Study to Assess the Safety & Efficacy of OCU400 for Retinitis Pigmentosa &

Status Recruiting

Clinical Trial 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.

Clinical Trial 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. 3 adult LCA patients with CEP290 mutations and 3 pediatric subjects with RP and LCA, will be enrolled in the Phase 2 portion. Sample Size Justification: The trial will enroll up to 24 patients (18 adult RP, 3 adult LCA, 2 pediatric RP, and 1 pediatric LCA) in both Phase 1 and Phase 2 components. 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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT05203939
Study type	Interventional
Source	Ocugen
Contact	Murthy Chavali, PhD
Phone	405-714-4198
Email	murthy.chavali@ocugen.com
Status	Recruiting
Phase	Phase 1/Phase 2
Start date	January 24, 2022
Completion date	December 2024

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Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

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

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms