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Verstraete and colleagues are correct that our study would not have had adequate power to detect a treatment effect of similar size to that of riluzole. The sample size of 250 patients and the futility stopping rule used in our trial design-which were chosen because of the large effect seen in the pilot study and lack of reproducibility of clinical data by other amyotrophic lateral sclerosis (ALS) scientists-would have allowed the study to continue if there was a positive effect after 85 patients were assessed.
机译:Verstraete及其同事是正确的,我们的研究没有足够的能力来检测与利鲁唑相似的治疗作用。之所以选择250名患者的样本量和我们在试验设计中使用的无效终止规则是因为允许在先导研究中看到巨大的效果以及其他肌萎缩性侧索硬化症(ALS)科学家缺乏临床数据的可重复性而选择在评估了85位患者后,如果有阳性结果,则继续研究。

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