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Chapter One
Phase I Clinical Trials
Anastasia Ivanova
Department of Biostatistics, University of North Carolina, Chapel Hill,
North Carolina
Nancy Flournoy
Department of Statistics, University of Missouri, Columbia, Missouri
1.1 INTRODUCTION
Phase I trials are conducted to find a dose to use in subsequent trials. They provide data on the
rate of adverse events at different dose levels and provide data for studying the pharmacokinetics
and pharmacology of the drug. Dose-finding studies that involve therapies with little
or no toxicity often enroll healthy volunteers and usually have a control group. Trials in oncology
and other life-threatening diseases such as HIV enroll patients because treatments are
usually highly toxic and to enroll healthy volunteers would not be ethical. The primary
outcome for phase I trials in oncology and HIV is typically dose-limiting toxicity. Such
studies require different design strategies.
In Section 1.2, we review dose-finding p ... read full excerpt from Statistical Advances in the Biomedical Sciences: Clinical Trials, Epidemiology, Survival Analysis, and Bioinformatics ebook
