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

Last updated: March 17, 2025
Sponsor: Ocugen
Overall Status: Active - Recruiting

Phase

1/2

Condition

Geographic Atrophy

Treatment

OCU410

Clinical Study ID

NCT06018558
OCU410-101
  • Ages > 50
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Subjects 50 years of age or older.

  2. BCVA of approximately 21 letters or more using Early Treatment Diabetic RetinopathyStudy (ETDRS) chart (20/320 Snellen equivalent).

  3. Fundus autofluorescence (FAF) imaging shows:

  4. Total GA area ≥2.0 and ≤20.5 mm2 (1 and 8 disk areas [DA], respectively)

  5. If GA is multifocal, at least one focal lesion must be ≥1.25 mm2 (0.5 DA), withthe overall aggregate area of GA as specified above in 3.a

  6. The entire GA lesion must be completely visualized on the macula-centered imageand must be able to be imaged in its entirety, and not contiguous with anyareas of peripapillary atrophy

  7. Presence of any pattern of hyper-autofluorescence in the junctional zone of GA

  8. Subjects who had prior treatment with an approved drug for AMD, e.g. Izerway® (Avacincaptad pegol) or Syfovre® (Pegcetacoplan injection) can be included, after awashout period of at least 3 months in study eye or fellow eye

Exclusion

Exclusion Criteria:

  1. Previous treatment with a gene-therapy or cell therapy product

  2. GA due to causes other than AMD such as Stargardt disease, cone rod dystrophy ortoxic maculopathies like Plaquenil maculopathy. However, benign conditions of thevitreous or peripheral retina are not exclusionary (i.e., pavingstone degeneration).

  3. Spherical equivalent of the refractive error demonstrating > 6 diopters of myopia oran axial length >26 mm, inability to fixate, uncontrolled glaucoma, advancedcataract, corneal abnormalities, medium haze, and other retinal pathologies.

  4. Any history or current evidence of exudative ("wet") AMD including any evidence ofretinal pigment epithelium rips, branch retinal artery or vein occlusion, cornealtransplant, or evidence of neovascularization anywhere in the retina based onfluorescein angiogram.

Study Design

Total Participants: 63
Treatment Group(s): 1
Primary Treatment: OCU410
Phase: 1/2
Study Start date:
August 23, 2023
Estimated Completion Date:
September 23, 2025

Study 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.

Connect with a study center

  • Associated Retina Consultants

    Phoenix, Arizona 85020
    United States

    Active - Recruiting

  • Miidwest Eye Institute

    Carmel, Indiana 46290
    United States

    Active - Recruiting

  • Mississippi Retina Associates

    Jackson, Mississippi 39202
    United States

    Active - Recruiting

  • Mid Atlantic Retina

    Cherry Hill, New Jersey 08034
    United States

    Active - Recruiting

  • Duke Eye Center

    Durham, North Carolina 27710
    United States

    Active - Recruiting

  • The University of Pittsburgh

    Pittsburgh, Pennsylvania 15260
    United States

    Active - Recruiting

  • B) Retina Consultants of Texas

    Bellaire, Texas 77401
    United States

    Active - Recruiting

  • Retina Consultants of Texas

    Bellaire, Texas 77401
    United States

    Active - Recruiting

  • A) Retina Foundation of the Southwest

    Dallas, Texas 75231
    United States

    Active - Recruiting

  • Retina Foundation of the Southwest

    Dallas, Texas 75231
    United States

    Active - Recruiting

  • Gundersen Health System

    La Crosse, Wisconsin 54601
    United States

    Active - Recruiting

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