Phase II Trials for Heterogeneous Patient Populations with a Time-to-Event Endpoint

Main Article Content

Sin-Ho Jung

Abstract

In this paper, we consider a single-arm phase II trial with a time-to-event end-point. We assume that the study population has multiple subpopulations with different prognosis, but the study treatment is expected to be similarly efficacious across the subpopulations. We review a stratified one-sample log-rank test and present its sample size calculation method under some practical design settings. Our sample size method requires specification of the prevalence of subpopulations. We observe that the power of the resulting sample size is not very sensitive to misspecification of the prevalence.

Article Details

How to Cite
JUNG, Sin-Ho. Phase II Trials for Heterogeneous Patient Populations with a Time-to-Event Endpoint. Medical Research Archives, [S.l.], v. 5, n. 7, july 2017. ISSN 2375-1924. Available at: <https://esmed.org/MRA/mra/article/view/1377>. Date accessed: 13 july 2024.
Keywords
Censoring, prevalence, sample size formula, historical control, stratified 1-sample log-rank test
Section
Research Articles

References

Berry G. The analysis of mortality by the subject-years methods. Biometrics 1983; 39:173- 184.
Finkelstein DM, Muzikansky A, Schoenfeld DA. Comparing survival of a sample to that of a standard population. Journal of the National Cancer Institute 2003; 95:1434-1439.
Fleming TR, Harrington DP. (1991). Counting Processes and Survival Analysis. New York: Wiley.
Jung SH. (2013). Randomized phase II cancer clinical trials. New York: Chapman & Hall.

Jung SH, Chang M, Kang S. Phase II cancer clinical trials with heterogeneous patient populations. Journal of Biopharmaceutical Statistics 2012; 22:312-328.
Jung SH, Lee TY, Kim KM, George S. Admissible two-stage designs for phase II cancer clinical trials. Statistics in Medicine 2004; 23:561-569.
Kwak MJ, Jung SH. Phase II clinical trials with time-to-event endpoints: Optimal two-stage designs with one-sample log-rank test. Statistics in Medicine 2013; 33:2004-2016.
London WB, Chang MN. One- and two-stage designs for stratified phase II clinical trials.
Statistics in Medicine 2005; 24:2597-2611.

Simon R. Optimal two-stage designs for phase II clinical trials. Controlled Clinical Trials
1989; 10:1-10.

Sposto R, Gaynon PS. An adjustment for for patient heterogeneity in the design of two-stage phase II trials. Statistics in Medicine 2009; 28:2566-2579.
Woolson RF. Rank-tests and a one-sample log-rank test for comparing observed survival- data to a standard population. Biometrics 1981; 37:687-696.