title: Two-stage designs for phase II cancer trials with ordinal responses.
abstract: A common approach to the design of phase II clinical trials in oncology is to conduct a two-stage trial so as to control type I and type II error rates. A number of researchers have proposed methods for the design of such trials when the response can take one of a number of ordered values such as tumor response, stable disease or progressive disease. In this case, the problem may be formulated as that of testing a complex null hypothesis. Control of the type I error rate thus requires specification of the null region and construction of a test that limits the maximum error rate over this region. In this paper we propose that the null region should be bounded by a line in the two-dimensional parameter space for the setting with a response with three levels and more generally by a plane or hyperplane. We then propose a test based on the likelihood ratio statistic and show how this may be calculated in this case for a three-level response. The method is illustrated using an example of a clinical trial to evaluate a new treatment for breast cancer.
authors.affiliation: Warwick Medical School, University of Warwick, Coventry, UK. n.stallard@warwick.ac.uk
authors.forename: Nigel
authors.initials: N
authors.lastname: Stallard
authors.forename: Louise
authors.initials: L
authors.lastname: Cockey
mesh_terms: D001943:Breast Neoplasms / Q000188:drug therapy*; D017322:Clinical Trials, Phase II as Topic; D003627:Data Interpretation, Statistical; D005260:Female; D006801:Humans; D016013:Likelihood Functions; D015233:Models, Statistical; D015999:Multivariate Analysis; D012107:Research Design