A small n sequential multiple assignment randomized trial design for use in rare disease research.

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.

RCT Entities:   Population Interventions Outcomes