A small n sequential multiple assignment randomized trial design for use in rare disease research
Contemporary Clinical Trials(2016)
摘要
Background
Clinical 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.
Purpose
This paper illustrates the design, sample size estimation and operating characteristics of an snSMART.
Methods
We investigate the performance of a class of weighted Z statistics via computer simulations.
Results
We 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.
Conclusion
The snSMART design is promising in a rare disease setting where several alternative treatments are under consideration and small sample sizes are necessary.
更多查看译文
关键词
Re-randomization,Binary data,Weighted Z statistic
AI 理解论文
溯源树
样例
生成溯源树,研究论文发展脉络
Chat Paper
正在生成论文摘要