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Abstract：
Background: This report describes the study design and baseline characteristics of patients with Stargardt disease (STGD1) enrolled in the STArgardt Remofuscin Treatment Trial (STARTT). Methods: In total, 87 patients with genetically confirmed STGD1 were randomized in a double-masked, placebo-controlled proof of concept trial to evaluate the safety and efficacy of 20 milligram oral remofuscin for 24 months. The primary outcome measure is change in mean quantitative autofluorescence value of an 8-segment ring centred on the fovea (qAF 8). Secondary efficacy variables are best corrected visual acuity (BCVA), low-luminance visual acuity (LLVA), mesopic microperimetry (mMP), spectral domain optical coherence tomography (SD-OCT), reading speed on Radner reading charts, and patient-reported visual function as assessed by the National Eye Institute Visual Functioning Questionnaire 25 (NEI VFQ-25) and Functional Reading Independence (FRI) Index. Results: Mean age of participants was 35±11 years with 49 (56%) female. Median qAF 8 value was 438 Units (range 210-729). Median BCVA and LLVA in decimal units were 0.50 (range 0.13-0.80) and 0.20 (range 0.06-0.63), respectively. The median of the mean retinal sensitivity with mMP was 20.4 dB (range 0.0-28.8). SD-OCT showed median central subfield retinal thickness of 142 µm (range 72-265) and median macular volume of 1.65 mm 3 (range 1.13-2.19). Compared to persons without vision impairment, both reading performance and patient-reported visual function were significantly lower (p<0.001, one sample t-test). Mean reading speed was 108±39 words/minute with logRAD-score of 0.45±0.28. Mean VFQ-25 composite score was 72±13. Mean FRI Index score 2.8±0.6. Conclusions: This trial design may serve as reference for future clinical trials as it explores the utility of qAF 8 as primary outcome measure. The baseline data represent the largest, multi-national, STGD1 cohort to date that underwent standardized qAF imaging, reading speed assessment and vision-related quality of life measures which all contribute to the characterization of STGD1. EudraCT registration: 2018-001496-20 (09/05/2019).
摘要：
背景：本报告描述了参加STArgardtRemofuscin治疗试验（STARTT）的Stargardt病患者（STGD1）的研究设计和基线特征。方法：总计，87例基因证实的STGD1患者被随机分为双掩蔽组，安慰剂对照概念验证试验，以评估20毫克口服脱褐素24个月的安全性和有效性。主要结果测量是以中央凹（qAF8）为中心的8段环的平均定量自发荧光值的变化。次要功效变量是最佳矫正视力(BCVA)，低亮度视力（LLVA），介孔显微视野（mMP），谱域光学相干层析成像(SD-OCT),在Radner阅读图表上的阅读速度，通过国家眼科研究所视觉功能问卷25（NEIVFQ-25）和功能阅读独立性（FRI）指数评估患者报告的视觉功能。结果：参与者的平均年龄为35±11岁，其中49岁（56%）为女性。qAF8的中值为438个单位(范围210-729)。十进制单位的中位数BCVA和LLVA分别为0.50（范围0.13-0.80）和0.20（范围0.06-0.63），分别。mMP的平均视网膜灵敏度中位数为20.4dB（范围为0.0-28.8）。SD-OCT显示中位中央视野下视网膜厚度为142µm（范围72-265），中位黄斑体积为1.65mm3（范围1.13-2.19）。与没有视力障碍的人相比，阅读能力和患者报告的视觉功能均显著降低(p<0.001,单样本t检验).平均阅读速度为108±39字/分钟，logRAD评分为0.45±0.28。平均VFQ-25综合评分为72±13。平均FRI指数评分2.8±0.6。结论：该试验设计可作为未来临床试验的参考，因为它探索了qAF8作为主要结局指标的实用性。基线数据代表最大的，多国,迄今为止接受标准化qAF成像的STGD1队列，阅读速度评估和与视觉相关的生活质量测量都有助于STGD1的表征。EudraCT注册：2018-001496-20（2019年5月9日）。
