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NCT Number: NCT06388083

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

The goal of this clinical trial is to evaluate the safety, tolerability, and efficacy of tinlarebant in subjects with Stargardt Disease

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

STGD1 Congenital, Hereditary, and Neonatal Diseases and Abnormalities Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Macular Degeneration Retinal Degeneration Retinal Diseases Stargardt Disease Stargardt Disease 1

Age range

12 year–20 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Belite Study Site, Kobe, Japan

Loading trial locations.

About this study

This study will be comprised of a Phase 1b part conducted in Japan and a Phase 2/3 part. The Phase 1b part of the study will be open-label and will evaluate the pharmacokinetics (PK), pharmacodynamics (PD), safety, and tolerability of daily doses of 5 mg tinlarebant, administered for 7 days, in Japanese subjects with Stargardt Disease (STGD1). The Phase 2/3 part of the study will be randomized, double masked, and placebo controlled to evaluate the safety, tolerability, and efficacy of daily doses of 5 mg tinlarebant, administered for 24 months, in subjects with STGD1.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Subjects must have clinically diagnosed STGD1 with at least one mutation identified in the ABCA4 gene.
  • Subjects must have a defined aggregate atrophic lesion in 1 or both eyes.
  • Minimum BCVA is required in the study eye

Exclusion criteria

  • Any ocular disease other than STGD1 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.
  • Any prior gene therapy.

Treatment and study plan

Tinlarebant

Drug

5 mg tablet

Other names: LBS-008

Placebo

Drug

Placebo tablets

Primary outcomes

  1. To measure the annualized rate of change from baseline lesion size in aggregate area of atrophy

    Time frame: From baseline to Month 24

Secondary outcomes

  1. To measure the annualized rate of change in total area of atrophy

    Time frame: From baseline to Month 24

  2. Change in BCVA measured by the ETDRS method

    Time frame: From baseline to Month 24

Sponsors and collaborators

Lead sponsor

Belite Bio, Inc

Industry

Registry information

Official study title

A Phase 1b Open-label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Tinlarebant in Japanese Subjects With Stargardt Disease and a Phase 2/3 Randomized, Double-masked, and Placebo-controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Tinlarebant in Subjects With Stargardt Disease

Acronym: DRAGON II

Important dates

Study start
2024
Primary completion
2028
Study completion
2028
First posted
Apr 29, 2024
Registry last updated
Mar 12, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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