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

NCT ID: NCT05244304

Last Updated: 2025-10-31

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

104 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-03-28

Study Completion Date

2025-09-12

Brief Summary

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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

Detailed Description

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Approximately 90 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.

Conditions

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Stargardt Disease 1

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

This is a Phase 3 randomized, double-masked, parallel group, multicenter study to evaluate the efficacy and safety of Tinlarebant 5 mg in the treatment of adolescent subjects with STGD1.
Primary Study Purpose

TREATMENT

Blinding Strategy

DOUBLE

Participants Investigators
Eligible subjects will be randomly assigned to begin treatment in 2:1 ratio to receive the study drug (either Tinlarebant 5 mg or matching placebo)

Study Groups

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Tinlarebant

5 mg tablet taken orally once a day

Group Type EXPERIMENTAL

Tinlarebant

Intervention Type DRUG

Tinlarebant drug substance is a white to off-white substance and is dispensed as a tablet for oral administration.

Placebo

Placebo tablets for tinlarebant 5 mg are prepared similarly but use microcrystalline cellulose, NF, in place of the active drug substance and will be identical in size and appearance.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Not active drug

Interventions

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Tinlarebant

Tinlarebant drug substance is a white to off-white substance and is dispensed as a tablet for oral administration.

Intervention Type DRUG

Placebo

Not active drug

Intervention Type DRUG

Eligibility Criteria

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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).
Minimum Eligible Age

12 Years

Maximum Eligible Age

20 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Belite Bio, Inc

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Belite Study Site

Palo Alto, California, United States

Site Status

Belite Study Site

Minneapolis, Minnesota, United States

Site Status

Belite Study Site

Salt Lake City, Utah, United States

Site Status

Belite Study Site

Westmead, New South Wales, Australia

Site Status

Belite Study Site

East Melbourne, Victoria, Australia

Site Status

Belite Study Site

South Brisbane, , Australia

Site Status

Belite Study Site

Ghent, , Belgium

Site Status

Belite Study Site

Beijing, , China

Site Status

Belite Study Site

Shanghai, , China

Site Status

Belite Study Site

Paris, , France

Site Status

Belite Study Site

Bonn, , Germany

Site Status

Belite Study Site

Tübingen, , Germany

Site Status

Belite Study Site

Kowloon, , Hong Kong

Site Status

Belite Study Site

Nijmegen, , Netherlands

Site Status

Belite Study Site

Basel, , Switzerland

Site Status

Belite Study Site

Taipei, , Taiwan

Site Status

Belite Study Site

Taoyuan, , Taiwan

Site Status

Belite Study Site

London, , United Kingdom

Site Status

Belite Study Site

Southampton, , United Kingdom

Site Status

Countries

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United States Australia Belgium China France Germany Hong Kong Netherlands Switzerland Taiwan United Kingdom

Other Identifiers

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LBS-008-CT03

Identifier Type: -

Identifier Source: org_study_id

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