Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt Disease

Completed · Phase 3 · Has a placebo group

Conditions studied: Stargardt Disease 1

In brief

The primary objective of this trial is to assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1

Key facts

Study ID

NCT05244304

Run by

Belite Bio, Inc

People needed

104

Starts

2022-03-28

Expected to finish

2025-09-11

Last updated by the study team

2026-07-23

Who can join

Age: 12 and older, up to 20. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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).

Where it is running

• Belite Study Site — Palo Alto, California, United States
• Belite Study Site — Minneapolis, Minnesota, United States
• Belite Study Site — Salt Lake City, Utah, 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

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.