Special Issue Paper
How a new stratified adaptive phase II design could improve targeting population
Article first published online: 22 MAR 2011
Copyright © 2011 John Wiley & Sons, Ltd.
Statistics in Medicine
Special Issue: Sixth International French Society of Statistics meeting on statistical methods in biopharmacy: Recent advances and trends in statistics as applied to clinical trials
Volume 30, Issue 13, pages 1555–1562, 15 June 2011
How to Cite
Tournoux-Facon,, C., De Rycke, Y. and Tubert-Bitter, P. (2011), How a new stratified adaptive phase II design could improve targeting population. Statist. Med., 30: 1555–1562. doi: 10.1002/sim.4211
- Issue published online: 19 MAY 2011
- Article first published online: 22 MAR 2011
- Manuscript Accepted: 7 JAN 2011
- Manuscript Received: 29 JAN 2010
- adaptive phase II design;
- target population;
Phase II clinical trials in oncology are used for initial evaluation of the therapeutic efficacy of a new treatment regimen. Simon's two-stage or Fleming designs are commonly used for such trials. Treatment effect dilution may be observed because of heterogeneity of the population leading to negative conclusion on the whole population and termination of drug development while patients with particular characteristics may have benefit. Several authors have proposed alternative strategies based on Simon's design, including stratification on the patients' characteristics. In this paper, the authors develop a new stratified phase II design, allowing early stop of the trial for efficacy as it is an extension of Fleming's design. An example based on real data is given and the results from exact binomial calculations are developed to explore several assumptions on response rates, prevalence of each population and errors. Copyright © 2011 John Wiley & Sons, Ltd.