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Gene Therapy Trial Report

Summary

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


NCTID NCT06018558 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Geographic Atrophy
Disease Ontology Term DOID:10871
Compound Name OCU410
Sponsor Ocugen
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 60 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant RORA
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Subretinal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV5
Editor Type none
Dose 1 2.5E10 vg/mL
Dose 2 5E10 vg/mL
Dose 3 1.5E11 vg/mL

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2023-06-30
Completion Date 2026-05-29
Last Update 2025-12-05

Participation Criteria


Eligible Age >=50 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. Subjects 50 years of age or older. 2. BCVA of approximately 21 letters or more using Early Treatment Diabetic Retinopathy Study (ETDRS) chart (20/320 Snellen equivalent). 3. Fundus autofluorescence (FAF) imaging shows: 1. Total GA area ≥2.0 and ≤20.5 mm2 (1 and 8 disk areas \[DA\], respectively) 2. If GA is multifocal, at least one focal lesion must be ≥1.25 mm2 (0.5 DA), with the overall aggregate area of GA as specified above in 3.a 3. The entire GA lesion must be completely visualized on the macula-centered image and must be able to be imaged in its entirety, and not contiguous with any areas of peripapillary atrophy 4. Presence of any pattern of hyper-autofluorescence in the junctional zone of GA 4. Subjects who had prior treatment with an approved drug for AMD, e.g. Izerway® (Avacincaptad pegol) or Syfovre® (Pegcetacoplan injection) can be included, after a washout period of at least 3 months in study eye. Subjects can receive an approved drug for AMD in the fellow eye, if required. 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 or toxic maculopathies like Plaquenil maculopathy. However, benign conditions of the vitreous or peripheral retina are not exclusionary (i.e., pavingstone degeneration). 3. Spherical equivalent of the refractive error demonstrating \> 6 diopters of myopia or an axial length \>26 mm, inability to fixate, uncontrolled glaucoma, advanced cataract, corneal abnormalities, medium haze, and other retinal pathologies. 4. Any history or current evidence of exudative ("wet") AMD including any evidence of retinal pigment epithelium rips, branch retinal artery or vein occlusion, corneal transplant, or evidence of neovascularization anywhere in the retina based on fluorescein angiogram.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 12
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates OCU410 positive preliminary Phase 2 data announced in January. Full Phase 2 data expected in March 2026

Resources/Links