Description Module
The Description Module contains narrative descriptions of the clinical trial, including a brief summary and detailed description. These descriptions provide important information about the study's purpose, methodology, and key details in language accessible to both researchers and the general public.
Description Module path is as follows:
Study -> Protocol Section -> Description Module
Description Module
Ignite Creation Date:
2025-12-25 @ 12:19 AM
Ignite Modification Date:
2025-12-25 @ 12:19 AM
NCT ID:
NCT06018558
Brief Summary:
This is a Phase 1/2 Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration (AMD).
This is a multicenter study, which will be conducted in two phases and will enroll up to a total of 60 subjects.
Detailed Description:
Name of Sponsor/Company:
Ocugen, Inc. 11 Great Valley Parkway Malvern, PA 19355
Name of Investigational Product: OCU410
Name of Active Ingredient:
Adeno-associated viral vector 5 human RORA (AAV5-hRORA) Protocol Number: OCU410-101 Phase: 1/2 Country: US
Title of Study:
A Phase 1/2 Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration.
Study Center(s): Approximately 14 clinical study centers in the US.
Background:
Age-related Macular Degeneration (AMD) is an ocular disease where macular degenerative occurs. AMD manifests in two forms, Dry (nonexudative, atrophic) AMD and Wet (exudative, neovascular) AMD. Geographic atrophy (GA) is an advanced stage of dry AMD that affects nearly 1 million people in the US and 5 million people worldwide, with its prevalence increasing exponentially with age. It leads to progressive and irreversible loss of visual function due to the growth of atrophic lesions that destroy the retinal cells responsible for vision.
OCU410 Product Information:
Ocugen, Inc., has developed a proprietary modifier gene therapy platform, OCU410, as the second agent in a novel class of NHR-based gene modifier therapy for patients with dry AMD. The proposed indication for OCU410 (AAV5-hRORA) is for the treatment of GA secondary to dry AMD. The drug product is a sterile ophthalmic suspension for subretinal injection. OCU410 therapy regulates gene pathways contributing to GA by restoring homeostasis in the eye and thereby serving as a therapeutic candidate for dry AMD. The modifier gene therapy platform is a new way of addressing a genetic disease arising through a multitude of genetic mutations in various genes but leading to the same end result (phenotype) of a diseased condition.
This study will be conducted in two phases enrolling up to 60 subjects. Treated subjects will receive a single subretinal injection of OCU410 in the study eye.
Phase 1 is a multicenter, open-label, dose-ranging/dose-escalating study with a 3+3 design enrolling 9 subjects.
Phase 2 is a dose-expansion phase of the study, where up to 51 subjects will be randomized in 1:1:1 ratio to either two OCU410 dose groups (n=17 per group) or to an untreated control group (n=17).
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Study:
NCT06018558
Study Brief:
Protocol Section:
NCT06018558