Study on Tinlarebant for Treating Stargardt Disease in Adolescents

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What is this study about?

This clinical trial is focused on studying Stargardt Disease, a genetic eye disorder that affects vision, particularly in adolescents. The study is testing a treatment called Tinlarebant, also known by its code name LBS-008. This medication is taken in the form of a tablet. The purpose of the study is to evaluate how effective Tinlarebant is in slowing down the progression of vision loss in young people with Stargardt Disease.

Participants in the study will be randomly assigned to receive either Tinlarebant or a placebo. The study is designed to be “double-masked,” meaning neither the participants nor the researchers will know who is receiving the actual medication and who is receiving the placebo. This helps ensure that the results are unbiased. The study will take place over a period of time, during which participants will have regular check-ups to monitor their eye health and any changes in their vision.

The main goal of the study is to see if Tinlarebant can slow the growth of areas in the eye that are losing function, known as atrophic lesions. These areas are monitored using a special type of eye photography called FAF (fundus autofluorescence). Additionally, the study will look at changes in the thickness and structure of the retina, the part of the eye that is crucial for vision, using a method called SDOCT (spectral-domain optical coherence tomography). The study aims to provide valuable information on the safety and effectiveness of Tinlarebant for treating Stargardt Disease in adolescents.

1 joining the study

Upon joining the study, the participant will be informed about the trial’s purpose and procedures. Consent must be provided by the participant and their parent or legal guardian.

2 initial assessment

An initial assessment will be conducted to confirm the diagnosis of Stargardt Disease and to ensure eligibility. This includes genetic testing for mutations in the ABCA4 gene and imaging of the eye to measure the size of atrophic lesions.

3 randomization

Participants will be randomly assigned to receive either Tinlarebant or a placebo. This process is double-masked, meaning neither the participant nor the study team will know which treatment is being administered.

4 medication administration

Participants will take the assigned medication orally. The medication is in tablet form, with a dosage of 5 mg. The frequency and duration of administration will be specified in the study protocol.

5 regular follow-up visits

Regular follow-up visits will be scheduled to monitor the participant’s health and the effects of the medication. These visits will include eye examinations and imaging to assess changes in the atrophic lesions.

6 end of study

The study is expected to conclude by September 19, 2025. At the end of the study, a final assessment will be conducted to evaluate the overall effects of the treatment.

Who Can Join the Study?

  • Participants must be male or female and between 12 to 20 years old.
  • Participants must have a clinical diagnosis of Stargardt Disease (STGD1), which is a genetic eye condition, with at least one mutation found in the ABCA4 gene.
  • Participants must have a specific size of the affected area in the eye, which should be within 3 disc areas (7.62 mm²), as seen in a special eye image called FAF. They must also have a visual acuity of 20/200 or better in the study eye, which means they can see at a certain level of clarity.
  • Participants and their parent(s) or legal guardian must agree to sign a consent form approved by an ethics committee before starting any study-related activities.

Who Cannot Join the Study?

  • Patients who have any other eye disease that could affect the study results.
  • Patients who have had eye surgery in the last 3 months.
  • Patients who are currently taking medications that could interfere with the study treatment.
  • Patients who have a history of allergic reactions to similar medications.
  • Patients who are pregnant or breastfeeding.
  • Patients who have participated in another clinical trial within the last 30 days.
  • Patients who have any serious health condition that could affect their ability to participate in the study.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

No sites found in this category

Other Sites

Site Name City Country Status
Universitair Ziekenhuis Gent Gent Belgium
Quinze-Vingts National Ophthalmology Hospital Paris France
Rheinische Friedrich-Wilhelms-Universitaet Bonn Bonn Germany
Sxuudzffl Rxusclg Ultrpuzwtm Mumauox Cellmu Nijmegen The Netherlands

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Belgium Belgium
Not recruiting
30.05.2022
France France
Not recruiting
30.05.2022
Germany Germany
Not recruiting
30.05.2022
The Netherlands The Netherlands
Not recruiting
30.05.2022

Trial locations

Investigated drugs:

Tinlarebant is being studied to see if it can help slow down the progression of Stargardt Disease, a genetic eye disorder that affects young people. The trial aims to determine if this medication can reduce the growth of damaged areas in the retina, which is crucial for maintaining vision.

Investigated diseases:

Stargardt Disease – Stargardt Disease is a genetic eye disorder that typically begins in childhood or adolescence. It is characterized by progressive vision loss due to the degeneration of the macula, a small area in the center of the retina responsible for sharp, central vision. As the disease progresses, individuals may experience difficulty with tasks that require detailed vision, such as reading or recognizing faces. The condition is caused by mutations in the ABCA4 gene, leading to the accumulation of toxic substances in the retina. Over time, this accumulation results in the formation of atrophic lesions, which further impair vision. The progression of vision loss can vary, but it generally leads to significant visual impairment.

Trial ID:
2024-513483-26-00
Protocol code:
LBS-008-CT03
NCT ID:
NCT05244304
Trial Phase:
Therapeutic confirmatory (Phase III)

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