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Statistical justification of expansion cohorts in phase 1 cancer trials

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Phase I cancer trials increasingly incorporate dose‐expansion cohorts (DECs), reflecting a growing demand to acquire more information about investigational drugs. Protocols commonly fail to provide a sample‐size justification or analysis… Click to show full abstract

Phase I cancer trials increasingly incorporate dose‐expansion cohorts (DECs), reflecting a growing demand to acquire more information about investigational drugs. Protocols commonly fail to provide a sample‐size justification or analysis plan for the DEC. In this study, we develop a statistical framework for the design of DECs.

Keywords: cancer; phase cancer; expansion cohorts; cancer trials

Journal Title: Cancer
Year Published: 2018

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