal dominant RHO mutations will be selected for dose escalation. For the Phase I portion of the study, the 3+3 design for sequential dose-escalating cohorts will be used with scheduled 3 dosing levels between 9 and 18 subjects will be used to follow the design. Up to 3 additional adult LCA patients with CEP290 mutations and at least 1 pediatric LCA subject, will be enrolled in the Phase 2 portion. Sample Size Justification: The trial will enroll up to 24 patients (18 adult RP, up to 3 LCA patients, and at least 1 pediatric LCA patient) in both Phase 1 and Phase 2 components. Participants who meet eligibility criteria will be enrolled and receive a single subretinal injection of OCU400 in one study eye. Participants are considered to have completed this study if they complete the final EOS visit Week 48 (12 months following the IP dose). The study duration will be approximately 58 weeks for each participant and will be followed in Long Term Safety Follow Up for an additional 2 years. Participants from the Phase 1/2 study who previously received the investigational product (OCU400) in one eye may be eligible to receive OCU400 in the untreated fellow eye, provided they meet the inclusion/exclusion criteria and have completed week 48 follow up visit. Natural History Study (OCU400-104, A Prospective and Retrospective Natural History Study of RP and LCA): This is an observatory study for the prospective natural history of RP and LCA in adult and pediatric subjects. The study will also collect and review retrospective data and ophthalmology examination of natural history and progression of disease for all subjects starting with the earliest timepoint on or after the date of their diagnosis of RP or LCA. Enrollment for this study has closed.

Conditions

• Retinitis Pigmentosa
• Leber Congenital Amaurosis

Interventions

Type	Name	Description
DRUG	OCU400 Low Dose	subretinal injection of up to 1.66×10E10 vg/mL
DRUG	OCU400 Med Dose	subretinal injection of up to 3.33×10E10 vg/mL
DRUG	OCU400 High Dose	subretinal injection of up to 1.66×10E11 vg/mL
DRUG	OCU400 Second Eye Dosing	subretinal injection of 1.0x10E11vg/mL in 250 μl

Timeline

• Start date: 2022-01-24
• Primary completion: 2027-03-01
• Completion: 2027-03-01
• First posted: 2022-01-24
• Last updated: 2025-08-06

Locations

7 sites across 1 country: United States

Regulatory

• FDA-regulated drug study