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)

ONL Therapeutics23 sites in 2 countries324 target enrollmentOctober 28, 2025

ConditionsGeographic Atrophy (GA)Age - Related Macular Degeneration (AMD)

InterventionsONL1204 Opthalmic solution Sham Avacincaptad Pegol intravitreal solution

DrugsONL1204 Opthalmic solution Avacincaptad Pegol intravitreal solution

Overview

Phase: Phase 2
Intervention: ONL1204 Opthalmic solution
Conditions: Geographic Atrophy (GA)
Sponsor: ONL Therapeutics
Enrollment: 324
Locations: 23
Primary Endpoint: Primary Endpoint
Status: Recruiting
Last Updated: 4 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials Related News

Overview

Brief 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.

Registry

clinicaltrials.gov

Start Date

October 28, 2025

End Date

November 1, 2028

Last Updated

4 months ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

ONL Therapeutics

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•55 years of age or older at Screening.
•Able and willing to give informed consent and attend study visits.
•Women or intersex individuals must be willing to use 2 forms of effective contraception during the study and for at least 90 days following the last dose of study drug, be postmenopausal for at least 12 months prior to study entry, or surgically sterile. If of childbearing potential, a negative urine pregnancy test is required at Screening and prior to the administration of study drug at each visit.
•Men or intersex individuals with partners of childbearing potential must be willing to use permissible methods of contraception and refrain from sperm donation during the study and for at least 90 days following the last dose of study drug.
•If currently using Age-related Eye Disease Study 2 (AREDS, AREDS2, or similar nutraceutical therapy at Screening, patient must be willing to continue use for the duration of the study. If not currently using AREDS2 or similar, patient must be willing to continue not to use therapy for the duration of the study. Patient must agree to choose either approach.
•Study Eye Inclusion Criteria:
•BCVA of 24 letters or more using ETDRS charts in the study eye at Screening.
•Intraocular pressure (IOP) of 22 mmHg or lower in the study eye at Screening.
•The GA lesion must meet all the following criteria:
•Non-foveal GA associated with AMD without eMNV in the study eye per Investigator's evaluation and as confirmed by the central RC.

Exclusion Criteria

•Current or planned participation in another investigational clinical study or use of any other investigational drugs or devices at least 6 months prior to enrollment or during the study period without prior written Sponsor approval.
•Previous ophthalmic disease gene therapy or planned participation in any gene therapy clinical study during the study period.
•Current or planned use of systemic complement inhibitors during the study period.
•Any ocular or systemic condition that, in the opinion of the Investigator, makes the patient unsuitable for treatment with an investigational drug or that would compromise the safety or efficacy assessments of the study.
•Treatment with any ocular or systemic medication that is known to be toxic to the lens, retina, or optic nerve (including, but not limited to, aminoglycosides, vancomycin, hydroxychloroquine, interferon, tacrolimus, cisplatin, bis-chloroethyl nitrosourea, carmustine, ethambutol, and tamoxifen) within 90 days prior to Screening or anticipated during the study period.
•Any previous treatment of pentosan polysulfate (Elmiron®) is exclusionary.
•Known allergy to fluorescein, povidone iodine, or any ingredients of the study drug or avacincaptad pegol intravitreal solution.
•Individuals who are currently pregnant, planning to become pregnant, or are nursing at Screening or during the study period.
•Study Eye Exclusion Criteria:
•Active ocular or periocular infection in the study eye.