title: Single-arm Phase II cancer survival trial designs.
abstract: The current practice for designing single-arm Phase II trials with time-to-event endpoints is limited to using either a maximum likelihood estimate test under the exponential model or a naive approach based on dichotomizing the event time at a landmark time point. A trial designed under the exponential model may not be reliable, and the naive approach is inefficient. The modified one-sample log-rank test statistic proposed in this article fills the void. In general, the proposed test can be used to design single-arm Phase II survival trials under any parametric survival distribution. Simulation results showed that it preserves type I error well and provides adequate power for Phase II cancer trial designs with time-to-event endpoints.
authors.affiliation: a Department of Biostatistics , St. Jude Children's Research Hospital , Memphis , Tennessee , USA.
authors.forename: Jianrong
authors.initials: J
authors.lastname: Wu
mesh_terms: D017322:Clinical Trials, Phase II as Topic; D003627:Data Interpretation, Statistical; D023381:Endpoint Determination; D006801:Humans; D016013:Likelihood Functions; D009369:Neoplasms / Q000628:therapy; D012107:Research Design; D018401:Sample Size
keywords: Clinical trial design; one-sample log-rank test; sample size; single-arm Phase II; survival analysis; time-to-event