A Phase 3 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Safety, Tolerability, and Efficacy of Gildeuretinol Acetate (ALK-001) on the Progression of Stargardt Disease (STGD) - NORTHSTAR
Stargardt disease is an inherited eye condition that causes gradual loss of central vision, often starting in childhood or young adulthood. At present, there are no approved treatments to stop or slow it down. The NORTHSTAR trial is looking at a new investigational capsule called gildeuretinol acetate (ALK-001) to see if it can slow the progression of retinal damage. In this phase 3 trial, adult volunteers take either the study drug or a matched dummy capsule (placebo) once a day for two years. Neither the volunteers nor the study doctors will know who is taking which capsule. Throughout the study, specialists will use eye scans, reading tests, and vision charts to check whether the medicine protects the retina and helps people keep their sight for longer.
At a glance
What is this study about?
Stargardt disease is a rare genetic condition that damages the macula, the small central area of the retina at the back of your eye. The macula is responsible for your sharp, detailed vision, which you need for reading, recognising faces, and watching television. Over time, harmful waste products build up in the retina and cause light-sensing cells to break down, leading to blurry vision or blind spots. Currently, there is no cure or approved medicine to stop this from happening.
This clinical trial, called NORTHSTAR, is testing a new experimental medicine called gildeuretinol acetate (also known as ALK-001). The medicine is a modified form of vitamin A designed to reduce the build-up of toxic waste in the eye, without interfering with normal vision. Researchers want to find out if taking this capsule every day can slow down the spread of damaged areas in the retina.
Because this is a phase 3 study—the final stage before a drug can be considered for general approval—it involves a large group of people to collect reliable evidence. Half of the participants will receive the active medicine and the other half will receive a dummy pill (placebo). By comparing the two groups over two years, doctors hope to prove whether this treatment can safely protect sight.
Key takeaways
- The trial tests a new capsule (gildeuretinol acetate) designed to slow down Stargardt disease.
- It is a phase 3 study comparing the active medicine against a dummy pill (placebo) over 24 months.
- Open to adults aged 18 and older with a confirmed diagnosis of Stargardt disease.
- Involves regular clinic visits for painless retinal scans, vision tests, and reading checks.
- Participation is voluntary, and you can withdraw at any time without affecting your normal care.
Who may be eligible?
This study is open to adults aged 18 and older who have been diagnosed with Stargardt disease (specifically type 1, or STGD1). Men and women can both take part.
To join, participants must have visible signs of retinal damage that can be clearly measured on standard eye scans, but still retain enough sight for doctors to track changes over time. Your eye specialist will look at past genetic tests or eye photos to confirm the diagnosis.
You may not be eligible if you have other serious eye conditions, such as advanced glaucoma or diabetic eye problems, or if you are taking certain high-dose vitamin supplements that could interfere with the study medicine. Women who are pregnant or planning to become pregnant may also be excluded, as the safety of the drug during pregnancy is not yet known.
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.
- I am aged 18 or over.
- I have a confirmed diagnosis of Stargardt disease (STGD1).
- I am able and willing to swallow a capsule every day for two years.
- I can attend regular hospital eye clinic visits for checks and scans.
- I do not have other major eye conditions that explain my vision loss.
What does participation involve?
Taking part in the NORTHSTAR trial lasts for at least two years (24 months). During this time, you will be asked to take one capsule by mouth every day. You will have regular appointments at a specialist hospital clinic—usually every few months—so the research team can monitor your health and your eyes.
At each visit, you will have painless eye examinations. These include eye scans (such as OCT and special retinal photos), standard letter-board vision tests, low-light vision checks, and reading speed tests. You will also be asked to complete quality-of-life questionnaires to share how your vision affects your daily routine and work. Travel expenses for these clinic visits are typically covered by the trial sponsor.
Potential risks and benefits
Locations (5)
- —UnverifiedBelgium
- —UnverifiedFrance
- —UnverifiedSpain
- —UnverifiedItaly
- —UnverifiedGermany
Common questions
What is a placebo and why is it used?
A placebo is a dummy capsule that looks identical to the real medicine but contains no active ingredients. It helps researchers prove whether any improvements or side effects are truly caused by the study drug.
Can I choose to get the real medicine instead of the placebo?
No. A computer randomly decides who gets the real medicine and who gets the dummy pill. Neither you nor your doctor can choose or know which one you receive during the study.
Will taking part cure my Stargardt disease?
No. The medicine is designed to slow down or halt further damage to your eye cells. It cannot repair cells that have already been lost or cure the condition.
Are the eye tests painful?
No. The tests involve reading charts, looking into cameras, and having special light scans taken of the back of your eye. Some scans may require eye drops to enlarge your pupils, which can temporarily blur your vision.
Does it cost anything to join the trial?
No. The study medicine, medical visits, and tests are provided free of charge, and trial sponsors usually reimburse reasonable travel expenses.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
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