Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects Lesion(s) in Adolescent Subjects With STGD1

• Status: Not yet recruiting
• Trial end date: 2025-02-15
• Target enrollment: 0
• Participant gender: All

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

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Belite Bio, Inc

Criteria

Inclusion Criteria:

• Male or female subjects 12 to 18 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 Criteria:

• 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).