Adaptive Dose-Finding in First-in-Man: Case Study
View Presentation View Presentation
*Anastasia Ivanova, University of North Carolina at Chapel Hill 

Keywords: Unmasking, interim analyses

Dose-finding trials are good candidates for using adaptive trial designs, because often the major emphasis is on estimation, rather than hypothesis testing. Operational issues come into play in these studies, due to the need to make informed decisions about responses at different dose levels based on interim analyses of study data. Unmasking is required to conduct the interim analyses, and the analysis results must be shared with decision makers, with the end result that more persons may be unmasked to interim results than is typically the case in a confirmatory or later phase trial. We provide case studies of adaptive designs and discuss the challenges associated with study operations in each case. Issues specific to unmasking procedures, data management practices to ensure the quality of interim findings, randomization procedures, and results dissemination are discussed.