An adaptive optimal design for the E(max) model and its application in clinical trials.

A project team working on a compound to treat Alzheimer's disease is carrying out a first-time-in-human dose-escalation study in patients. The team wished to maximize the efficiency of the study by using doses targeted at maximizing information about the dose-response relationship within certain safety constraints. We have developed an adaptive optimal design tool to recommend doses when the response follows an E(max) model, with functionality for pretrial simulation and in-stream analysis. We present the results of a simulation to investigate the operating characteristics of the applied algorithm.