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Original Articles

Seamless phase II/III/IIIb clinical trial designs with different endpoints for different phases

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Pages 5436-5454 | Received 13 Feb 2018, Accepted 09 May 2019, Published online: 30 May 2019

References

  • Bauer, P., and M. Posch. 2004. Modification of the sample size and the schedule of interim analyses in survival trials based on data inspections by H. Schafer and H. -H. Muller, Statistics in Medicine 2001; 20: 3741-3751. Statistics in Medicine 23 (8):1333–4. doi: 10.1002/sim.1759.
  • Brannath, W., F. Konig, and P. Bauer. 2006. Estimation in flexible two stage designs. Statistics in Medicine 25 (19):3366–81. doi: 10.1002/sim.2258.
  • Brannath, W., M. Posch, and P. Bauer. 2002. Recursive combination tests. Journal of American Statistical Association 97 (457):236–44. doi: 10.1198/016214502753479374.
  • Bretz, F., H. Schmidli, F. Konig, A. Racine, and W. Maurer. 2006. Confirmatory seamless phase II/III clinical trials with hypotheses selection at interim: General concepts. Biometrical Journal 48 (4):623–34. doi: 10.1002/bimj.200510232.
  • Bruix, J., T. Poynard, M. Colombo, E. Schiff, K. Burak, E. J. L. Heathcote, T. Berg, J. –L. Poo, C. B. Mello, and R. Guenther. 2011. Maintenance therapy with peginterferon Alfa-2b does not prevent hapatocelluar carcinoma in cirrhotic patients with chronic hepatitis C. Gastroenterology 140 (7):1990–9. doi: 10.1053/j.gastro.2011.03.010.
  • Cui, L., H. M. Hung, and S. J. Wang. 1999. Modification of sample size in group sequential clinical trials. Biometrics 55 (3):853–7. doi: 10.1111/j.0006-341X.1999.00853.x.
  • Dimick-Santos, L. 2016. “Regulatory considerations.” Liver forum #5. Boston, MA. November 10. http://www.hivforum.org/storage/documents/2016/LF5/02_Dimick-Santos.pdf
  • Friede, T., N. Parsons, N. Stallard, S. Todd, E. V. Marquez, J. Chataway, and R. Nicholas. 2011. Designing a seamless phase II/III clinical trial using early outcomes for treatment selection: An application in multiple sclerosis. Statistics in Medicine 30:1528–40.
  • Gallo, P., C. Chuang-Stein, V. Dragalin, B. Gaydos, M. Krams, and J. Pinheiro. 2006. Adaptive designs in clinical drug development – An executive summary of the pharma working group. Journal of Biopharmaceutical Statistics 16 (3):275–83. doi: 10.1080/10543400600614742.
  • Gyawali, B., T. Shimokata, and Y. Ando. 2017. Correlation and differences in effect sizes between progression free survival (PFS) and overall survival (OS) among PD-1 inhibitors. Annals of Oncology 28 (suppl_5), 411, 1 September, mdx376.022. doi:10.1093/annonc/mdx376.022
  • Heritier, S., S. N. Lo, and C. C. Morgan. 2011. An adaptive confirmatory trial with interim treatment selection: Practical experience and unbalanced randomization. Statistics in Medicine 30:1541–54.
  • Hochberg, Y. 1988. A sharper Bonferroni procedure for multiple tests of significance. Biometrika 75 (4):800–2. doi: 10.2307/2336325.
  • Kunz, C. U., T. Friede, N. Parsons, S. Todd, and N. Stallard. 2014. Data-driven treatment selection for seamless phase II/III trials incorporating early-outcome data. Pharmaceutical Statistics 13 (4):238–46. doi: 10.1002/pst.1619.
  • Luo, X., and H. Quan. 2018. An adaptive timeline determination of survival trials. Statistics in Biopharmaceutical Research 10 (3):204–12. doi:10.1080/19466315.2018.1459311.
  • Maca, J., S. Bhattacharya, V. Dragalin, P. Gallo, and M. Krams. 2006. Adaptive seamless phase II/III designs – Background, operational aspects, and examples. Drug Information Journal 40 (4):463–73. doi: 10.1177/216847900604000412.
  • Posch, M., F. Koenig, M. Branson, W. Brannath, C. Dunger-Baldauf, and P. Bauer. 2005. Testing and estimation in flexible group sequential designs with adaptive treatment selection. Statistics in Medicine 24 (24):3697–714. doi: 10.1002/sim.2389.
  • Posch, M., W. Maurer, and F. Bretz. 2011. Type I error rate control in adaptive designs for confirmatory clinical trials with treatment selection at interim. Pharmaceutical Statistics 10 (2):96–104. doi: 10.1002/pst.413.
  • Quan, H., D. Zhou, P. Mancini, P. He, and G. Koch. 2012. Adaptive patient population selection design in clinical trials. Statistics in Biopharmaceutical Research 4 (1):86–99. doi: 10.1080/19466315.2011.633874.
  • Stallard, N. 2010. A confirmatory seamless phase II/III clinical trial design incorporating short-term endpoint information. Statistics in Medicine 29 (9):959–71. doi: 10.1002/sim.3863.
  • Stallard, N., C. U. Kunz, S. Todd, N. Parsons, and T. Friede. 2015. Flexible selection of a single treatment incorporating short-term endpoint information in a phase II/III clinical trial. Statistics in Medicine 34 (23):3104–15. doi: 10.1002/sim.6567.
  • Todd, S., and N. Stallard. 2005. A new clinical trial design combining phases II and III: Sequential designs with treatment selection and a change of endpoint. Drug Information Journal 39 (2):109–18. doi: 10.1177/009286150503900201.

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