Choosing Alzheimer's disease prevention clinical trial populations

Neurobiol Aging. 2014 Mar;35(3):466-71. doi: 10.1016/j.neurobiolaging.2013.09.001. Epub 2013 Oct 9.


To assist investigators in making design choices, we modeled Alzheimer's disease prevention clinical trials. We used longitudinal Clinical Dementia Rating Scale Sum of Boxes data, retention rates, and the proportions of trial-eligible cognitively normal participants age 65 and older in the National Alzheimer's Coordinating Center Uniform Data Set to model trial sample sizes, the numbers needed to enroll to account for drop out, and the numbers needed to screen to successfully complete enrollment. We examined how enrichment strategies affected each component of the model. Relative to trials enrolling 65-year-old individuals, trials enriching for older (minimum 70 or 75) age required reduced sample sizes, numbers needed to enroll, and numbers needed to screen. Enriching for subjective memory complaints reduced sample sizes and numbers needed to enroll more than age enrichment, but increased the number needed to screen. We conclude that Alzheimer's disease prevention trials can enroll elderly participants with minimal effect on trial retention and that enriching for older individuals with memory complaints might afford efficient trial designs.

Keywords: Alzheimer's disease; Clinical trials; Prevention.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Age Factors
  • Aged
  • Aged, 80 and over
  • Alleles
  • Alzheimer Disease / diagnosis
  • Alzheimer Disease / genetics
  • Alzheimer Disease / prevention & control*
  • Alzheimer Disease / psychology
  • Apolipoproteins E / genetics
  • Clinical Trials as Topic*
  • Cognition
  • Cognitive Dysfunction
  • Education
  • Female
  • Genotype
  • Heterozygote
  • Humans
  • Interview, Psychological
  • Male
  • Memory
  • Patient Dropouts / statistics & numerical data
  • Patient Selection*
  • Retention, Psychology
  • Sample Size


  • Apolipoproteins E