Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects - LBS-008-CT03

Belite Bio Inc.0 个研究点目标入组 107 人开始时间: 2024年4月16日最近更新: 2024年7月22日

适应症

试验速览

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•Male or female subjects 12 to 20 years old, inclusive., Subject must have clinically diagnosed STGD1 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. For the complete list, please refer to protocol.

•Any ocular disease other than STGD1 at screening 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 deficiency as defined based upon plasma values less than 20 µg/dL (=0.7 µmol/L). For the complete list, please refer to protocol.

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