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

Outcome Measures for Clinical Trials in Down Syndrome.

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Anna J. Esbensen, Cincinnati Children's Hospital Medical Center; Stephen R. Hooper, University of North Carolina - Chapel Hill; Deborah Fidler, Colorado State University; Sigan L. Hartley, University of Wisconsin - Madison; Jamie Edgin, University of Arizona; Xavier Liogier d'Ardhuy, Roche Innovation Center Basel; George Capone, Kennedy Krieger Institute; Frances A. Conners, University of Alabama; Carolyn B. Mervis, University of Louisville; Leonard Abbeduto, MIND Institute, University of California, Davis School of Medicine; Michael Rafii, University of Southern California and University of California, San Diego; Sharon J. Krinsky-McHale, New York State Institute for Basic Research in Developmental Disabilities and Columbia University Medical Center; Tiina Urv, Eunice Kennedy Shriver National Institute of Child Health and Human Development; and Outcome Measures Working Group. Note: Outcome Measures Working Group Participants: Cognition Working Group-Leonard Abbeduto, Frances Conners, Jamie Edgin, Michael Harpold, Stephen Hooper, Sharon J. Krinsky-McHale, Nancy Raitano Lee, Xavier Liogier d'Ardhuy, Carolyn Mervis, Roger Reeves, Michael Ropacki, Paul Wang; Behavior Working Group-George Capone, Elisabeth Dykens, Anna Esbensen, Deborah Fidler, Sigan Hartley, Seth Keller, Michael Rafii, Sara Weir.


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.


Down syndrome; assessment; behavior; clinical trials; cognition; intellectual disability

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