Article
Early phase trial designs and endpoints for targeted therapies in rare genotype subsets.
American Society of Clinical Oncology educational book. American Society of Clinical Oncology. Annual Meeting - 1 Jan 2014
Mandrekar Sumithra J
Abstract excerpt
The primary goal of phase I dose-finding trials has been historically to understand the safety profile of a new treatment/regimen and establish the maximum tolerated dose (MTD) for further investigation in a specific tumor type. These designs hinged on the assumption that the highest safe dose is also likely to be the most efficacious dose. In the current era of stratified medicine and targeted therapies, these...
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