Critical design considerations for time-to-event endpoints in amyotrophic lateral sclerosis clinical trials.
Journal of neurology, neurosurgery, and psychiatry(2019)
摘要
Implementing distributional knowledge and incorporating accrual at the design stage could achieve large gains in the efficiency of ALS clinical trials with time-to-event endpoints. We provide an open-source platform that helps investigators to make more accurate sample size calculations and optimise the use of their available resources.
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关键词
amyotrophic lateral sclerosis,parametric survival,time-to-event endpoints,trial design
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