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

Last updated: February 12, 2025
Sponsor: Belite Bio, Inc
Overall Status: Active - Recruiting

Phase

2/3

Condition

Macular Degeneration

Geographic Atrophy

Myopic Macular Degeneration

Treatment

Placebo

Tinlarebant

Clinical Study ID

NCT06388083
LBS-008-CT07
  • Ages 12-20
  • All Genders

Study Summary

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

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Subjects must have clinically diagnosed STGD1 with at least one mutation identifiedin 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

Exclusion Criteria:

  • Any ocular disease other than STGD1 that, in the opinion of the investigator, wouldcomplicate assessment of a treatment effect.

  • History of ocular surgery in the study eye in the last 3 months.

  • Any prior gene therapy.

Study Design

Total Participants: 60
Treatment Group(s): 2
Primary Treatment: Placebo
Phase: 2/3
Study Start date:
July 31, 2024
Estimated Completion Date:
July 31, 2027

Study Description

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.

Connect with a study center

  • Belite Study Site

    Kobe,
    Japan

    Active - Recruiting

  • Belite Study Site

    Kyoto,
    Japan

    Site Not Available

  • Belite Study Site

    Tokyo,
    Japan

    Active - Recruiting

  • Bellite Study Site

    London,
    United Kingdom

    Active - Recruiting

  • Belite Study Site

    Phoenix, Arizona 85020
    United States

    Site Not Available

  • Belite Study Site

    La Jolla, California 92093
    United States

    Active - Recruiting

  • Belite Study Site

    San Diego, California 92037
    United States

    Site Not Available

  • Belite Study Site

    Miami, Florida 33136
    United States

    Site Not Available

  • Belite Study Site

    Boston, Massachusetts 02115
    United States

    Site Not Available

  • Belite Study Site

    Rochester, Minnesota 55905
    United States

    Active - Recruiting

  • Belite Study Site

    Durham, North Carolina 27705
    United States

    Active - Recruiting

  • Belite Study Site

    Philadelphia, Pennsylvania 19107
    United States

    Site Not Available

  • Belite Study Site

    Dallas, Texas 75231
    United States

    Active - Recruiting

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