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Gene Therapy Trial Report

Summary

Phase 3 Study to Assess Efficacy, Safety & Tolerability of OCU410 For Geographic Atrophy Secondary to Dry AMD


NCTID NCT07770828 (View at clinicaltrials.gov)
Description
Development Status 🔄 Active
Indication Dry Age-related Macular Degeneration
Disease Ontology Term 🔄 DOID:10871
Drug Product Name 🔄 OCU410
Sponsor Ocugen
Funder Type Industry
Recruitment Status
Enrollment Count 237 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant 🔄 RORA
Therapeutic Modality 🔄 Gene transfer
Therapy Route 🔄 in vivo
Mechanism of Action 🔄 Overexpression of protective allele/gene
Route of Administration 🔄 Subretinal
Drug Product Type 🔄 Viral vector
Gene Delivery System Type 🔄 Viral transduction
Vector Type 🔄 Adeno-associated virus
Viral Vector Subtype AAV5
Dose 1 🔄 Phase 1/2 study: 5E9 vg/eye, 1E10 vg/eye, 3E10 vg/eye (200ul injection volume for each)
Dose 2 🔄 Phase 3 study: 1E10 vg/eye (200ul injection volume)

Study Record Dates


Current Phase Phase3
Submit Date 2026-08-13
Completion Date 2031-07-30
Last Update 2026-08-19

Participation Criteria


Eligible Age >=55 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1. Subjects 55 years of age or older at consent. 2. BCVA of ≥25 ETDRS letters or more using Early Treatment Diabetic Retinopathy Study (ETDRS) chart (20/320 Snellen Equivalent) and ≤80 ETDRS Letters (20/25 Snellen Equivalent) in the study eye. 3. Fundus autofluorescence (FAF) imaging shows: a)Total GA area ≥2.5 and ≤ 17.5 mm2 (1 to 7 disk areas \[DA\], respectively); b)If GA is multifocal, at least one lesion must be ≥1.25 mm2 (0.5 DA), with the overall aggregate area of GA as specified above in 3a. c)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. Subjects with history of CNV diagnosis (inactive) in the fellow eye for ≥2 years prior to Screening; stable and treated CNV treatment prior to screening (stable and inactive); no active exudation at screening (confirmed by imaging at the discretion of the investigator) 5. Subjects who had prior treatment with an approved drug for AMD, e.g. Izervay® (avacincaptad pegol) or Syfovre® (pegcetacoplan injection) can be included, after a washout period of at least 3 months in study eye from screening visit. 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., paving stone 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 in the study eye 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. Subjects with active exudative CNV in the fellow eye will be exclude. 5. Presence of double-layer sign on SD-OCT, suggestive of subclinical macular neovascularization.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 3
Locations United States

Regulatory Information


Has US IND True
FDA Designations 🔄 Regenerative Medicine Advanced Therapy (RMAT)
Recent Updates Topline Phase 2 data announced in March 2026, Ocugen announce in September 2026 that the first patient in the registrational trial had been dosed

Resources/Links