Two-stage response adaptive randomization designs for multi-arm trials with binary outcome

References

• Chow, S. C., and Y. H. Tu. 2008. On two-stage seamless adaptive design in clinical trials. Journal of the Formosan Medical Association 107 (12 Suppl):52–60. doi:10.1016/S0929-6646(09)60009-7.
   PubMed Web of Science ®Google Scholar
• Gan, H. K., A. Grothey, G. R. Pond, M. J. Moore, L. L. Siu, and D. Sargent. 2010. Randomized phase II trials: Inevitable or inadvisable? Journal of Clinical Oncology 28 (15):2641–2647. doi:10.1200/JCO.2009.26.3343.
   PubMed Web of Science ®Google Scholar
• Grossman, S. A., K. C. Schreck, K. Ballman, and B. Alexander. 2017. Point/Counterpoint: Randomized versus single-arm phase II clinical trials for patients with newly diagnosed glioblastoma. Neuro-Oncology 19 (4):469–474. doi:10.1093/neuonc/nox030.
   PubMed Web of Science ®Google Scholar
• Jin, H., and Z. Wei. 2012. A new adaptive design based on Simon’s two-stage optimal design for phase II clinical trials. Contemporary Clinical Trials. 33(6):1255–1260. doi:10.1016/j.cct.2012.07.003.
   PubMed Web of Science ®Google Scholar
• Kim, J., and M. J. Schell. 2019. Modified Simon’s minimax and optimal two-stage designs for single-arm phase II cancer clinical trials. Oncotarget. 10(42):4255–4261. doi:10.18632/oncotarget.26981.
   PubMedGoogle Scholar
• Lee, J. J., and L. Feng. 2005. Randomized phase II designs in cancer clinical trials: current status and future directions. Journal of Clinical Oncology 23 (19):4450–4457. doi:10.1200/JCO.2005.03.197.
   PubMed Web of Science ®Google Scholar
• Lin, Y., and W. J. Shih. 2004. Adaptive two-stage designs for single-arm phase IIA cancer clinical trials. Biometrics 60 (2):482–490. doi:10.1111/j.0006-341X.2004.00193.x.
   PubMed Web of Science ®Google Scholar
• Melfi, V. F., C. Page, and M. Geraldes. 2001. An adaptive randomized design with application to estimation. Canadian Journal of Statistics 29 (1):107–116. doi:10.2307/3316054.
   Web of Science ®Google Scholar
• O’Reilly, E. M., J. W. Lee, M. Zalupski, M. Capanu, J. Park, T. Golan, E. Tahover, M. A. Lowery, J. F. Chou, V. Sahai, et al. 2020. Randomized, multicenter, phase II trial of gemcitabine and cisplatin with or without veliparib in patients with pancreas adenocarcinoma and a germline BRCA/PALB2 mutation. Journal of Clinical Oncology 38 (13):1378–1388. doi:10.1200/JCO.19.02931.
   PubMed Web of Science ®Google Scholar
• Ratain, M. J., and D. J. Sargent. 2009. Optimising the design of phase II oncology trials: The importance of randomisation. European Journal of Cancer. 45(2):275–280. doi:10.1016/j.ejca.2008.10.029.
   PubMed Web of Science ®Google Scholar
• Rosenberger, W. F., N. Stallard, A. Ivanova, C. N. Harper, and M. L. Ricks. 2001. Optimal adaptive designs for binary response trials. Biometrics 57 (3):909–913. doi:10.1111/j.0006-341X.2001.00909.x.
   PubMed Web of Science ®Google Scholar
• Rosenberger, W. F., O. Sverdlov, and F. Hu. 2012. Adaptive randomization for clinical trials. Journal of Biopharmaceutical Statistics 22 (4):719–736. doi:10.1080/10543406.2012.676535.
   PubMed Web of Science ®Google Scholar
• Sambucini, V. 2015. Comparison of single-arm vs. randomized phase II clinical trials: A Bayesian approach. Journal of Biopharmaceutical Statistics 25 (3):474–489. doi:10.1080/10543406.2014.920856.
   PubMed Web of Science ®Google Scholar
• Shan, G. Promising zone two-stage design for a single-arm study with binary outcome. Statistical methods in medical research. 2023 Mar; in press: 096228022311647. Available from: https://journals.sagepub.com/eprint/KYAMUKXKMABA3CFRW6Z5/full.
   Google Scholar
• Shan, G. Exact statistical inference for categorical data. 1st. Academic Press: San Diego, CA. 2015. doi:10.1016/B978-0-08-100681-8.00001-4.
   Google Scholar
• Shan, G. mar 2021. Optimal two-stage designs based on restricted mean survival time for a single-arm study. Contemporary Clinical Trials Communications 21:100732. doi: 10.1016/j.conctc.2021.100732.
   PubMed Web of Science ®Google Scholar
• Shan, G. 2022. Randomized two-stage optimal design for interval-censored data. Journal of Biopharmaceutical Statistics 32 (2):298–307. doi:10.1080/10543406.2021.2009499.
   PubMed Web of Science ®Google Scholar
• Shan, G. 2023. Response adaptive randomization design for a two-stage study with binary response. Journal of Biopharmaceutical Statistics 1–11. doi:10.1080/10543406.2023.2170399.
   PubMed Web of Science ®Google Scholar
• Shan, G., S. Gerstenberger, and P. V. Benos. 2017, dec. Fisher’s exact approach for post hoc analysis of a chi-squared test. PLOS ONE 12 (12):e0188709. doi: 10.1371/journal.pone.0188709.
   PubMed Web of Science ®Google Scholar
• Shan, G., A. Ritter, J. Miller, C. Bernick Effects of dose change on the success of clinical trials. Contemporary Clinical Trials Communications. 2022 100988. Available from: https://linkinghub.elsevier.com/retrieve/pii/S2451865422001053.
   Google Scholar
• Shan, G., G. E. Wilding, A. D. Hutson, and S. Gerstenberger. 2016, apr. Optimal adaptive two-stage designs for early phase II clinical trials. Statistics in Medicine 35 (8):1257–1266. doi: 10.1002/sim.6794.
   PubMed Web of Science ®Google Scholar
• Shan, G., H. Zhang, and T. Jiang. 2016, aug. Minimax and admissible adaptive two-stage designs in phase II clinical trials. BMC Medical Research Methodology 16 (1):90. doi: http://dx.doi.org/10.1186/s12874-016-0194-3.
   PubMed Web of Science ®Google Scholar
• Simon, R. 1989. Optimal two-stage designs for phase II clinical trials. Controlled Clinical Trials 10 (1):1–10. doi:10.1016/0197-2456(89)90015-9.
   PubMedGoogle Scholar
• Tang, H., N. R. Foster, A. Grothey, S. M. Ansell, R. M. Goldberg, and D. J. Sargent. 2010. Comparison of error rates in single-arm versus randomized phase II cancer clinical trials. Journal of Clinical Oncology 28 (11):1936–1941. doi:10.1200/JCO.2009.25.5489.
   PubMed Web of Science ®Google Scholar
• Tehranisa, J. S., and W. J. Meurer. 2014. Can response-adaptive randomization increase participation in acute stroke trials? Stroke 45 (7):2131–2133. doi:10.1161/STROKEAHA.114.005418.
   PubMed Web of Science ®Google Scholar
• Thall, P., P. Fox, and J. Wathen. 2015. Statistical controversies in clinical research: Scientific and ethical problems with adaptive randomization in comparative clinical trials. Annals of Oncology 26 (8):1621–1628. doi:10.1093/annonc/mdv238.
   PubMed Web of Science ®Google Scholar
• Thall, P. F., R. Simon, and S. S. Ellenberg. 1988. Two-stage selection and testing designs for comparative clinical trials. Biometrika. 75(2):303–310. doi:10.1093/biomet/75.2.303.
   Web of Science ®Google Scholar
• Viele, K., K. Broglio, A. McGlothlin, and B. R. Saville. 2020. Comparison of methods for control allocation in multiple arm studies using response adaptive randomization. Clinical Trials 17 (1):52–60. doi:10.1177/1740774519877836.
   PubMed Web of Science ®Google Scholar
• Wathen, J. K., and P. F. Thall. 2017. A simulation study of outcome adaptive randomization in multi-arm clinical trials. Clinical Trials 14 (5):432–440. doi:10.1177/1740774517692302.
   PubMed Web of Science ®Google Scholar
• Zelen, M. 1969. Play the winner rule and the controlled clinical trial. Journal of the American Statistical Association. 64(325):131–146. doi:10.1080/01621459.1969.10500959.
   Web of Science ®Google Scholar