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Phase I Cancer Clinical Trial Design: Single and Combination Agents

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Biopharmaceutical Applied Statistics Symposium

Part of the book series: ICSA Book Series in Statistics ((ICSABSS))

Abstract

The chapter provides comprehensive review and comparison on single-agent and drug-combination phase I clinical trial designs, with particular focus on novel designs, including the continual reassessment method (CRM) , the Bayesian model averaging CRM (BMA-CRM) , the modified toxicity probability interval (mTPI) design, the Bayesian optimal interval (BOIN) design, and the Keyboard design. We discuss the pros and cons of these designs. Numerical study shows that the CRM, BOIN and Keyboard designs provide comparable, excellent operating characteristics, and each outperforms the mTPI design. These designs are more likely to correctly select the MTD and less likely to overdose a large percentage of patients. The extensions of the BOIN design to drug combination trials are briefly discussed. The software to implement these innovative designs is described and provided.

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Authors and Affiliations

  1. Department of Biostatistics, The University of Texas MD Anderson Cancer Center, Houston, TX, 77030, USA

    Ying Yuan, Heng Zhou & Yanhong Zhou

Authors
  1. Ying Yuan
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  2. Heng Zhou
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  3. Yanhong Zhou
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Corresponding author

Correspondence to Ying Yuan .

Editor information

Editors and Affiliations

  1. Jiann-Ping Hsu College of Public Health, Georgia Southern University, Statesboro, Georgia, USA

    Karl E. Peace

  2. School of Social Work & Gillings School of Global Public Health, University of North Carolina, Chapel Hill, North Carolina, USA

    Ding-Geng Chen

  3. Boston University, Cambridge, Massachusetts, USA

    Sandeep Menon

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Yuan, Y., Zhou, H., Zhou, Y. (2018). Phase I Cancer Clinical Trial Design: Single and Combination Agents. In: Peace, K., Chen, DG., Menon, S. (eds) Biopharmaceutical Applied Statistics Symposium . ICSA Book Series in Statistics. Springer, Singapore. https://doi.org/10.1007/978-981-10-7829-3_8

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