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

Stargardt Disease 1 Clinical Trial

— DRAGON II  

Official title:

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06388083
• Other study ID #: LBS-008-CT07
• Status: Not yet recruiting
• Phase: Phase 2/Phase 3
• Start date: June 2024
• Est. completion date: July 2027

Study information

• Verified date: April 2024
• Source: Belite Bio, Inc
• Contact: Belite Bio Clinical Operations
• Phone: +886 972 080 097
• Email: clinicaltrial[@]belitebio.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 60
• Est. completion date: July 2027
• Est. primary completion date: June 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 20 Years

Eligibility

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

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.

Related Conditions & MeSH terms

• Macular Degeneration
• Stargardt Disease
• Stargardt Disease 1

Intervention

Drug:
• Tinlarebant
5 mg tablet
• Placebo
Placebo tablets

Locations

Country	Name	City	State
United States	Belite Study Site	Dallas	Texas

Sponsors (1)

Lead Sponsor	Collaborator
Belite Bio, Inc

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	To measure the annualized rate of change from baseline lesion size in aggregate area of atrophy		From baseline to Month 24
Secondary	To measure the annualized rate of change in total area of atrophy		From baseline to Month 24
Secondary	Change in BCVA measured by the ETDRS method		From baseline to Month 24