Summary
This study is a Phase 3 multicenter, randomized, controlled, assessor blinded, adaptive study in which subjects will be randomized in a 2:1(OCU410: control) ratio to treatment arm or control arm
Detailed Description
This study is a Phase 3 multicenter, randomized, controlled, assessor blinded, adaptive study in which subjects will be randomized in a 2:1(OCU410: control) ratio to either of the following arms:
Treatment arm (OCU410) study subjects will receive single subretinal injection of 200 µL, OCU410 in the study eye with the optimal dose (N=158)
Control arm (untreated) (N=79)
A total of 237 subjects will be enrolled in the study. Subjects who are enrolled in the untreated control group of the study will not receive any treatment. Subjects randomized to the treatment arm may receive a single subretinal injection of OCU410 in the study eye, provided they meet all treatment eligibility criteria. Subjects who are randomized to the control arm will not receive any intervention during the initial 52 weeks (12 Months) of the study.
Eligibility Criteria
Age Range: 55 years to No maximum
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.