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

Phase 2

Recruiting

• Conditions: Stargardt Disease 1; STGD1
• Interventions: Drug: Placebo; Drug: Tinlarebant

• Registration Number: NCT06388083
• Lead Sponsor: Belite Bio, Inc

Brief Summary

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

Detailed 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.

Study Timeline

• Study First Submitted: 2024-04-23
• Study First Submitted QC: 2024-04-26
• Study First Posted: 2024-04-29
• Study Start: 2024-07-31
• Status Verified: 2024-08
• Last Update Submitted: 2024-08-27
• Last Update Posted: 2024-08-28
• Primary Completion: 2027-06
• Study Completion: 2027-07

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

• Subjects must have clinically diagnosed STGD1 with at least one mutation identified in the ABCA4 gene.
• Subjects must have a defined aggregate atrophic lesion in 1 or both eyes.
• Minimum BCVA is required in the study eye

Read More

Exclusion Criteria

• Any ocular disease other than STGD1 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.
• Any prior gene therapy.

Read More

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Placebo tablets for tinlarebant 5 mg prepared similarly
LBS-008, Tinlarebant	Tinlarebant	5 mg tablet taken orally once a day

Primary Outcome Measures

Name	Time	Method
To measure the annualized rate of change from baseline lesion size in aggregate area of atrophy	From baseline to Month 24

Secondary Outcome Measures

Name	Time	Method
Change in BCVA measured by the ETDRS method	From baseline to Month 24
To measure the annualized rate of change in total area of atrophy	From baseline to Month 24

Trial Locations

Locations (2)

Bellite Study Site; 🇬🇧 London, United Kingdom

Belite Study Site; 🇯🇵 Tokyo, Japan