ONL1204 for Age-Related Macular Degeneration

Recruiting at 9 trial locations

Overseen By Study Coordinator

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: ONL Therapeutics

Must not be taking: Systemic complement inhibitors, toxic medications

Disqualifiers: Pregnancy, Gene therapy, Active infection, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 12

Duration: 48 weeks

324 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

The trial aims to test a new treatment called ONL1204 for individuals with age-related macular degeneration (AMD), a common cause of vision loss. The study will compare different doses and schedules of ONL1204 injections to evaluate their effectiveness and safety. Individuals with non-foveal geographic atrophy, a type of vision loss that does not affect the very center of sight, linked to AMD might be suitable candidates for this trial. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important advancements in AMD treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot use medications known to be toxic to the eye within 90 days before the trial or during it. If you are using Age-related Eye Disease Study (AREDS) supplements, you must continue or not use them consistently throughout the study.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that ONL1204 may protect eye cells in various early-stage studies of eye diseases. In previous human studies, participants generally tolerated ONL1204 well, with most side effects being mild and temporary. Common reactions included irritation at the injection site or mild discomfort. Importantly, no serious side effects directly related to the treatment have been reported so far.

This is a Phase 2 trial, indicating that the treatment has already undergone basic safety testing in humans. This trial will provide more detailed information on safety and any side effects. Although the FDA has not yet approved ONL1204 for any condition, early results suggest it has a promising safety profile. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Unlike the standard treatments for age-related macular degeneration, which often include anti-VEGF injections like ranibizumab or aflibercept, ONL1204 offers a unique approach. Researchers are excited about ONL1204 because it targets photoreceptor protection, aiming to prevent cell death directly, rather than just inhibiting blood vessel growth. This new mechanism could potentially provide a complementary benefit by preserving vision longer and improving outcomes for patients. Additionally, ONL1204 is administered less frequently, either every 12 or 24 weeks, which may improve patient convenience and adherence compared to the more frequent dosing schedules of current treatments.

What evidence suggests that this trial's treatments could be effective for age-related macular degeneration?

Research has shown that ONL1204 could help treat geographic atrophy (GA), a condition related to age-related macular degeneration (AMD). Early lab studies demonstrated that ONL1204 protected various eye cells by blocking pathways leading to cell death. This suggests it might slow the damage causing vision loss. Initial results also indicate that ONL1204 could slow the growth of retinal lesions, potentially helping people with GA maintain their vision longer. However, further research with human participants is necessary to confirm these benefits. Participants in this trial may receive ONL1204 in one of several treatment groups or a comparator treatment. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

This trial is for individuals aged 55 or older with a specific eye condition called GA associated with AMD. Participants must have a certain level of vision, be willing to use or avoid specific nutritional supplements, and agree to contraception if applicable. They should not have any other major eye conditions that could interfere with the study.

Inclusion Criteria

My vision in the study eye is good enough to read at least 24 letters on an eye chart.

My eye condition affects between 2.125 mm2 and 20.125 mm2 of my retina.

My eye pressure is 22 mmHg or lower.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive intravitreal injections of ONL1204 or comparator treatments at specified intervals

48 weeks

Every 12 or 24 weeks depending on treatment group

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• ONL1204

Trial Overview The study tests ONL1204 Ophthalmic solution at two dose levels and frequencies against Avacincaptad Pegol intravitreal solution and a sham treatment in patients with GA due to AMD. ONL1204 aims to protect retinal cells by inhibiting cell death pathways.

How Is the Trial Designed?

Group I: ONL1204 Opthalmic solution (3 of 6 Treatment Groups) administered by intravitreal injection Experimental Treatment 1 Intervention

ONL1204 200 µg administered by intravitreal (IVT) injection every 24 weeks

Group II: Avacincaptad pegol intravitreal solution (6 of 6 Treatment Groups - US ONLY) Active Control 1 Intervention

Avacincaptad pegol administered by intravitreal (IVT) injection - (2 mg (0.1 mL of 20 mg/mL solution) once monthly

Group III: ONL1204 Opthalmic solution (1 of 6 Treatment Groups) administered by intravitreal injection Active Control 1 Intervention

ONL1204 100 µg administered by intravitreal (IVT) injection every 12 weeks

Group IV: ONL1204 Opthalmic solution (2 of 6 Treatment Groups) administered by intravitreal injection Active Control 1 Intervention

ONL1204 200 µg administered by intravitreal (IVT) injection every 12 weeks

Group V: Sham Comparator (4 of 6 Treatment Groups) Placebo Group 1 Intervention

Sham injection every 12 weeks

Group VI: Sham Comparator (5 of 6 Treatment Groups) Placebo Group 1 Intervention

Sham injection every 24 weeks

Who Is Running the Clinical Trial?

ONL Therapeutics

Lead Sponsor

Trials

5

Recruited

530+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06659445

NCT06659445 | Study to Evaluate Efficacy and Safety of ... Study to Evaluate Efficacy and Safety of ONL1204 in Patients With GA Associated With AMD ... Associated With Age-related Macular Degeneration (AMD). Conditions.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12467535/

In Vivo Characterization of ONL1204, a Small Peptide Inhibitor ... 1. Introduction. Age-related macular degeneration (AMD) is a leading cause of irreversible vision loss among the elderly in the developed world ...

3. onltherapeutics.com onltherapeutics.com/clinical-trials/

Clinical Trials ... Efficacy and Safety of ONL1204 (Xelafaslatide) in Patients With Geographic Atrophy (GA) Associated With Age-related Macular Degeneration (AMD). The purpose ...

4. sciencedirect.com sciencedirect.com/science/article/pii/S2666914525002520

Phase 1b Study Evaluating Safety, Tolerability, and Efficacy Age-related macular degeneration (AMD) is a leading cause of severe irreversible vision loss globally in individuals over the age of 55 years.

5. ophthalmologyadvisor.com ophthalmologyadvisor.com/reports/onl1204-fas-inhibition-treatment-may-slow-lesion-growth-in-geographic-atrophy/

Fas Inhibitor May Slow Lesion Growth in Geographic Atrophy ... ONL1204 Fas inhibition treatment for geographic atrophy secondary to age-related macular degeneration. Poster presented at: The Association ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT04744662

NCT04744662 | ONL1204 Ophthalmic Solution in Patients ... ... Safety and Tolerability of ONL1204 Ophthalmic Solution in Patients With Geographic Atrophy (GA) Associated With Age-related Macular Degeneration (AMD.