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Original Articles

An approach to use of an adaptive procedure to clinical trials for molecularly heterogenous subject selection at interim

Pages 421-429 | Received 20 Jan 2018, Accepted 23 Oct 2018, Published online: 04 Dec 2018

References

  • Bauer, P., F. Bretz, V. Dragalin, F. Königa, and G. Wassmer. 2016. Twenty-five years of confirmatory adaptive designs: opportunities and pitfalls. Statistics in Medicine 35:325–47.
  • Betensky, R. A., D. N. Louis, and J. G. Cairncross. 2002. Influence of unrecognized molecular heterogeneity on randomized clinical trials. Journal of Clinical Oncology 20:2495–9.
  • Fan, C., D. S. Oh, L. Wessels, B. Weigelt, D. S. A. Nuyten, A. B. Nobel, L. J. van't Veer, and C. M. Perou. 2006. Concordance among gene-expression-based predictors for breast cancer. The New England Journal of Medicine 355 (6):560–9.
  • Freidlin, B., and R. Simon. 2005. Adaptive signature design: an adaptive clinical trial design for generating and prospectively testing a gene expression signature for sensitive patients. Clinical Cancer Therapy 11 (21):560–9.
  • Freidlin, B., W. Jiang, and R. Simon. 2010. The cross-validated adaptive signature design. Clinical Cancer Therapy 16 (2):691–8.
  • Ko, F. S. 2017. Evaluate the relative efficiency of a targeted clinical trial design to an untargeted design under the issue of cost. Communications in Statistics - Theory and Methods 46 (24):12336–44.
  • Ko, F. S. 2018. Discussion on the issue of sample size determination for a targeted to an untargeted and to a mixed effect model-based clinical trial design. Journal of Applied Statistics 45 (11):2007–19.
  • Li, G., W. J. Shih, T. Xie, and J. Lu. 2002. A sample size adjustment procedure for clinical trials based on conditional power. Biostatistics 3 (2):277–87.
  • Lynch, T. J., D. W. Bell, R. Sordella, S. Gurubhagavatula, R. A. Okimoto, B. W. Brannigan, P. L. Harris, S. M. Haserlat, J. G. Supko, F. G. Haluska, et al. 2004. Activating mutations in the epidermal growth factor receptor underlying responsiveness of non-small-cell lung cancer to gefitinib. New England Journal of Medicine 350 (21):2129–39.
  • Maitournam, A., and R. Simon. 2005. On the efficiency of targeted clinical trials. Statistics in Medicine 24 (3):329–39.
  • Paez, J. G., P. A. Janne, J. C. Lee, S. Tracy, H. Greulich, S. Gabriel, P. Herman, F. J. Kaye, N. Lindeman, T. J. Boggon, et al. 2004. EGFR mutations in lung cancer: Correlation with clinical response to gefitinib therapy. Science 304 (5676):1497–500.
  • Pao, W., T. Y. Wang, G. J. Riely., V. A. Miller, Q. Pan, M. Ladanyi, M. F. Zakowski, R. T. Heelan, M. G. Kris, and H. E. Varmus. 2005. KRAS mutations and primary resistance of lung adenocarcinomas to gefitinib or erlotinib. PLoS Medicine 2:57–61.
  • Shih, W. J. 2001. Sample size re-estimation - journey for a decade. Statistics in Medicine 20 (4):515–8.
  • Simon, N., and R. Simon. 2013. Adaptive enrichment designs for clinical trials. Biostatistics 14 (4):613–25.
  • Simon, R., and A. Maitournam. 2004. Evaluating the efficiency of targeted designs for randomized clinical trials. Clinical Cancer Research: An Official Journal of the American Association for Cancer Research 10 (20):6759–63.
  • Spencer, A. V., C. Harbron, A. Mander, J. Wason, and I. Peers. 2016. An adaptive design for updating the threshold value of a continuous biomarker. Statistics in Medicine 35 (27):4909–23.
  • Tibaldi, C., E. Giovannetti, E. Vasile, V. Mey, A. C. Laan, S. Nannizzi, R. Di Marsico, A. Antonuzzo, C. Orlandini, S. Ricciardi., et al. 2008. Correlation of CDA, ERCC1, and XPD polymorphisms with response and survival in gemcitabine/cisplatin-treated advanced non-small cell lung cancer patients. Clinical Cancer Research 14 (6):1797–803.
  • Wang, S.-J., R. T. O'Neill, and H. M. J. Hung. 2007. Approaches to evaluation of treatment effect in randomized clinical trials with genomic subset. Pharmaceutical Statistics 6 (3):227–44.
  • Wu, Y. J., C. T. Chen, H. H. Tsou, and C. F. Hsiao. 2015. Evaluating the relative cost of a targeted design versus an untargeted design for randomized clinical trials. Drug Designing 4:120.
  • Zhag, Y., and B. Guo. 2018. Optimal two stage enrichment design correcting for biomarker classification. Statistical Methods in Medical Research 27 (1):1–15.

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