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

Phase 3

Active, not recruiting

• Conditions: Stargardt Disease 1
• Interventions: Drug: Placebo; Drug: Tinlarebant

• Registration Number: NCT05244304
• Lead Sponsor: Belite Bio, Inc

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

Detailed Description

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.

Study Timeline

• Study First Submitted: 2022-01-20
• Study First Submitted QC: 2022-02-16
• Study First Posted: 2022-02-17
• Study Start: 2022-03-28
• Status Verified: 2024-06
• Last Update Submitted: 2024-06-05
• Last Update Posted: 2024-06-07
• Primary Completion: 2025-08
• Study Completion: 2025-10

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 104

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.

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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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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	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.
Tinlarebant	Tinlarebant	5 mg tablet taken orally once a day

Primary Outcome Measures

Name	Time	Method
To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline	Baseline thru month 24

Secondary Outcome Measures

Name	Time	Method
To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) 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
To assess the systemic and ocular safety and tolerability of tinlarebant	Baseline thru month 24	Frequency, duration, and severity of AEs
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

Trial Locations

Locations (1)

Belite Study Site; 🇬🇧 Southampton, United Kingdom