Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Belite Bio, Inc
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.
Age
12–20
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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.
You may not be if
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).
