title: Single-arm phase II trial design under parametric cure models.
abstract: The current practice of designing single-arm phase II survival trials is limited under the exponential model. Trial design under the exponential model may not be appropriate when a portion of patients are cured. There is no literature available for designing single-arm phase II trials under the parametric cure model. In this paper, a test statistic is proposed, and a sample size formula is derived for designing single-arm phase II trials under a class of parametric cure models. Extensive simulations showed that the proposed test and sample size formula perform very well under different scenarios.
authors.affiliation: Department of Biostatistics, St. Jude Children's Research Hospital, 262 Danny Thomas Place, Memphis, 38105, TN, USA.
authors.forename: Jianrong
authors.initials: J
authors.lastname: Wu
mesh_terms: D017322:Clinical Trials, Phase II as Topic / Q000379:methods*; D006801:Humans; D053208:Kaplan-Meier Estimate; D015233:Models, Statistical; D009369:Neoplasms / Q000188:drug therapy / Q000401:mortality; D012107:Research Design; D018401:Sample Size; D013223:Statistics as Topic; D016019:Survival Analysis; D016896:Treatment Outcome
keywords: clinical trial design; cure model; sample size; single-arm phase II trial; time-to-event
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