title: A two-stage phase II trial design utilizing both primary and secondary endpoints.
abstract: Phase II trials in oncology drug development are usually conducted to perform the initial assessment of treatment activity. The common designs in this setting, for example, Simon 2-stage designs, are often developed based on testing whether a parameter of interest, usually a proportion (e.g. response rate), is less than a certain level or not. These designs usually consider only one parameter. However, sometimes we may encounter situations where we need to consider not a single parameter, but multiple parameters. This paper presents a two-stage design in which both primary and secondary endpoints are utilized in the decision rules. The family-wise Type 1 error rate and statistical power of the proposed design are investigated under a variety of situations by means of Monte-Carlo simulations.
authors.affiliation: Pfizer Global Research and Development, La Jolla Laboratories, 10555 Science Center Drive, San Diego, CA 92121, USA. Xun.Lin@Pfizer.com
authors.forename: Xun
authors.initials: X
authors.lastname: Lin
authors.forename: Randy
authors.initials: R
authors.lastname: Allred
authors.forename: Glen
authors.initials: G
authors.lastname: Andrews
mesh_terms: D017322:Clinical Trials, Phase II as Topic / Q000379:methods*; D023381:Endpoint Determination; D006801:Humans; D009010:Monte Carlo Method; D012107:Research Design