The i3+3 design for phase I clinical trials

J Biopharm Stat. 2020 Mar;30(2):294-304. doi: 10.1080/10543406.2019.1636811. Epub 2019 Jul 15.

Abstract

The traditional rule-based design, 3 + 3, has been shown to be less likely to achieve the objectives of dose-finding trials when compared with model-based designs. We propose a new rule-based design called i3 + 3, which is based on simple but more advanced rules that account for the variabilities in the observed data. We compare the operating characteristics for the proposed i3 + 3 design with other popular phase I designs by simulation. The i3 + 3 design is far superior than the 3 + 3 design in trial safety and the ability to identify the true MTD. Compared with model-based phase I designs, i3 + 3 also demonstrates comparable performances.

Keywords: 3+3; dose finding design; model-based design; rule-based design; toxicity.

MeSH terms

  • Algorithms
  • Clinical Trials, Phase I as Topic / methods*
  • Clinical Trials, Phase I as Topic / statistics & numerical data*
  • Cohort Studies
  • Computer Simulation / statistics & numerical data*
  • Dose-Response Relationship, Drug
  • Drug-Related Side Effects and Adverse Reactions*
  • Humans
  • Pharmaceutical Preparations / administration & dosage
  • Research Design / statistics & numerical data*

Substances

  • Pharmaceutical Preparations