Two-sample binary phase 2 trials with low type I error and low sample size.

ABSTRACT: We address design of two-stage clinical trials comparing experimental and control patients. Our end point is success or failure, however measured, with null hypothesis that the chance of success in both arms is p₀ and alternative that it is p₀ among controls and p₁  > p₀ among experimental patients. Standard rules will have the null hypothesis rejected when the number of successes in the (E)xperimental arm, E, sufficiently exceeds C, that among (C)ontrols. Here, we combine one-sample rejection decision rules, E⩾m, with two-sample rules of the form E - C > r to achieve two-sample tests with low sample number and low type I error. We find designs with sample numbers not far from the minimum possible using standard two-sample rules, but with type I

SUBMITTER: Litwin S 

PROVIDER: S-EPMC5378610 | biostudies-literature | 2017 Apr

REPOSITORIES: biostudies-literature