Chapter 5
Phase 1 trials
A. Lawrence Gould,
A. Lawrence Gould
Merck Research Laboratories, 770 Sumneytown Pike, West Point, PA 19486, USA
Search for more papers by this authorA. Lawrence Gould,
A. Lawrence Gould
Merck Research Laboratories, 770 Sumneytown Pike, West Point, PA 19486, USA
Search for more papers by this authorBook Editor(s):A. Lawrence Gould,
A. Lawrence Gould
Merck Research Laboratories, 770 Sumneytown Pike, West Point, PA 19486, USA
Search for more papers by this authorFirst published: 12 December 2014
Summary
The main goal of Phase 1 trials, especially those of anticancer drugs, is to determine the dose or doses to carry forward into Phase 2. Phase 1 trials of these drugs seek to determine the maximum tolerated dose (MTD). There are three general categories of design for Phase 1 cancer trial designs seeking to determine an appropriate dose to carry forward for future study based on expected toxicity. This chapter describes a number of approaches to the design of Phase 1 trials aimed at determining appropriate doses of drugs to carry forward into later stages of development. The primary, but not exclusive, focus is on identifying maximal doses of drugs for treating various forms of cancer. More recent designs, appropriate for molecularly targeted drugs, consider both efficacy and toxicity because the effectiveness of these agents is not necessarily monotonically related to dose, even though their toxicity may be.
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