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

NCT05203939 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 22

Last updated 2025-08-06

No results posted yet for this study

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.

Conditions

Interventions

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

Sponsors & Collaborators

Principal Investigators

  • Huma Qamar, MD, MPH, CMI · Ocugen

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
6 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2022-01-24
Primary Completion
2027-03-31
Completion
2027-03-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Diseases
Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05203939 on ClinicalTrials.gov