Ontology highlight
ABSTRACT: 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.
SUBMITTER: Tamura RN
PROVIDER: S-EPMC4695231 | biostudies-literature | 2016 Jan
REPOSITORIES: biostudies-literature
Tamura Roy N RN Krischer Jeffrey P JP Pagnoux Christian C Micheletti Robert R Grayson Peter C PC Chen Yeh-Fong YF Merkel Peter A PA
Contemporary clinical trials 20151114
<h4>Background</h4>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.<h4>Purpose</h4>This paper illustrates the design, sample size estimation and operating characteristics of an snSMART.<h4>Methods</h4>We investigate the perfor ...[more]