title: Phase II cancer clinical trials with a one-sample log-rank test and its corrections based on the Edgeworth expansion.
abstract: The response rate has been frequently used as the primary endpoint of phase II cancer clinical trials. It may not be an appropriate endpoint when a new treatment is not expected to produce any tumour shrinkage. When a large database for a historical control is available, the direct comparison of survival curves between a new treatment and the historical control may be made in phase II cancer clinical trials. In this paper, a one-sample log-rank test is introduced for the design and analysis of phase II cancer clinical trials with time-to-event endpoints. Corrections to the one-sample log-rank test are also derived based on the Edgeworth expansion. Simulations showed that the original one-sample log-rank test may be preferred if strictly controlling for type I error is important or when the sample size of a phase II trial is as large as 50, and a corrected one-sample log-rank test is used if the sample size of a phase II trial is small. A data set from a clinical trial conducted by the NCIC Clinical Trials Group is used to illustrate the proposed procedures.
authors.affiliation: Department of Mathematics and Statistics, Queen's University, Kingston, Ontario, Canada K7L 3N6.
authors.forename: Xiaoqun
authors.initials: X
authors.lastname: Sun
authors.forename: Paul
authors.initials: P
authors.lastname: Peng
authors.forename: Dongsheng
authors.initials: D
authors.lastname: Tu
mesh_terms: D000465:Algorithms; D001943:Breast Neoplasms / Q000188:drug therapy; D017024:Chemotherapy, Adjuvant / Q000706:statistics & numerical data; D017322:Clinical Trials, Phase II as Topic / Q000379:methods*; D003198:Computer Simulation; D003627:Data Interpretation, Statistical; D023381:Endpoint Determination / Q000379:methods*; D005260:Female; D006801:Humans; D053208:Kaplan-Meier Estimate; D015233:Models, Statistical; D009010:Monte Carlo Method; D012107:Research Design