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In phase II clinical trials, patients are recruited sequentially and consequently the time required to complete the clinical trial will become long if the accrual rate is low. To speed up the drug development process and account for ethical issues, stochastically and non-stochastically curtailed two-stage designs have been proposed in single-arm phase II clinical trials. More recently, randomized phase II clinical trials are being increasingly recommended to avoid biased evaluation of the treatment effect when compared with a historical control. The current patient population and the historical one may be quite heterogeneous. Moreover, it is impossible to randomly assign patients for treatments. Consequently, various two-stage designs have been presented for comparing two arms. Since the sample size required in a randomized phase II trial is usually larger than that required in a single-arm phase II trial, we introduce the concept of curtailed sampling procedure to develop curtailed two-stage design for two-armed, randomized phase II clinical trials. The proposed design does not require pairwise patient response comparison, yet it allows a trial to be stopped early as soon as the difference in therapeutic effect of the experimental therapy and the standard at the end of a trial is foreknown. Copyright © 2017 Taylor & Francis Group, LLC [ABSTRACT FROM AUTHOR]

This article considers Bayesian variable selection problems for binary responses via stochastic search variable selection and Bayesian Lasso. To avoid matrix inversion in the corresponding Markov chain Monte Carlo implementations, the componentwise Gibbs sampler (CGS) idea is adopted. Moreover, we also propose automatic hyperparameter tuning rules for the proposed approaches. Simulation studies and a real example are used to demonstrate the performances of the proposed approaches. These results show that CGS approaches do not only have good performances in variable selection but also have the lower batch mean standard error values than those of original methods, especially for large number of covariates. [ABSTRACT FROM PUBLISHER]

For left-truncated and right-censored data, the technique proposed by Brookmeyer and Crowley (1982) is extended to construct a point-wise confidence interval for median residual lifetime. This procedure is computationally simpler than the score type confidence interval in Jeong et al. (2008) and empirical likelihood ratio confidence interval in Zhou and Jeong (2011). Further, transformations of the estimator are applied to improve the approximation to the asymptotic distribution for small sample sizes. A simulation study is conducted to investigate the accuracy of these confidence intervals and the implementation of these confidence intervals to two real datasets is illustrated. [ABSTRACT FROM PUBLISHER]

In clinical research, it is not uncommon to modify a trial procedure and/or statistical methods of ongoing clinical trials through protocol amendments. A major modification to the study protocol could result in a shift in target patient population. In addition, frequent and significant modifications could lead to a totally different study that is unable to address the medical questions that the original study intended to answer. In this article, we propose a logistic regression model for statistical inference based on a binary study endpoint for trials with protocol amendments. Under the proposed method, sample size adjustment is also derived. [ABSTRACT FROM AUTHOR]

In clinical development, an adaptive design combining results from two separate studies (e.g., a seamless adaptive design with a dose finding study phase and a confirmatory study phase) is commonly considered. The purpose of an adaptive design is not only to reduce lead time between the two studies, but also to evaluate the treatment effect in a more efficient way. In this paper, the focus is on the case where the study objectives are the same but the time durations of the study periods are different in the two stages. In particular, event data are collected in both stages. Statistical procedure for combining data observed from the two different stages is discussed. Furthermore, results on hypotheses testing and sample size calculation are derived for the comparison of two treatments. [ABSTRACT FROM AUTHOR]

It is well known that the logrank test is locally most powerful under proportional hazards model for right censored data when two groups have same censoring distributions. For a given data set, however, one seldom knows what the exact alternative is. Therefore, Lee (1) constructed versatile tests by combining four weighted logrank (WLR) tests with various weight functions detecting proportional, early, late and middle hazards differences, respectively. However, Pepe and Fleming (2) had pointed out that WLR tests are based on ranks, and these tests might not be sensitive to the magnitude of the difference in survival times against a specific alternative. Hence, they constructed weighted Kaplan-Meier (WKM) tests that are more sensitive than the logrank test under various alternatives. Therefore, another type of versatile tests based on the simultaneous use of WLR and WKM tests is developed in this paper and the performances of the proposed tests and Lee's tests are compared by simulation. None of the tests investigated in this paper is uniformly better than the others. Lee's maximum test is more robust for detecting various alternatives; however, for computation simplicity, the linear combination of WLR and WKM tests is recommended to apply in practice and its overall performance is better than Lee's linear combination test. [ABSTRACT FROM AUTHOR]