A Phase 1b Multicenter, Randomized, Controlled, Multi-dose Study of the Safety and Tolerability of ONL1204 Ophthalmic Solution in Patients With Geographic Atrophy (GA) Associated With Age-related Macular Degeneration (AMD
The purpose of this study is to evaluate the safety and tolerability of intravitreal injection of ONL1204 Ophthalmic Solution in patients with geographic atrophy associated with AMD. GA associated with AMD is one of the world's leading causes of visual disability. It is a progressive disease with no approved therapy to slow or arrest the process of continual photoreceptor and retinal epithelial (RPE) cell loss. A safe and effective therapy for GA will have vast societal benefits. ONL1204 is being developed for this purpose. ONL1204 is a first-in-class inhibitor of fragment apoptosis stimulator receptor-mediated cell death in development for to reduce rates of vision in patients with GA associated with AMD. ONL1204 has demonstrated protection of multiple retinal cell types in several preclinical models of acute ocular injury and the protection of RPE in AMD models. ONL1204 Ophthalmic Solution is currently in a Phase 1 clinical study in patients with macula-off retinal detachment to evaluate safety and tolerability of a multi-dose of ONL1204 Ophthalmic Solution. The study is ongoing and uses the same doses and route of administration as this Phase 1b study in patients with GA.
• Males and females, ≥ 55 years old
• Able to give informed consent and attend study visits
• Bilateral GA secondary to AMD without choroidal neovascularization in either eye
• ETDRS BCVA 20/400 (Snellen equivalent) or better in both eyes
• GA ≥1 disc area (DA) (DA, 2.5 mm2)
• If GA in study eye is multifocal, at least one focal lesion must have a DA of ≥ 1.25 mm2
• Entire GA area must be visible within the standard FAF field of view
• Presence of banded or diffuse hyperautofluoresence adjacent to GA lesion in study eye
• Female subjects must be:
• Women of non-childbearing potential, or
• WOCBP with a negative pregnancy test at screening and willing to use permissible methods of contraception for the duration of the study
• Males with female partners of childbearing potential must agree to use permissible methods of contraception and agree to refrain from donating sperm for the duration of the study.