کد مقاله | کد نشریه | سال انتشار | مقاله انگلیسی | نسخه تمام متن |
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6150797 | 1596430 | 2016 | 4 صفحه PDF | دانلود رایگان |
BackgroundClinical trials in rare diseases are difficult to conduct due to the limited number of patients available with each disorder. We developed a Phase 2 trial which is a small n sequential multiple assignment randomized trial (snSMART) design to test several treatments for a rare disease for which no standard therapy exists.PurposeThis paper illustrates the design, sample size estimation and operating characteristics of an snSMART.MethodsWe investigate the performance of a class of weighted Z statistics via computer simulations.ResultsWe demonstrate the increase in power over traditional single stage designs, and indicate how the power changes as a function of the weight given to each stage.ConclusionThe snSMART design is promising in a rare disease setting where several alternative treatments are under consideration and small sample sizes are necessary.
Journal: Contemporary Clinical Trials - Volume 46, January 2016, Pages 48-51