Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects Lesion(s) in Adolescent Subjects With STGD1

  • STATUS
    Recruiting
  • End date
    Feb 15, 2025
  • participants needed
    60
  • sponsor
    Belite Bio, Inc
Updated on 4 October 2022

Summary

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

Description

Approximately 60 subjects will be enrolled in this study. Subjects will be assigned to study drug (tinlarebant 5 mg/placebo) with treatment period of upto 24 months with 28 days of follow-up.

Details
Condition Stargardt Disease 1
Treatment Placebo, Tinlarebant
Clinical Study IdentifierNCT05244304
SponsorBelite Bio, Inc
Last Modified on4 October 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

Male or female subjects 12 to 18 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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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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