Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt Disease

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.

Inclusion criteria

• 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 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).