[Translation] A Phase III, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt's Disease in Adolescent Subjects
主要目的:评估 Tinlarebant 在 Stargardt病1型(STGD1)青少年受试者中减缓萎缩病变增长率的疗效;
次要目的:评估 Tinlarebant 在 STGD1 青少年受试者中的疗效次要终点(次要疗效终点将在第 24 个月评估);评价 Tinlarebant 在 STGD1 青少年受试者中的药效学 (PD);评估 Tinlarebant 的全身和眼部安全性和耐受性;
探索性目的:评估 Tinlarebant 在 STGD1 青少年受试者中的疗效探索性终点。
[Translation] Primary objective: To evaluate the efficacy of Tinlarebant in slowing the growth rate of atrophic lesions in adolescent subjects with Stargardt disease type 1 (STGD1);
Secondary objectives: To assess secondary efficacy endpoints of Tinlarebant in adolescent subjects with STGD1 (secondary efficacy endpoints will be assessed at Month 24); to evaluate pharmacodynamics (PD) of Tinlarebant in adolescent subjects with STGD1; to assess Systemic and ocular safety and tolerability of Tinlarebant;
Exploratory Objectives: To evaluate the exploratory endpoints of the efficacy of Tinlarebant in adolescent subjects with STGD1.