Choosing a Phase I Design

Authored by: Barry E. Storer

Handbook of Statistics in Clinical Oncology

Print publication date:  March  2012
Online publication date:  March  2012

Print ISBN: 9781439862001
eBook ISBN: 9781439862018
Adobe ISBN:


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Although the term phase I is sometimes applied generically to almost any “early” trial, in cancer drug development, it usually refers specifically to a dose finding trial whose major endpoint is toxicity. The goal is to find the highest dose of a potential therapeutic agent that has acceptable toxicity; this dose is referred to as the MTD (“maximum tolerable dose”), and is presumably the dose that will be used in subsequent phase II trials evaluating efficacy. Occasionally, one may encounter trials that are intermediate between phase I and phase II and are referred to as phase IB trials. This is a more heterogeneous group, but typically includes trials that are evaluating some measure of biologic efficacy over a range of doses that have been found to have acceptable toxicity in a phase I trial. In that context, the trial determining the MTD may be referred to as a phase IA trial. This chapter will focus exclusively on phase I trials with a toxicity endpoint.

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