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Research Article

Adaptive two-stage seamless sequential design for clinical trials

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Received 16 Apr 2023, Accepted 09 Apr 2024, Published online: 05 May 2024

References

  • Armitage, P., C. K. McPherson, and B. C. Rowe. 1969. Repeated significance tests on accumulating data. Journal of the Royal Statistical Society Series A (General) 132 (2):235–244. doi:10.2307/2343787.
  • Bauer, P., F. Bretz, V. Dragalin, F. König, and G. Wassmer. 15 Mar 2015. Twenty-five years of confirmatory adaptive designs: Opportunities and pitfalls. Statistics in Medicine 35(3):325–347. doi:10.1002/sim.6472.
  • Bauer, P., and M. Kieser. 1999. Combining different phases in the development of medical treatments within a single trial. Statistics in Medicine 18 (14):1833–1848. doi:10.1002/(SICI)1097-0258(19990730)18:14<1833:AID-SIM221>3.0.CO;2-3.
  • Bretz, F., H. Schmidli, F. Koenig, A. Racine, and W. Maurer. 2006. Confirmatory Seamless Phase II/III clinical trials with hypotheses selection at Interim: General concepts. Biometrical Journal 48 (4):623–634. doi:10.1002/bimj.200510232.
  • Chen, C., K. Anderson, D. V. Mehrotra, E. H. Rubin, and A. Tse. 2018. A 2-in-1 adaptive phase 2/3 design for expedited oncology drug development, contemp. Clinical Trials 64:238–242. doi:10.1016/j.cct.2017.09.006.
  • Gao, P., L. Y. Liu, and C. Mehta. 2013. Exact inference for adaptive group sequential designs. Statistics in Medicine 32 (23):3991–4005. doi:10.1002/sim.5847.
  • Gao, P., L. Y. Liu, and C. Mehta. 2014. Adaptive sequential testing for multiple comparisons. Journal of Biopharmaceutical Statistics 24 (5):1035–1058. doi:10.1080/10543406.2014.931409.
  • Gao, P., J. H. Ware, and C. Mehta. 2008. Sample size re-estimation for adaptive sequential designs. Journal of Biopharmaceutical Statistics 18 (6):1184–1196. doi:10.1080/10543400802369053.
  • Glimm, E., M. Bezuidenhoudt, A. Caputo, and W. Maurer. 2018. A testing strategy with adaptive dose selection and two endpoints. Statistics in Biopharmaceutical Research 10 (3):196–203. doi:10.1080/19466315.2018.1497531.
  • Huffer, F. 1986. Slepian’s inequality via the central limit theorem. Canadian Journal of Statistics 367–370. doi:10.2307/3315195.
  • Jennison, C., and B. W. Turnbull. 1997. Group sequential analysis incorporating covariance information. Journal of the American Statistical Association 92 (440):1330–1441. doi:10.1080/01621459.1997.10473654.
  • Jennison, C., and B. W. Turnbull. 2000. Group sequential methods with applications to clinical trials. Boca Raton, FL: Chapman & Hall/CRC.
  • Jin, M., and P. Zhang. 2022. A seamless adaptive 2-in-1 design expanding a phase 2 trial for treatment or dose selection into a phase 3 trial. Statistics in Biopharmaceutical Research 14 (3):334–341. doi:10.1080/19466315.2021.1914717.
  • Klinglmueller, F., M. Posch, and F. Koenig. 2014. Adaptive graph-based multiple testing procedures. Pharmaceutical Statistics 13 (6):345–356. doi:10.1002/pst.1640.
  • Koenig, F., W. Brannath, F. Bretz, and M. Posch. 2008. Adaptive Dunnett tests for treatment selection. Statistics in Medicine 27 (10):1612–1625. doi:10.1002/sim.3048.
  • Li, R., L. Wu, R. Liu, and J. Lin. 0000. Flexible seamless 2-in-1 design with sample size adaptation. https://arxiv.org/abs/2212.11433.
  • Marcus, R., P. Eric, and K. R. Gabriel. 1976. On closed testing procedures with special reference to ordered analysis of variance. Biometrika 63 (3):655–660. doi:10.1093/biomet/63.3.655.
  • O’Brien, P. C., and T. R. Fleming. 1979, A multiple testing procedure for clinical trials. Biometrics Bulletin 35(3):549–556. Sep. doi:10.2307/2530245.
  • Pocock, S. J. 1977, Group sequential methods in the design and analysis of clinical trials. Biometrika 64(2):191–199. August. doi:10.1093/biomet/64.2.191.
  • Slepian, D. 1962. The one-sided barrier problem for gaussian noise. Bell System Technical Journal 41 (2):463–501. doi:10.1002/j.1538-7305.1962.tb02419.x.
  • Stallard, N., and T. Friede. 2008. A group-sequential design for clinical trials with treatment selection. Statistics in Medicine 27 (29):6209–6227. doi:10.1002/sim.3436.
  • Stallard, N., and S. Todd. 2003. Sequential designs for phase III clinical trials incorporating treatment selection. Statistics in Medicine 22 (5):689–703. doi:10.1002/sim.1362.
  • Stallard, N., and S. Todd. 2010. Seamless phase II/III designs. Statistical Methods in Medical Research 20 (6):623–634. doi:10.1177/0962280210379035.
  • Sugitani, T., T. Hamasaki, and C. Hamada. 2013. Partition testing in confirmatory adaptive designs with structured objectives. Biometrical Journal 55 (3):341–359. doi:10.1002/bimj.201200218.
  • U.S. 2019. Department of Health and Human Services Food and Drug Administration (CDER and CBER). Guidance for Industry Adaptive Design Clinical Trials for Drugs and Biologics.
  • Whitehead, J. 1997. The design and analysis of sequential clinical trials. Rev. 2nd ed. England: John Wiley & Sons Ltd: West Sussex.
  • Zhang, P., X. Li, K. Lu, and C. Wu. 2022. A 2-in-1 adaptive design to seamlessly expand a selected dose from a phase 2 trial to a phase 3 trial for oncology drug development. Contemporary Clinical Trials 122:106931. doi:10.1016/j.cct.2022.106931.
  • Zhang, P., X. Li, K. Lu, C. Wu, and M. Ge. 2023. A variation of a 2-in-1 adaptive design to seamlessly expand a selected dose from a phase 2 trial to a phase 3 trial for oncology drug development. Contemporary Clinical Trials 127:107119. doi:10.1016/j.cct.2023.107119.