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Research Article

A simulation study of approaches for handling disease progression in dose-finding clinical trials

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Pages 156-167 | Received 28 Oct 2019, Accepted 24 Jul 2020, Published online: 15 Sep 2020

References

  • Cheung, Y. K. 2013. Sample size formulae for the Bayesian continual reassessment method. Clinical Trials 10 (6):852–861. doi:10.1177/1740774513497294.
  • Cheung, Y. K., and R. Chappell. 2000. Sequential designs for phase I clinical trials with late-onset toxicities. Biometrics 56 (4):1177–1182. doi:10.1111/j.0006-341X.2000.01177.x.
  • Chevret, S., ed. 2006. Statistical methods for dose-finding experiments. Chichester, England: John Wiley & Sons, Ltd.
  • Koopmeiners JS and Modiano J. A Bayesian adaptive phase III clinical trial for evaluating efficacy and toxicity with delayed outcomes. Clin Trials 2014; 11: 38–48
  • Kummar, S., M. Gutierrez, J. H. Doroshow, and A. J. Murgo. 2006. Drug development in oncology: Classical cytotoxics and molecularly targeted agents. British Journal of Clinical Pharmacology 62 (1):15–26. doi:10.1111/j.1365-2125.2006.02713.x.
  • Le Tourneau, C., V. Diéras, P. Tresca, W. Cacheux, and X. Paoletti. 2010. Current challenges for the early clinical development of anticancer drugs in the era of molecularly targeted agents. Targeted Oncology 5 (1):65–72. doi:10.1007/s11523-010-0137-6.
  • Lee, S. M., D. Backenroth, Y. K. K. Cheung, D. L. Hershman, D. Vulih, B. Anderson, P. Ivy, and L. Minasian. 2016. Case example of dose optimization using data from bortezomib dose-finding clinical trials. Journal of Clinical Oncology 34 (12):1395–1401. doi:10.1200/JCO.2015.66.0662..
  • Lee, S. M., and Y. K. Cheung. 2009. Model calibration in the continual reassessment method. Clinical Trials 6 (3):227–238. doi:10.1177/1740774509105076.
  • Maki, R. G., D. R. D’Adamo, M. L. Keohan, M. Saulle, S. M. Schuetze, S. D. Undevia, M. B. Livingston, M. M. Cooney, M. L. Hensley, M. M. Mita, et al. 2009. Phase II study of sorafenib in patients with metastatic or recurrent sarcomas. Journal of Clinical Oncology 27 (19):3133–3140. doi:10.1200/JCO.2008.20.4495.
  • Mathijssen, R. H. J., A. Sparreboom, and J. Verweij. 2014. Determining the optimal dose in the development of anticancer agents. Nature Reviews Clinical Oncology 11 (5):272–281. doi:10.1038/nrclinonc.2014.40.
  • Postel-Vinay, S., C. Gomez-Roca, L. Rhoda Molife, B. Anghan, A. Levy, I. Judson, J. De Bono, J.-C. Soria, S. Kaye, and X. Paoletti. 2011. Phase I trials of molecularly targeted agents: Should we pay more attention to late toxicities? Journal of Clinical Oncology 29 (13):1728–1735. doi:10.1200/JCO.2010.31.9236.
  • Postel-Vinay, S., L. Collette, X. Paoletti, E. Rizzo, C. Massard, D. Olmos, C. Fowst, B. Levy, P. Mancini, D. Lacombe, et al. 2014. Towards new methods for the determination of dose limiting toxicities and the assessment of the recommended dose for further studies of molecularly targeted agents – dose-limiting toxicity and toxicity assessment recommendation group for early trials of targeted therapies, an European organisation for research and treatment of cancer-led study. European Journal of Cancer 50 (12):2040–2049. doi:10.1016/j.ejca.2014.04.031.
  • Riviere, M.-K., Y. Yuan, J.-H. Jourdan, F. Dubois, and S. Zohar. 2018. Phase I/II dose-finding design for molecularly targeted agent: Plateau determination using adaptive randomization. Statistical Methods in Medical Research 27 (2):466–479. doi:10.1177/0962280216631763.
  • Wages, N. A., and C. Tait. 2015. Seamless phase I/II adaptive design for oncology trials of molecularly targeted agents. Journal of Biopharmaceutical Statistics 25 (5):903–920. doi:10.1080/10543406.2014.920873.
  • Winther, S. B., T. Lembrecht Jørgensen, P. Pfeiffer, and C. Qvortrup. 2016. Can we predict toxicity and efficacy in older patients with cancer? Older patients with colorectal cancer as an example. ESMO Open 1 (3):e000021. doi:10.1136/esmoopen-2015-000021.
  • Yuan, Y., and G. Yin. 2009. Bayesian dose finding by jointly modelling toxicity and efficacy as time-to-event outcomes. Journal of the Royal Statistical Society: Series C (Applied Statistics) 58 (5):719–736. doi:10.1111/j.1467-9876.2009.00674.x.
  • Zang, Y. J., J. Lee, and Y. Yuan. 2014. Adaptive designs for identifying optimal biological dose for molecularly targeted agents. Clinical Trials 11 (3):319–327. doi:10.1177/1740774514529848.
  • Zhang, W., D. J. Sargent, and S. Mandrekar. 2006. An adaptive dose-finding design incorporating both toxicity and efficacy. Statistics in Medicine 25 (14):2365–2383. doi:10.1002/sim.2325.

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