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Published on: March 20, 2021

Randomized phase II trial designs with biomarkers

Boris Freidlin1, Lisa M McShane, Mei-Yin C Polley

  • 1Biometric Research Branch, EPN-8129, National Cancer Institute, Bethesda, MD 20852, USA. freidlinb@ctep.nci.nih.gov

Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology
|August 8, 2012
PubMed

Insights

This study introduces a novel randomized phase II biomarker trial design. This design effectively guides the selection of appropriate phase III clinical trial strategies for targeted therapies and biomarkers.

Area of Science:

  • Clinical Trials
  • Biostatistics
  • Oncology

Background:

  • Targeted therapies require biomarkers to identify patient subpopulations.
  • Phase III trials are crucial for definitive evaluation but require robust phase II data.
  • Existing phase II designs may not adequately inform phase III biomarker strategies.

Purpose of the Study:

  • To propose a new randomized phase II biomarker trial design.
  • To ensure phase II trials provide recommendations for subsequent phase III trial designs.
  • To optimize the development pathway for targeted therapies and biomarkers.

Main Methods:

  • Development of a novel randomized phase II biomarker trial design.
  • Utilizing simulations and published data for design evaluation.
  • Assessing the design's ability to recommend phase III strategies.

Main Results:

  • The proposed phase II design effectively recommends phase III trial approaches.
  • Recommendations include proceeding with or without biomarker use, or halting further development.
  • Evaluations confirmed the design's utility in guiding future clinical trial planning.

Conclusions:

  • The proposed randomized phase II biomarker trial design is effective.
  • It provides crucial guidance for selecting optimal phase III trial designs.
  • This approach enhances the efficient development of targeted therapies.

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