NCT06388083

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

A Phase 1b Open-label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Tinlarebant in Japanese Subjects With Stargardt Disease and a Phase 2/3 Randomized, Double-masked, and Placebo-controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Tinlarebant in Subjects With Stargardt Disease

• Status: Active Not Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 60 (estimated)
• Sponsor: Belite Bio, Inc · Industry
• Sex: All
• Age: 12 Years – 20 Years
• Healthy volunteers: Not accepted

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.

Conditions

• STGD1
• Stargardt Disease 1

Interventions

Type	Name	Description
DRUG	Tinlarebant	5 mg tablet
DRUG	Placebo	Placebo tablets

Timeline

• Start date: 2024-07-31
• Primary completion: 2028-02-01
• Completion: 2028-02-01
• First posted: 2024-04-29
• Last updated: 2026-03-12

Locations

18 sites across 3 countries: United States, Japan, United Kingdom

Regulatory

• FDA-regulated drug study