Evaluating tinlarebant for 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
This study is testing a new drug called tinlarebant to see if it can safely help people with Stargardt Disease improve their vision over two years.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Enrollment | 60 (estimated) |
| Ages | 12 Years to 20 Years |
| Sex | All |
| Sponsor | Belite Bio, Inc Industry-sponsored |
| Locations | 12 sites (Phoenix, Arizona and 11 other locations) |
| Trial ID | NCT06388083 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to assess the safety, tolerability, and efficacy of tinlarebant in patients with Stargardt Disease (STGD1). It consists of a Phase 1b part conducted in Japan, which is open-label and focuses on pharmacokinetics, pharmacodynamics, safety, and tolerability of the drug over 7 days. The subsequent Phase 2/3 part will be randomized, double-masked, and placebo-controlled, administering daily doses of tinlarebant for 24 months to evaluate its long-term effects. The study will include subjects with clinically diagnosed STGD1 and specific ocular conditions.
Who should consider this trial
Good fit: Ideal candidates are individuals with clinically diagnosed STGD1 and at least one mutation in the ABCA4 gene.
Not a fit: Patients with other ocular diseases or recent ocular surgeries may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could improve vision and quality of life for patients with Stargardt Disease.
How similar studies have performed: While there have been studies on Stargardt Disease, the specific approach with tinlarebant is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Phoenix, Arizona and 11 other locations
- Belite Study Site — Phoenix, Arizona, United States (Not_yet_recruiting)
- Belite Study Site — La Jolla, California, United States (Recruiting)
- Belite Study Site — Miami, Florida, United States (Not_yet_recruiting)
- Belite Study Site — Boston, Massachusetts, United States (Not_yet_recruiting)
- Belite Study Site — Rochester, Minnesota, United States (Recruiting)
- Belite Study Site — Durham, North Carolina, United States (Recruiting)
- Belite Study Site — Philadelphia, Pennsylvania, United States (Not_yet_recruiting)
- Belite Study Site — Dallas, Texas, United States (Recruiting)
- Belite Study Site — Kobe, Japan (Recruiting)
- Belite Study Site — Kyoto, Japan (Not_yet_recruiting)
- Belite Study Site — Tokyo, Japan (Recruiting)
- Bellite Study Site — London, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Belite Bio Clinical Operations
- Email: clinicaltrial@belitebio.com
- Phone: +886 972 080 097
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.