Publication Cover
Sequential Analysis
Design Methods and Applications
Volume 38, 2019 - Issue 2
120
Views
5
CrossRef citations to date
0
Altmetric
Articles

Adaptive two-stage optimal designs for phase II clinical studies that allow early futility stopping

, &
Pages 199-213 | Received 13 Nov 2017, Accepted 20 Apr 2019, Published online: 09 Jul 2019

References

  • Banerjee, A. and Tsiatis, A. A. (2006). Adaptive Two-Stage Designs in Phase II Clinical Trials, Statistics in Medicine 25: 3382–3395.
  • Berry, D. A. (2011). Adaptive Clinical Trials: The Promise and the Caution, Journal of Clinical Oncology 29: 606–609.
  • Chawla, S. P., Staddon, A. P., Baker, L. H., Schuetze, S. M., Tolcher, A. W., D’Amato, G. Z., Blay, J.-Y. Y., Mita, M. M., Sankhala, K. K., Berk, L., Rivera, V. M., Clackson, T., Loewy, J. W., Haluska, F. G., and Demetri, G. D. (2012). Phase II Study of the Mammalian Target of RAPAMYCIN Inhibitor Ridaforolimus in Patients with Advanced Bone and Soft Tissue Sarcomas, Journal of Clinical Oncology 30: 78–84.
  • Chow, S.-C. C. and Chang, M. (2008). Adaptive Design Methods in Clinical Trials – A Review. Orphanet Journal of Rare Diseases 3: 11.
  • Englert, S. and Kieser, M. (2013). Optimal Adaptive Two-Stage Designs for Phase II Cancer Clinical Trials, Biometrical Journal 55: 955–968.
  • Katz, P. O., Gerson, L. B., and Vela, M. F. (2013). Guidelines for the Diagnosis and Management of Gastroesophageal Reflux Disease, American Journal of Gastroenterology 108: 308-328.
  • Koon, H. B., Krown, S. E., Lee, J. Y., Honda, K., Rapisuwon, S., Wang, Z., Aboulafia, D., Reid, E. G., Rudek, M. A., Dezube, B. J., and Noy, A. (2014). Phase II Trial of Imatinib in AIDS-Associated Kaposi’s Sarcoma: AIDS Malignancy Consortium Protocol 042. Journal of Clinical Oncology 32: 402–408.
  • Kwak, M. and Jung, S.-H. H. (2014). Phase II Clinical Trials with Time-to-Event Endpoints: Optimal Two-Stage Designs with One-Sample Log-Rank Test, Statistics in Medicine 33: 2004–2016.
  • Lin, Y. and Shih, W. J. (2004). Adaptive Two-Stage Designs for Single-Arm Phase IIA Cancer Clinical Trials, Biometrics 60: 482–490.
  • Noy, A., Scadden, D. T., Lee, J., Dezube, B. J., Aboulafia, D., Tulpule, A., Walmsley, S., and Gill, P. (2005). Angiogenesis Inhibitor IM862 Is Ineffective against AIDS-Kaposi’s Sarcoma in a Phase III Trial, but Demonstrates Sustained, Potent Effect of Highly Active Antiretroviral Therapy: From the AIDS Malignancy Consortium and IM862 Study Team, Journal of Clinical Oncology 23: 990–998.
  • Shan, G. (2018a). Exact Confidence Limits for the Probability of Response in Two-Stage Designs, Statistics 52: 1086–1095.
  • Shan, G. (2018b). Exact Confidence Limits for the Response Rate in Two-Stage Designs with Over- or Under-enrollment in the Second Stage, Statistical Methods in Medical Research 27: 1045–1055.
  • Shan, G., Hutson, A. D., and Wilding, G. E. (2012). Two-Stage k-Sample Designs for the Ordered Alternative Problem, Pharmaceutical Statistics 11: 287–294.
  • Shan, G. and Ma, C. (2016). Unconditional Tests for Comparing Two Ordered Multinomials, Statistical Methods in Medical Research 25: 241–254.
  • Shan, G., Wilding, G. E., Hutson, A. D., and Gerstenberger, S. (2016). Optimal Adaptive Two-Stage Designs for Early Phase II Clinical Trials, Statistics in Medicine 35: 1257–1266.
  • Shan, G., Zhang, H., and Jiang, T. (2016). Minimax and Admissible Adaptive Two-Stage Designs in Phase II Clinical Trials, BMC Medical Research Methodology 16: 90.
  • Shan, G., Zhang, H., and Jiang, T. (2017). Efficient confidence Limits for Adaptive One-Arm Two-Stage Clinical Trials with Binary Endpoints, BMC Medical Research Methodology 17: 22.
  • Simon, R. (1989). Optimal Two-Stage Designs for Phase II Clinical Trials, Controlled Clinical Trials 10: 1–10.
  • Therasse, P., Arbuck, S. G., Eisenhauer, E. A., Wanders, J., Kaplan, R. S., Rubinstein, L., Verweij, J., Van Glabbeke, M., van Oosterom, A. T., Christian, M. C., and Gwyther, S. G. (2000). New Guidelines to Evaluate the Response to Treatment in Solid Tumors. European Organization for Research and Treatment of Cancer, National Cancer Institute of the United States, National Cancer Institute of Canada, Journal of National Cancer Institute 92: 205–216.
  • Trippa, L., Lee, E. Q., Wen, P. Y., Batchelor, T. T., Cloughesy, T., Parmigiani, G., and Alexander, B. M. (2012). Bayesian Adaptive Randomized Trial Design for Patients with Recurrent Glioblastoma, Journal of Clinical Oncology 30: 3258–3263.
  • U. S. Food and Drug Administration. (2010). Guidance for Industry: Adaptive Design Clinical Trials for Drugs and Biologics.
  • Wilding, G. E., Shan, G., and Hutson, A. D. (2012). Exact Two-Stage Designs for Phase II Activity Trials with Rank-Based Endpoints, Contemporary Clinical Trials 33: 332–341.
  • Wolsey, L. A. (1998). Integer Programming, New York: Wiley.
  • Zhang, H. and Shan, G. (2019). Letter to the Editor: A Novel Confidence Interval for a Single Proportion in the Presence of Clustered Binary Outcome Data (SMMR, 2019), Statistical Methods in Medical Research.
  • Zheng, L., Rosenkranz, S. L., Taiwo, B., Para, M. F., Eron, J. J., and Hughes, M. D. (2012). The Design of Single-Arm Clinical Trials of Combination Antiretroviral Regimens for Treatment-Naive HIV-Infected Patients, AIDS Research and Human Retroviruses 29: 652–657.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.