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一项旨在探究tinlarebant治疗地图样萎缩的安全性和疗效的III期、多中心、随机、双盲、安慰剂对照研究(PHOENIX研究)
[Translation] A Phase III, multicenter, randomized, double-blind, placebo-controlled study to investigate the safety and efficacy of tinlarebant in the treatment of geographic atrophy (PHOENIX study)
主要目的
◆ 通过眼底自发荧光(FAF)成像评估地图样萎缩(GA)病变大小,自基线至第24个月的变化率(增长率的斜率)。 关键次要目的
◆ 采用早期治疗糖尿病视网膜病变研究(ETDRS)视力表评估最佳矫正视力(BCVA),分别在标准照度和低照度条件下测定自基线至第24个月的变化;
◆ 通过谱域光学相干断层扫描(SD-OCT)评估光感受器内/外节段连接处面积和大小(即椭圆体带[EZ]损伤),自基线至第24个月的变化;
◆ 通过SD-OCT评估视网膜平均厚度(即,外侧、中间和中央区域),自基线至第24个月的变化。
[Translation] Primary Objectives
◆ The rate of change (slope of growth rate) in geographic atrophy (GA) lesion size from baseline to month 24 assessed by fundus autofluorescence (FAF) imaging. Key Secondary Objectives
◆ To assess the best corrected visual acuity (BCVA) using the Early Treatment Diabetic Retinopathy Study (ETDRS) visual acuity chart, measured from baseline to month 24 under standard illumination and low illumination conditions;
◆ To assess the area and size of the photoreceptor inner/outer segment junction (i.e., ellipsoid zone [EZ] lesions) from baseline to month 24 by spectral domain optical coherence tomography (SD-OCT);
◆ To assess the mean retinal thickness (i.e., outer, middle, and central zones) from baseline to month 24 by SD-OCT.
一项评价Tinlarebant在青少年受试者Stargardt病治疗中的安全性和疗效的 III 期、多中心、随机、双盲、安慰剂对照研究
[Translation] A Phase III, multicenter, randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of Tinlarebant in the treatment of Stargardt 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 evaluate the efficacy secondary endpoint of Tinlarebant in adolescent subjects with STGD1 (secondary efficacy endpoint will be evaluated at 24 months); To evaluate the pharmacodynamics (PD) of Tinlarebant in adolescent subjects with STGD1; To evaluate the systemic and ocular safety and tolerability of Tinlarebant;
Exploratory objectives: To evaluate the efficacy exploratory endpoint of Tinlarebant in adolescent subjects with STGD1.
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