Design and Execution of a Two-Stage Adaptive Design in Pediatric Migraine

• Activity Number: 416
• Type: Contributed
• Date/Time: Tuesday, August 2, 2011 : 2:00 PM to 3:50 PM
• Sponsor: Biopharmaceutical Section
• Author(s): Christopher Assaid*+ and Ying Zhang and Xiaoyin Frank Fan and Tony W. Ho
• Companies: Merck & Co., Inc. and Merck & Co., Inc. and Vertex Pharmaceuticals and Merck & Co., Inc.
• Keywords: Adaptive Design ; Placebo Effect ; Migraine ; Pediatric Clinical Trials

Abstract

Treatments that have been demonstrated to be effective in reducing/eliminating migraine-related pain and associated symptoms have historically failed to demonstrate efficacy in pediatric patients. One observation from completed trials is that the placebo response rate tends to be substantially higher in pediatric trials as compared to adult trials, leading to the potential for a ceiling effect.

In this presentation we will describe the multitude of historical challenges and potential theories that might explain the null results of these trials. We will also describe aspects of a novel adaptive design that were identified with a goal of maximizing the probability of success of a trial of a well-established, effective (in adults) treatment of acute migraine in pediatric patients. Additional discussion will surround some of the logistical issues associated with an adaptive design in acute migraine, particularly around multiple randomizations in a short time window for pediatric patients.