NCT06659445

Study to Evaluate Efficacy and Safety of ONL1204 in Patients With GA Associated With AMD

A Phase 2 Multicenter, Randomized, Double-masked, Sham-controlled, Reference-arm Study to Evaluate Efficacy and Safety of ONL1204 in Patients With Geographic Atrophy (GA) Associated With Age-related Macular Degeneration (AMD)

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 324 (estimated)
• Sponsor: ONL Therapeutics · Industry
• Sex: All
• Age: 55 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to collect efficacy and safety information of intravitreal injection for 3 experimental arms, including 2 dose levels of ONL1204 and 2 treatment frequencies in patients with GA associated with AMD. GA associated with AMD is one of the world's leading causes of visual disability and legal blindness globally. Associated with aging, cigarette smoking, obesity, diets low in certain nutrients, a lifestyle related to cardiac risk, and a growing list of genetic factors, AMD is becoming an increasingly prevalent public health concern, especially as the global population ages. ONL1204 is a first-in-class inhibitor of fragment apoptosis stimulator receptor-mediated cell death that has demonstrated protection of multiple retinal cell types in numerous preclinical models of retinal disease, including models of dry AMD.

Conditions

• Geographic Atrophy (GA)
• Age - Related Macular Degeneration (AMD)

Interventions

Type	Name	Description
DRUG	ONL1204 Opthalmic solution	Liquid formulation ONL1204 Ophthalmic Solution administered by intravitreal (IVT) injection
DRUG	Avacincaptad Pegol intravitreal solution	Liquid formulation Avacincaptad Pegol administered by intravitreal (IVT) injection
OTHER	Sham	Sham injection

Timeline

• Start date: 2025-10-28
• Primary completion: 2028-11-01
• Completion: 2028-11-01
• First posted: 2024-10-26
• Last updated: 2025-12-05

Locations

28 sites across 2 countries: United States, Canada

Regulatory

• FDA-regulated drug study