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Adaptive Phase II Trials

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Principles and Practice of Clinical Trials

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Abstract

Phase II trials are designed to obtain preliminary efficacy information about a new therapy in order to assess whether the new therapy should be tested in definitive (phase III) trials. Adaptive trial designs allow the design of a trial to be changed during its conduct, possibly using accruing outcome data. Adaptations to phase II trials considered in this chapter include formal interim monitoring, phase II/III trial designs, adaptations related to biomarker subgroups, sample size reassessment, outcome-adaptive randomization, and adaptive pooling of outcome results across patient subgroups. Adaptive phase II trials allow for the possibility of trials reaching their conclusions earlier, with more patients being treated with therapies that have activity for them.

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Authors and Affiliations

  1. Biometric Research Program, Division of Cancer Treatment and Diagnosis, National Cancer Institute, Bethesda, MD, USA

    Boris Freidlin & Edward L. Korn

Authors
  1. Boris Freidlin
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  2. Edward L. Korn
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Corresponding author

Correspondence to Boris Freidlin .

Editor information

Editors and Affiliations

  1. Samuel Oschin Comprehensive Cancer Insti, WEST HOLLYWOOD, CA, USA

    Steven Piantadosi

  2. Bloomberg School of Public Health, Johns Hopkins Center for Clinical Trials Bloomberg School of Public Health, Baltimore, MD, USA

    Curtis L. Meinert

Section Editor information

  1. Statistician, MRC Clinical Trials Unit at UCL, Institute of Clinical Trials and Methodology, London, UK

    Babak Choodari-Oskooei

  2. MRC Clinical Trials Unit and Institute of Clinical Trials and Methodology, University College of London, London, England

    Mahesh Parmar

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© 2020 This is a U.S. Government work and not under copyright protection in the U.S.; foreign copyright protection may apply

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Freidlin, B., Korn, E.L. (2020). Adaptive Phase II Trials. In: Piantadosi, S., Meinert, C. (eds) Principles and Practice of Clinical Trials. Springer, Cham. https://doi.org/10.1007/978-3-319-52677-5_276-1

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  • DOI: https://doi.org/10.1007/978-3-319-52677-5_276-1

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  • Publisher Name: Springer, Cham

  • Print ISBN: 978-3-319-52677-5

  • Online ISBN: 978-3-319-52677-5

  • eBook Packages: Springer Reference MathematicsReference Module Computer Science and Engineering

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