Minimizing the maximum expected sample size in two-stage Phase II clinical trials with continuous outcomes.
Two-stage designs are commonly used for Phase II trials. Optimal two-stage designs have the lowest expected sample size for a specific treatment effect, for example, the null value, but can perform poorly if the true treatment effect differs. Here we introduce a design for continuous treatment responses that minimizes the maximum expected sample size across all possible treatment effects. The proposed design performs well for a wider range of treatment effects and so is useful for Phase II trials. We compare the design to a previously used optimal design and show it has superior expected sample size properties.
MRC Biostatistics Unit Hub for Trials Methodology Research, Cambridge, United Kingdom. james.wason@mrc-bsu.cam.ac.uk
James M S
JM
Wason
Adrian P
AP
Mander
D000465:Algorithms; D017322:Clinical Trials, Phase II as Topic / Q000379:methods / Q000706:statistics & numerical data*; D003627:Data Interpretation, Statistical; D006801:Humans; D011336:Probability; D016032:Randomized Controlled Trials as Topic / Q000379:methods / Q000706:statistics & numerical data*; D012107:Research Design / Q000706:statistics & numerical data*; D018401:Sample Size