Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects

This research study is testing a new medication called Tinlarebant for Stargardt disease in young people. Stargardt disease is an eye condition that affects vision. The study wants to find out if Tinlarebant is safe and if it can help slow down the changes in the eye that happen with the condition. Participants will be given either Tinlarebant or a dummy treatment (placebo) without knowing which one they are receiving. Researchers will regularly check their eyes with special cameras and other tests over about two years to see if the medication makes a difference. The main goal is to see if it reduces the growth of affected areas in the eye.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Belite Bio Inc.

Enrolment target

Start

15 May 2024

Stargardt Disease

What is this study about?

This study is looking into a new treatment called Tinlarebant for Stargardt disease, a genetic eye condition that can cause vision loss, especially in young people. The goal of the study is to see if Tinlarebant is a safe and effective way to help manage the condition.

Researchers want to find out if this new medication can slow down how quickly the disease progresses. They'll be comparing Tinlarebant to a placebo, which is a dummy treatment that looks just like the real medicine but contains no active ingredients. This helps them understand if any changes are truly due to the new medication or other factors. Neither the patient nor the doctor will know who is receiving the actual medication or the placebo until the study is over.

The main thing the researchers will be watching for is how the affected areas in the back of the eye change over time. They'll use special eye scans and cameras to measure these changes over a period of about two years. If Tinlarebant can slow down the growth of these affected areas, it could be a meaningful step forward in treating Stargardt disease.

Key takeaways

This study is for young people with Stargardt disease.
It's testing a new medication called Tinlarebant against a dummy treatment.
The main goal is to see if Tinlarebant can slow down the eye condition.
Participation lasts for about two years with regular clinic visits.
Your health will be closely monitored throughout the study.

Who may be eligible?

To be considered for this study, you need to be an adult, 18 years old or older. There is no upper age limit, so people of many ages can potentially take part.

Both men and women are welcome to participate in this research. The study aims to gather information from a broad group of people to understand how the treatment works.

Beyond age and sex, there will be other specific health requirements that your doctor will check. These are important to make sure the study is safe for you and that the results are clear.

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.

Are you 18 years old or older?
Do you have a diagnosis of Stargardt disease?
Are you able to attend regular clinic appointments for about two years?
Are you comfortable with the possibility of receiving a placebo?
Are you willing to undergo various eye tests and general health checks?

Answer every question to see your result.

What does participation involve?

If you decide to take part, you would be involved for about two years. Throughout this time, you would have regular visits to the clinic. At these visits, you would receive either the study medication (Tinlarebant) or a dummy pill (placebo).

Doctors and nurses will regularly check your eyes using special cameras and other tests to see how your Stargardt disease is progressing. They will also do general health checks, like measuring your blood pressure, taking blood samples, and checking your vision. You might also fill out questionnaires about your vision. All these tests help the researchers understand if the medication is working and if it's safe.

The study team will monitor your health closely, including checking for any side effects. You will also have follow-up appointments after you finish taking the medication. It's important to attend all scheduled visits to ensure your safety and to help gather accurate information.

Potential risks and benefits

Taking part in a study like this might offer potential benefits, such as access to a new treatment before it's widely available, and careful monitoring of your eye health. However, there's no guarantee the treatment will work for you, and you might receive a placebo. Like all medications, Tinlarebant could have side effects, which the study team would explain and monitor closely. You are always free to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (4)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
France
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Unverified
Netherlands
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Unverified
Belgium
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Unverified
Germany

Common questions

What is Stargardt disease?

Stargardt disease is an inherited eye condition that affects the light-sensing cells in the back of your eye (the retina), leading to vision loss, often starting in childhood or adolescence.

What does 'Phase 3' mean for a study?

Phase 3 means this is one of the final stages of testing a new treatment. It involves more people than earlier phases and aims to confirm if the treatment is safe and effective before it can be approved for wider use.

What is a 'placebo'?

A placebo is a 'dummy' treatment that looks just like the real medicine but contains no active ingredients. It helps researchers compare the new medicine's effects accurately.

Will I know if I'm getting the real medicine or the placebo?

No, neither you nor your study doctor will know who is receiving the active medication versus the placebo until the study is over. This is called 'double-masked' and helps ensure unbiased results.

How long will I be in the study?

The main part of the study involves receiving treatment and having regular check-ups for about two years (25 months).