Sample size computation in phase II designs combining the A’Hern design and the Sargent and Goldberg design

References

• A’Hern, R. P. 2001. Sample size tables for exact single-stage phase II designs. Statistics in Medicine 20 (6):859–866. doi:10.1002/sim.721.
   PubMed Web of Science ®Google Scholar
• Bagcchi, S. 2015. Gene mutation order affects cancer behaviour. The Lancet Oncology 16:e112. doi:10.1016/S1470-2045(15)70039-2.
   PubMed Web of Science ®Google Scholar
• Bang, Y.-J., E. Van Cutsem, A. Feyereislova, H. C. Chung, L. Shen, A. Sawaki, F. Lordick, A. Ohtsu, Y. Omuro, T. Satoh, et al. 2010. Trastuzumab in combination with chemotherapy versus chemotherapy alone for treatment of HER2-positive advanced gastric or gastro-oesophageal junction cancer (ToGA): A phase 3, open-label, randomised controlled trial. Lance 687–697. doi:10.1016/S0140-6736(10)61121-X.
   PubMed Web of Science ®Google Scholar
• Cannistra, S. A. 2009. Phase II trials in journal of clinical oncology. Journal of Clinical Oncology 27 (19):3073–3076. doi:10.1200/JCO.2009.23.1811.
   PubMed Web of Science ®Google Scholar
• Dark, G. G., A. H. Calvert, R. Grimshaw, C. Poole, K. Swenerton, S. Kaye, R. Coleman, G. Jayson, T. Le, S. Ellard, et al. 2005. Randomized trial of two intravenous schedules of the topoisomerase I inhibitor liposomal lurtotecan in women with relapsed epithelial ovarian cancer: A trial of the national cancer institute of Canada clinical trials group. Journal of Clinical Oncology 23 (9):1859–1866. doi:10.1200/JCO.2005.02.028.
   PubMed Web of Science ®Google Scholar
• Estey, E., and P. Thall. 2003. New designs for phase 2 clinical trials. Blood 102 (2):442–448. doi:10.1182/blood-2002-09-2937.Reprints.
   PubMed Web of Science ®Google Scholar
• European Organisation for Research and Treatment of Cancer. 1997. Phase II trials in the EORTC. The protocol review committee, the data center, the research and Treatment division, and the new drug development office. European organization for research and trea. European Journal of Cancer (oxford, England : 1990) 33(9):1361–1363.
   PubMedGoogle 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 28:2641–2647. doi:10.1200/JCO.2009.26.3343.
   PubMed Web of Science ®Google Scholar
• Grayling, M. J., and A. P. Mander. 2016. Do single-arm trials have a role in drug development plans incorporating randomised trials? Pharmaceutical Statistics 15 (2):143–151. doi:10.1002/pst.1726.
   PubMed Web of Science ®Google Scholar
• Heinrich, M. C., H. Joensuu, G. D. Demetri, C. L. Corless, J. Apperley, J. A. Fletcher, D. Soulieres, S. Dirnhofer, A. Harlow, A. Town, et al. 2008. Phase II, open-label study evaluating the activity of imatinib in treating life-threatening malignancies known to be associated with imatinib- sensitivetyrosine kinases. Clinical Cancer Research 14 (9):2717–2725. doi:10.1158/1078-0432.CCR-07-4575.
   PubMed Web of Science ®Google Scholar
• Herson, J., and S. K. Carter. 1986. Calibrated phase II clinical trials in oncology. Statistics in Medicine 5 (5):441–447. doi:10.1002/(ISSN)1097-0258.
   PubMed Web of Science ®Google Scholar
• Hills, R. K., and A. K. Burnett. 2011. Applicability of a “pick a winner” trial design to acute myeloid leukemia. Blood 118:2389–2394. doi:10.1182/blood-2011-02-337261.
   PubMed Web of Science ®Google Scholar
• Hobbs, B. P., B. P. Carlin, S. J. Mandrekar, and D. J. Sargent. 2011. Hierarchical commensurate and power prior models for adaptive incorporation of historical information in clinical trials. Biometrics 67 (3):1047–1056. doi:10.1111/j.1541-0420.2011.01564.x.
   PubMed Web of Science ®Google Scholar
• Hyman, D. M., I. Puzanov, V. Subbiah, J. E. Faris, I. Chau, J.-Y. Blay, J. Wolf, N. S. Raje, E. L. Diamond, A. Hollebecque, et al. 2015. Vemurafenib in multiple nonmelanoma cancers with BRAF V600 mutations. New England Journal of Medicine 373 (8):726–736. doi:10.1056/NEJMoa1502309.
   PubMed Web of Science ®Google Scholar
• Korn, E. L., C. Healy, E. Calonje, M. O’Doherty, T. Nunan, C. Page, E. Higgins, and R. Russell-Jones. 2001. Clinical trial designs for cytostatic agents: Are new approaches needed? Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology 19 (1):265–272. doi:10.1200/JCO.2001.19.1.265.
   PubMed Web of Science ®Google Scholar
• Pond, G. R., and S. Abbasi. 2011. Quantitative evaluation of single-arm versus randomized phase II cancer clinical trials. Clinical Trials (london, England) 8 (3):260–269. doi:10.1177/1740774511401764.
   PubMed Web of Science ®Google Scholar
• Psioda, M. A., and J. G. Ibrahim. 2018. Bayesian clinical trial design using historical data that inform the treatment effect. Biostatistics kxy009–kxy009. doi:10.1093/biostatistics/kxy009.
   Web of Science ®Google Scholar
• Rubinstein, L. V., E. L. Korn, B. Freidlin, S. Hunsberger, S. P. Ivy, and M. A. Smith. 2005. Design issues of randomized phase II trials and a proposal for phase II screening trials. Journal of Clinical Oncology 23:7199–7206. doi:10.1200/JCO.2005.01.149.
   PubMed Web of Science ®Google Scholar
• Sargent, D. J., and R. M. Goldberg. 2001. A flexible design for multiple armed screening trials. Statistics in Medicine 20 (7):1051–1060. doi:10.1002/sim.704.
   PubMed Web of Science ®Google Scholar
• Seymour, L., W. J. Jessen, S. J. Miller, L. Kluwe, V. F. Mautner, M. R. Wallace, C. Lázaro, G. P. Page, P. F. Worley, B. J. Aronow, et al. 2010. The design of phase II clinical trials testing cancer therapeutics: Consensus recommendations from the clinical trial design task force of the national cancer institute investigational drug steering committee. Clinical Cancer Research : an Official Journal of the American Association for Cancer Research 16(6):1764–1769. doi:10.1158/1078-0432.CCR-09-3287.
   PubMed Web of Science ®Google Scholar
• Šidák, Z. 1967. Rectangular confidence regions for the means of multivariate normal distributions. Journal of the American Statistical Association 62 (318):626–633. doi:10.1080/01621459.1967.10482935.
   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
• Simon, R., R. E. Wittes, and S. S. Ellenberg. 1985. Randomized phase II clinical trials. Cancer Treatment Reports 1375–1381. http://www.nihtraining.com/cc/ippcr/current/downloads/SW1985.pdf/http://www.ncbi.nlm.nih.gov/pubmed/4075313.
   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
• Van Den Bent, M. J., A. A. Brandes, R. Rampling, M. C. M. Kouwenhoven, J. M. Kros, A. F. Carpentier, P. M. Clement, M. Frenay, M. Campone, J.-F. Baurain, et al. 2009. Randomized phase II trial of erlotinib versus temozolomide or carmustine in recurrent glioblastoma: EORTC brain tumor group study 26034. Journal of Clinical Oncology 27 (8):1268–1274. doi:10.1200/JCO.2008.17.5984.
   PubMed Web of Science ®Google Scholar
• Van Glabbeke, M., W. Steward, and J. P. Armand. 2002. Non-randomised phase II trials of drug combinations: Often meaningless, sometimes misleading. Are there alternative strategies? European Journal of Cancer 38:635–638. doi:10.1016/S0959-8049(01)00419-1.
   PubMed Web of Science ®Google Scholar
• Wu, W., B. Bot, Y. Hu, S. M. Geyer, and D. J. Sargent. 2013. A phase II flexible screening design allowing for interim analysis and comparison with historical control. Contemporary Clinical Trials 35 (2):128–137. doi:10.1016/j.cct.2013.05.007.
   PubMed Web of Science ®Google Scholar
• Yap, C., A. Pettitt, and L. Billingham. 2013. Screened selection design for randomised phase II oncology trials: An example in chronic lymphocytic leukaemia. BMC Medical Research Methodology 13 (1):87. doi:10.1186/1471-2288-13-87.
   PubMedGoogle Scholar