Status:
COMPLETED
Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt Disease
Lead Sponsor:
Belite Bio, Inc
Conditions:
Stargardt Disease 1
Eligibility:
All Genders
12-20 years
Phase:
PHASE3
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.
Eligibility Criteria
Inclusion
- 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.
Exclusion
- 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).
Key Trial Info
Start Date :
March 28 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 12 2025
Estimated Enrollment :
104 Patients enrolled
Trial Details
Trial ID
NCT05244304
Start Date
March 28 2022
End Date
September 12 2025
Last Update
October 31 2025
Active Locations (19)
Enter a location and click search to find clinical trials sorted by distance.
1
Belite Study Site
Palo Alto, California, United States, 94303
2
Belite Study Site
Minneapolis, Minnesota, United States, 55435
3
Belite Study Site
Salt Lake City, Utah, United States, 84132
4
Belite Study Site
Westmead, New South Wales, Australia