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Promising drug may slow blindness in teens with rare eye disease

NCT ID NCT05244304

Disease control Sponsor: Belite Bio, Inc Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jul 24, 2026 · Updated 2 times

Summary

This study tested an experimental drug called tinlarebant in 104 teenagers with Stargardt disease, a genetic condition that causes progressive vision loss. The goal was to see if the drug could slow the growth of damage in the back of the eye. Participants took the drug or a placebo daily for a set period, and researchers measured changes in eye scans and vision tests.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

104 people

The number who actually took part.

Started

Mar 2022

Finished

Sep 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 20 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Male or female subjects 12 to 20 years old, inclusive. * Subject must have clinically diagnosed STGD1 (Stargardt disease 1) with at least 1 mutation identified in the ABCA4 gene. * Subject must have a defined aggregate atrophic lesion size within 3 disc areas (7.62 mm2), as imaged by FAF in the study eye Subjects must have a BCVA of 20/200 or better for the study eye based on ETDRS letter score * Subject and their parent(s) or legal guardian are willing to provide their consent on an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)/Human Research Ethics Committee (HREC)-approved informed consent form (ICF) prior to participating in any study-related procedures. * Subject agrees to comply with all protocol requirements. Exclusion Criteria: * Any ocular disease other than Stargardt (STGD1) at baseline that, in the opinion of the investigator, would complicate assessment of a treatment effect. * History of ocular surgery in the study eye in the last 3 months. * Investigational drug use of any kind in the last 3 months or within 5 half-lives of the investigational drug, whichever is shorter. * Any prior gene therapy. * Vitamin A (retinol) deficiency as defined as a retinol serum level less than 20 mcg/dL (=0.7 μmol/L).

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Conditions

The condition(s) this trial relates to.

severe early-childhood-onset retinal dystrophy Stargardt disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Stargardt disease 1

Contacts and locations

Locations

  • Belite Study Site

    Palo Alto, California, 94303, United States

  • Belite Study Site

    Minneapolis, Minnesota, 55435, United States

  • Belite Study Site

    Salt Lake City, Utah, 84132, United States

  • Belite Study Site

    Westmead, New South Wales, Australia

  • Belite Study Site

    East Melbourne, Victoria, Australia

  • Belite Study Site

    South Brisbane, Australia

  • Belite Study Site

    Ghent, Belgium

  • Belite Study Site

    Beijing, China

  • Belite Study Site

    Shanghai, China

  • Belite Study Site

    Paris, France

  • Belite Study Site

    Bonn, Germany

  • Belite Study Site

    Tübingen, Germany

  • Belite Study Site

    Kowloon, Hong Kong

  • Belite Study Site

    Nijmegen, Netherlands

  • Belite Study Site

    Basel, Switzerland

  • Belite Study Site

    Taipei, Taiwan

  • Belite Study Site

    Taoyuan City, Taiwan

  • Belite Study Site

    London, United Kingdom

  • Belite Study Site

    Southampton, United Kingdom

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