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Adaptive Phase II Trials

  • Boris FreidlinEmail author
  • Edward L. Korn
Living reference work entry
  • 9 Downloads

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.

Keywords

Biomarkers Futility monitoring Interim monitoring Outcome-adaptive randomization Phase II/III Sample size reassessment 

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Copyright information

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

Authors and Affiliations

  1. 1.Biometric Research Program, Division of Cancer Treatment and DiagnosisNational Cancer InstituteBethesdaUSA

Section editors and affiliations

  • Babak Choodari-Oskooei
    • 1
  • Mahesh Parmar
    • 2
  1. 1.Statistician, MRC Clinical Trials Unit at UCLInstitute of Clinical Trials and MethodologyLondonUK
  2. 2.MRC Clinical Trials Unit and Institute of Clinical Trials and MethodologyUniversity College of LondonLondonEngland

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