New Approaches in Translational Medicine for Phase I Clinical Trials of CNS Drugs

Abstract Phase I clinical trials have traditionally been focused on populations of normal healthy volunteers with the goal of determining the safety, tolerability, and pharmacokinetic profile of new investigational agents. As CNS drug development shifts its focus to the development of novel molecular entities, this approach will undergo an evolution. In this chapter the authors describe the traditional Phase I approach and challenges facing CNS drug development. The chapter will propose modifications to the traditional Phase I design, including the incorporation of different populations, biomarker surrogates, and adaptive designs.