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

NCT05244304 | Completed Phase 3 DMC FDA Drug

• 1 other identifier: LBS-008-CT03
• Study Type: interventional
• Target: 104
• Locations: 11 countries
• Sites: 19

Brief 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

Conditions

Stargardt Disease 1

Outcome Measures

Primary Outcomes (1)

• To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline

  Baseline thru month 24

Secondary Outcomes (6)

• To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline

  Baseline thru month 24

• To measure the change in retinal morphology assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline

  Baseline thru month 24

• To measure change in BCVA (Best Corrected Visual Acuity) score measured by the EDTRS method from baseline

  Baseline thru month 24

• To measure change in plasma concentration of RBP4 levels (μM) from baseline

  Baseline thru month 24

• The correlation between change in plasma RBP4 level and the rate of lesion size growth (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline

  Baseline thru month 24

Other Outcomes (4)

• To measure change in total decreased autofluorescence (DAF) by FAF photography from baseline

  Baseline thru month 24

• To measure change in questionably decreased autofluorescence (QDAF) by FAF photography from baseline

  Baseline thru month 24

• To measure change in quantitative autofluorescence (qAF) level from baseline

  Baseline thru month 24

Study Arms (2)

Tinlarebant

EXPERIMENTAL

5 mg tablet taken orally once a day

Drug: Tinlarebant

Placebo

PLACEBO COMPARATOR

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.

Drug: Placebo

Interventions

Tinlarebant DRUG

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

Tinlarebant

Placebo DRUG

Not active drug

Placebo

Eligibility Criteria

• Age: 12 Years - 20 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

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).

Sponsors & Collaborators

• Belite Bio, Inc: lead

Study Sites (19)

Belite Study Site

Palo Alto, California, 94303, United States

Location

Belite Study Site

Minneapolis, Minnesota, 55435, United States

Location

Belite Study Site

Salt Lake City, Utah, 84132, United States

Location

Belite Study Site

Westmead, New South Wales, Australia

Location

Belite Study Site

East Melbourne, Victoria, Australia

Location

Belite Study Site

South Brisbane, Australia

Location

Belite Study Site

Ghent, Belgium

Location

Belite Study Site

Beijing, China

Location

Belite Study Site

Shanghai, China

Location

Belite Study Site

Paris, France

Location

Belite Study Site

Bonn, Germany

Location

Belite Study Site

Tübingen, Germany

Location

Belite Study Site

Kowloon, Hong Kong

Location

Belite Study Site

Nijmegen, Netherlands

Location

Belite Study Site

Basel, Switzerland

Location

Belite Study Site

Taipei, Taiwan

Location

Belite Study Site

Taoyuan, Taiwan

Location

Belite Study Site

London, United Kingdom

Location

Belite Study Site

Southampton, United Kingdom

Location

MeSH Terms

Conditions

Stargardt Disease

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Macular Degeneration; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Masking Details: 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)
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: January 20, 2022
• First Posted: February 17, 2022
• Study Start: March 28, 2022
• Primary Completion: August 4, 2025
• Study Completion: September 12, 2025
• Last Updated: October 31, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

HK (1); BE (1); CH (1); FR (1); TW (2); NL (1); GB (2); DE (2); AU (3); CN (2); US (3)