Enrollment and Stopping Rules for Managing Toxicity Requiring Long Follow-Up in Phase II Oncology Trials

J Biopharm Stat. 2015;25(6):1206-14. doi: 10.1080/10543406.2015.1086779. Epub 2015 Sep 18.

Abstract

Monitoring of toxicity is often conducted in Phase II trials in oncology to avoid an excessive number of toxicities if the wrong dose is chosen for Phase II. Existing stopping rules for toxicity use information from patients who have already completed follow-up. We describe a stopping rule that uses all available data to determine whether to stop for toxicity or not when follow-up for toxicity is long. We propose an enrollment rule that prescribes the maximum number of patients that may be enrolled at any given point in the trial.

Keywords: Delayed outcome; Enrollment rule; Phase II oncology trial; Pocock boundary; Stopping rule..

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Algorithms
  • Antineoplastic Agents / adverse effects*
  • Antineoplastic Agents / pharmacokinetics
  • Antineoplastic Agents / therapeutic use*
  • Clinical Trials, Phase II as Topic / statistics & numerical data*
  • Data Interpretation, Statistical
  • Early Termination of Clinical Trials / statistics & numerical data*
  • Follow-Up Studies
  • Humans
  • Research Design
  • Research Subjects

Substances

  • Antineoplastic Agents