A Phase 2 Study of ONL1204 in Patients with GA associated with AMD
Research type
Research Study
Full title
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)
IRAS ID
1010725
Contact name
Lindsay Puscas
Contact email
Sponsor organisation
ONL Therapeutics, Inc.
Eudract number
2024-513174-22
ISRCTN Number
Not known
Clinicaltrials.gov Identifier
Research summary
Age-related macular degeneration is a major cause of visual disability and legal blindness globally. This Phase 2 trial aims to assess the effectiveness and safety of the study drug (ONL1204) in slowing the progression of atrophic age-related macular degeneration. ONL1204 could potentially slow or halt the ongoing loss of retinal cells characteristic of this condition, potentially preserving vision in treated individuals. Eligible participants will be randomly assigned to one of five treatment groups; three groups receiving different doses of ONL1204 via injection into the eye and two groups receiving a sham, similar to a placebo (dummy). Depending on the group, participants will receive study treatments every 3 or 6 months. During this time, participants will have regular check-ups to monitor their eye health and any side effects by having their vital signs measured, answering questions about their health and feelings about their vision, having their blood drawn for laboratory assessments, undergoing eye exams and eye imaging, and having their vision checked. All study assessments are standard clinic procedures with the exception of reading speed and questionnaires, both of which pose no risk to the participants. Study visits will take place every 12 weeks to administer study treatment and/or complete safety assessments. Study participation is expected to last 72 weeks. The study will involve about 324 participants from the United States (US), Canada and Europe.
REC name
North West - Haydock Research Ethics Committee
REC reference
25/NW/0142
Date of REC Opinion
19 Jun 2025
REC opinion
Further Information Favourable Opinion