Disease-Modifying Trials on Alzheimer Disease: What They Really Mean and How They Can Be Designed
*Chengjie Xiong, Department of Biostatistics Keywords: Alzheimer disease, disease-modifying trials, optimum design Despite major scientific advance in understanding the clinical and neuropathological characteristics of Alzheimer disease (AD), its therapeutic intervention has been a major disappointment over more than a decade. Although disease-modifying (DM) therapies have been a popular topic for at least the same duration of time in AD research community, it remains unclear about what they really mean and how clinical trials for testing the DM efficacy can be optimally designed. This talk will discuss some of the most fundamental issues in AD research and the statistical consequences in designing DM trials. I will first review some of the recent development in AD research including the clinical and neuropathological correlation, the amyloid hypotheses, the concept of preclinical AD, and the role of imaging and fluid biomarkers. I will then discuss statistical issues in designing clinical trials to test the DM efficacy of a therapy, including the time window for intervention, the efficacy endpoints, optimum design parameters, and statistical power.
|
Key Dates
-
November 1 - December 17, 2013
Online proposal submission for a session, short course and Town Hall Open -
January 6 - March 11, 2014
Online proposal submission for Roundtables Open -
April 30 - May 28, 2014
Abstract Submission Open -
June 4, 2014
Online Registration Opens -
August 8 - August 22, 2014
Invited Abstract Editing -
August 11, 2014
Short Course materials due from Instructors -
September 1, 2014
Housing Deadline -
September 15, 2014
Cancellation Deadline and Registration Closes @ 11:59 pm EDT -
September 22 - September 24, 2014
Marriott Wardman Park, Washington, DC