Outcome measures for clinical trials in down syndrome

Anna J. Esbensen, Stephen R. Hooper, Deborah Fidler, Sigan L. Hartley, Jamie Edgin, Xavier Liogier D'Ardhuy, George Capone, Frances A. Conners, Carolyn B. Mervis, Leonard Abbeduto, Michael Rafii, Sharon J. Krinsky-Mchale, Tiina Urv, Elisabeth Dykens, Anna Esbenson, Sigan Hartlay, Seth Keller, Sara Weir

Research output: Contribution to journalArticlepeer-review

100 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)247-281
Number of pages35
JournalAmerican Journal on Intellectual and Developmental Disabilities
Issue number3
StatePublished - May 2017


  • Assessment
  • Behavior
  • Clinical trials
  • Cognition
  • Down syndrome
  • Intellectual disability

ASJC Scopus subject areas

  • General Medicine


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