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Updated: Feb 18, 2026

Preparation of Peripheral Blood Mononuclear Cell Pellets and Plasma from a Single Blood Draw at Clinical Trial Sites for Biomarker Analysis
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Evolution of early phase clinical trials in oncology

Nam Q Bui1, Shivaani Kummar2

  • 1Division of Medical Oncology, Stanford University School of Medicine, 780 Welch Road, CJ250L, Palo Alto, CA, 94305, USA.

Journal of Molecular Medicine (Berlin, Germany)
|November 28, 2017
PubMed

Insights

This review examines advancements in early phase oncology clinical trials, focusing on novel dose-finding strategies and expedited drug development. It explores statistical methods, seamless trial designs, and genomic matching for improved cancer therapeutics.

Area of Science:

  • Oncology
  • Clinical Trial Design
  • Pharmacology

Background:

  • Cancer treatment has advanced with targeted therapies and immuno-oncology.
  • Dramatic patient responses necessitate optimized early-phase trial designs.
  • Rethinking clinical trial processes is crucial for novel agent development.

Purpose of the Study:

  • To review the current state and future directions of phase I oncology clinical trials.
  • To discuss statistical methodologies for dose escalation and optimal dose selection.
  • To explore seamless expansion strategies and genomic matching trials.

Main Methods:

  • Review of statistical methodologies for dose escalation (rule-based and model-based).
  • Analysis of seamless expansion strategies in drug development.
  • Discussion of genomic matching trial concepts and early successes.

Main Results:

  • Exploration of dose selection beyond maximum tolerated dose.
  • Highlighting expedited drug development via seamless strategies (e.g., KEYNOTE-001).
  • Identification of challenges and successes in genomic matching trials.

Conclusions:

  • Phase I oncology trials are evolving with new statistical and strategic approaches.
  • Seamless designs and genomic matching offer promise but require careful consideration.
  • Future directions focus on optimizing dose selection and accelerating novel cancer therapies.
Keywords:
Basket trialsDrug developmentPhase 1 trialsUmbrella trials

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