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Targeting the Optimal Design in Randomized Clinical Trials with Binary Outcomes and No Covariate: Simulation Study

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  • Chambaz Antoine

    (Laboratoire MAP5, Université Paris Descartes and CNRS)

  • van der Laan Mark J.

    (University of California, Berkeley)

Abstract

We undertake here a comprehensive simulation study of the theoretical properties that we derive in a companion article devoted to the asymptotic study of adaptive group sequential designs in the case of randomized clinical trials (RCTs) with binary treatment, binary outcome and no covariate. By adaptive design, we mean in this setting a RCT design that allows the investigator to dynamically modify its course through data-driven adjustment of the randomization probability based on data accrued so far without negatively impacting on the statistical integrity of the trial. By adaptive group sequential design, we refer to the fact that group sequential testing methods can be equally well applied on top of adaptive designs.

Suggested Citation

  • Chambaz Antoine & van der Laan Mark J., 2011. "Targeting the Optimal Design in Randomized Clinical Trials with Binary Outcomes and No Covariate: Simulation Study," The International Journal of Biostatistics, De Gruyter, vol. 7(1), pages 1-30, January.
    • Handle: RePEc:bpj:ijbist:v:7:y:2011:i:1:n:11
    DOI: 10.2202/1557-4679.1310
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    References listed on IDEAS

    as
    1. William F. Rosenberger & Nigel Stallard & Anastasia Ivanova & Cherice N. Harper & Michelle L. Ricks, 2001. "Optimal Adaptive Designs for Binary Response Trials," Biometrics, The International Biometric Society, vol. 57(3), pages 909-913, September.
    2. van der Laan Mark J. & Rubin Daniel, 2006. "Targeted Maximum Likelihood Learning," The International Journal of Biostatistics, De Gruyter, vol. 2(1), pages 1-40, December.
    3. Chambaz Antoine & van der Laan Mark J., 2011. "Targeting the Optimal Design in Randomized Clinical Trials with Binary Outcomes and No Covariate: Theoretical Study," The International Journal of Biostatistics, De Gruyter, vol. 7(1), pages 1-32, January.
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    Cited by:

    1. Antoine Chambaz & Mark J. Laan, 2014. "Inference in Targeted Group-Sequential Covariate-Adjusted Randomized Clinical Trials," Scandinavian Journal of Statistics, Danish Society for Theoretical Statistics;Finnish Statistical Society;Norwegian Statistical Association;Swedish Statistical Association, vol. 41(1), pages 104-140, March.
    2. Chambaz Antoine & van der Laan Mark J., 2011. "Targeting the Optimal Design in Randomized Clinical Trials with Binary Outcomes and No Covariate: Theoretical Study," The International Journal of Biostatistics, De Gruyter, vol. 7(1), pages 1-32, January.

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