Outcome Measures for Clinical Trials in Down Syndrome

Am J Intellect Dev Disabil. 2017 May;122(3):247-281. doi: 10.1352/1944-7558-122.3.247.


Increasingly individuals with intellectual and developmental disabilities, including Down syndrome, are being targeted for clinical trials. However, a challenge exists in effectively evaluating the outcomes of these new pharmacological interventions. Few empirically evaluated, psychometrically sound outcome measures appropriate for use in clinical trials with individuals with Down syndrome have been identified. To address this challenge, the National Institutes of Health (NIH) assembled leading clinicians and scientists to review existing measures and identify those that currently are appropriate for trials; those that may be appropriate after expansion of age range addition of easier items, and/or downward extension of psychometric norms; and areas where new measures need to be developed. This article focuses on measures in the areas of cognition and behavior.

Keywords: Down syndrome; assessment; behavior; clinical trials; cognition; intellectual disability.

Publication types

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

MeSH terms

  • Adaptation, Psychological
  • Behavior
  • Clinical Trials as Topic*
  • Cognition
  • Down Syndrome / physiopathology
  • Down Syndrome / psychology
  • Down Syndrome / therapy*
  • Executive Function
  • Humans
  • Language
  • Learning
  • Memory
  • Outcome Assessment, Health Care*
  • Problem Behavior
  • Self-Control
  • Sleep
  • Social Perception