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RecruitingPHASE2INTERVENTIONAL

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

This research study is looking into new treatments for a specific type of vision loss called Geographic Atrophy (GA), which is a serious form of Age-related Macular Degeneration (AMD). GA can cause blurry vision and even blindness, especially as people get older. Researchers are testing an eye drop (ONL1204) and an injection (Avacincaptad Pegol) to see how effective and safe they are. Some participants will also receive a dummy treatment (sham) for comparison. The study involves different doses of ONL1204 and how often it's given. This is a Phase 2 study, meaning it's still in the earlier stages of testing new medications.

At a glance

Status
Recruiting
Phase
PHASE2
Sponsor
ONL Therapeutics
Enrolment target
324
Start
28 Oct 2025
Estimated completion
01 Nov 2028

What is this study about?

This study is exploring potential new treatments for Geographic Atrophy (GA), which is a severe and progressive form of 'dry' Age-related Macular Degeneration (AMD). AMD is a common eye condition that affects the macula, the part of your eye responsible for sharp, central vision needed for reading and recognising faces. GA causes patches of cells in the macula to waste away, leading to blind spots and vision loss. It's a major cause of serious vision problems globally, especially as people get older. Lifestyle factors like smoking and diet can increase the risk, as can genetics.

Researchers are investigating a new treatment called ONL1204. This treatment is designed to protect the eye's delicate cells from damage that can lead to GA. Early tests in lab settings have shown promise in protecting different types of cells in the eye that are affected by conditions like dry AMD. The study aims to gather important information on how well ONL1204 works and whether it is safe for people with GA.

In this study, participants will receive one of three treatments: the ONL1204 eye drops (at different doses and frequencies), a different injection treatment called Avacincaptad Pegol, or a ‘sham’ treatment. The sham treatment acts as a comparison, allowing researchers to see the true effect of the active treatments. By comparing these, the study hopes to find effective ways to slow down or stop the progression of GA and preserve vision.

Key takeaways

  • This study is testing new treatments for Geographic Atrophy (GA), a serious eye condition.
  • It involves eye drops, an injection, or a 'sham' (dummy) treatment.
  • Participation includes regular clinic visits and eye assessments.
  • It's a Phase 2 study, meaning the treatments are still being researched for effectiveness and safety.
  • You must be 55 or older with specific eye condition criteria to join.

Who may be eligible?

To join this study, you need to be 55 years old or older. You must be willing to give your consent and attend all scheduled study appointments. If you are a woman who could have children, you must agree to use two types of effective contraception throughout the study and for three months after your last treatment, or already be past menopause for at least a year, or have had surgery to prevent pregnancy. If you could become pregnant, you'll also need to have pregnancy tests before starting and at each treatment visit. Men with partners who could have children must also use contraception and avoid donating sperm during the study and for three months after their last treatment.

If you currently take special vitamin supplements for your eyes (like AREDS2), you must be willing to continue taking them for the whole study. If you don't take them, you must agree not to start them during the study. You'll need to choose one approach and stick to it.

For your study eye (the eye being treated), your vision must be at a certain level (24 letters or more on an eye chart), and the pressure inside your eye must be normal (22 mmHg or less). The area of Geographic Atrophy in your study eye must meet specific size and location requirements, and importantly, it should not be directly in the very centre of your vision (the fovea), but it can be close to it.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

  1. Are you 55 years old or older?
  2. Are you willing and able to attend regular study appointments?
  3. For women who can have children: are you using two types of contraception, past menopause (12+ months), or surgically sterile?
  4. For men with partners who can have children: are you willing to use contraception?
  5. Do you have Geographic Atrophy (GA) not directly in the very center of your vision?
  6. Is your vision in the study eye at a certain level (e.g., you can read some letters on an eye chart)?
Answer every question to see your result.

What does participation involve?

If you join this study, you will be randomly assigned to receive one of the study treatments: the ONL1204 eye drops (at different doses and frequencies), an injection of Avacincaptad Pegol, or a 'sham' treatment. The sham treatment involves a procedure that feels like an injection but doesn't deliver any active medication, allowing researchers to compare the real treatment effects. You will have regular visits to the clinic where doctors and nurses will monitor your eye health and overall well-being. These visits will include various eye tests, such as vision checks, eye pressure measurements, and detailed scans of your eye to see how the GA is progressing. The treatments, whether drops or injections, will be given at these clinic visits. The total length of your participation in the study, including treatments and follow-up, will be explained in detail by the study team, but clinical trials often last for several months to a couple of years.

Potential risks and benefits

Taking part in this study could offer potential benefits, such as gaining access to a new treatment that isn't yet widely available and helping researchers learn more about Geographic Atrophy and how to treat it. However, there are potential risks too, as with any medical treatment. These could include side effects from the eye drops or injections, such as eye discomfort, irritation, or, rarely, more serious eye issues. The 'sham' treatment means you might not receive an active drug for your condition. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (117)

  • Associated Retina Consultants
    Verified postcode
    Gilbert, United States· Recruiting
  • Associated Retina Consultants
    Verified postcode
    Phoenix, United States· Recruiting
  • California Retina Consultants
    Verified postcode
    Bakersfield, United States· Recruiting
  • Retina-Vitreous Associates Medical Group
    Verified postcode
    Beverly Hills, United States· Recruiting
  • Macula and Retina Institute
    Verified postcode
    Glendale, United States· Recruiting
  • Salehi Retina Institute, dba Retina Associates of Southern California
    Verified postcode
    Huntington Beach, United States· Recruiting
  • Desert Retina Consultants
    Verified postcode
    Palm Desert, United States· Recruiting
  • Retinal Consultants Medical Group
    Verified postcode
    Sacramento, United States· Recruiting
  • Bay Area Retina Associates
    Verified postcode
    Walnut Creek, United States· Recruiting
  • Retina Consultants of Southern Colorado
    Verified postcode
    Colorado Springs, United States· Recruiting
  • Retina Group of New England
    Verified postcode
    Waterford, United States· Recruiting
  • Florida Retina Institute
    Verified postcode
    Orlando, United States· Recruiting

Common questions

What is Geographic Atrophy (GA)?

GA is an advanced form of 'dry' Age-related Macular Degeneration (AMD) where patches of cells in your central vision area waste away, causing blind spots and vision loss.

What kind of treatments are being tested?

Researchers are testing a new eye drop called ONL1204, another injection treatment called Avacincaptad Pegol, and a dummy treatment called 'sham' to compare effects.

What does Phase 2 study mean?

Phase 2 means the treatment is still in earlier stages of testing. It's given to a larger group of people to see if it works and is safe, after initial small-scale tests.

Will I definitely get the new eye drop?

No, participants are randomly assigned to receive either the ONL1204 eye drops (at different doses), the Avacincaptad Pegol injection, or the 'sham' treatment.

How long will the study last?

The exact duration will be explained by the study team, but clinical trials typically involve regular visits over several months to a couple of years.

How to find out more

Study Coordinator

Always speak to your GP or specialist before deciding to take part in a study.

Interested in taking part?

Register your interest

Share your details and the research team for "Study to Evaluate Efficacy and Safety of ONL1204 in Patients…" will contact you if you may be eligible. Always speak to your GP before agreeing to take part.

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