A Phase 2/3 Study to Evaluate the Efficacy and Safety of Tinlarebant in Subjects With Stargardt Disease

To be considered for this study, you would need to be between 12 and 20 years old and have a clear diagnosis of Stargardt disease (type 1), which has been confirmed by a genetic test. This test checks for specific changes in a gene called ABCA4, which is linked to Stargardt disease. You would also need to have some damage, called an atrophic lesion, in one or both of your eyes, which is typical for the condition. Additionally, the vision in your 'study eye' (the eye mainly being looked at) would need to meet a certain level.

There are also some reasons why you might not be able to join. For example, if you have any other serious eye conditions that could make it difficult to properly assess how the study medicine works. You also shouldn't have had any eye surgery on your study eye in the past three months. Also, if you've ever received a special type of treatment called gene therapy previously, you wouldn't be able to participate.

If you join the study, you'll be given the study medicine, Tinlarebant, or a dummy medicine (placebo) to take every day for 24 months. You won't know whether you're getting the active medicine or the placebo, and neither will your doctor – this is called 'double-masked'. You'll have regular visits to the clinic where you'll have various eye tests and checks to monitor your eyesight and overall health. These assessments will help the doctors understand how the medicine is affecting your condition and if there are any side effects. The total duration of the main part of the study, including daily medication and follow-up, will be 24 months.