Study to Assess the Safety and Efficacy of OCU410 for Geographic Atrophy — ArMaDa  

Geographic Atrophy Clinical Trial

Official title: A Phase 1/2 Study to Assess the Safety And Efficacy Of OCU410 For Geographic Atrophy Secondary To Dry Age-Related Macular Degeneration

Status Recruiting

Clinical Trial 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 63 subjects.

Clinical Trial 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 five 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 63 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 up to 18 subjects.

Phase 2 is a randomized dose-expansion cohort in which 45 subjects will be randomized in a 1:1:1 ratio in to one of the 2 treatment arms or the untreated control arm. ;

Related Conditions & MeSH terms

• Atrophy
• Geographic Atrophy

• NCT number: NCT06018558
• Study type: Interventional
• Source: Ocugen
• Contact: Umair Qazi, MD, MPH
• Phone: 202 817 0787
• Email: umair.qazi@ocugen.com
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: August 23, 2023
• Completion date: September 23, 2025