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An adaptive Simon Two-Stage Design for Phase 2 studies of targeted therapies

Cheryl L Jones1, Eric Holmgren

  • 1Genentech, Inc., 1 DNA Way, South San Francisco, CA 94080, USA. jones.cheryl@gene.com

Contemporary Clinical Trials
|April 7, 2007
PubMed

Insights

This study introduces an adaptive Phase 2 clinical trial design for targeted cancer therapies. This approach enhances efficiency by potentially reducing sample size and speeding up decision-making in drug development.

Area of Science:

  • Oncology
  • Clinical Trial Design
  • Biostatistics

Background:

  • Specialized medicine and targeted cancer therapies are rapidly advancing.
  • Biomarker-driven patient stratification is crucial for personalized oncology.
  • Efficient clinical trial designs are needed to accelerate drug development.

Purpose of the Study:

  • To propose an adaptive Phase 2 clinical trial design for targeted cancer therapies.
  • To evaluate the design's ability to determine preliminary efficacy in biomarker-defined subpopulations.
  • To demonstrate potential sample size savings and improved decision-making efficiency.

Main Methods:

  • Adaptation of the established Simon Two-Stage Design.
  • Application to Phase 2 clinical trials for targeted cancer therapies.
  • Focus on biomarker status for sub-population definition.

Main Results:

  • The proposed design allows for preliminary efficacy determination in specific patient subgroups.
  • Examples demonstrate substantial savings in required sample size.
  • The adaptive approach can lead to greater efficiency in clinical decision-making.

Conclusions:

  • The adapted Simon Two-Stage Design offers an efficient approach for Phase 2 trials of targeted cancer therapies.
  • Biomarker-based sub-population analysis enhances precision and resource utilization.
  • This design facilitates faster and more informed decisions in the drug development pipeline.

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