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. 2017 May;122(3):247-281.
doi: 10.1352/1944-7558-122.3.247.

Outcome Measures for Clinical Trials in Down Syndrome

Anna J Esbensen  1 , Stephen R Hooper  1 , Deborah Fidler  1 , Sigan L Hartley  1 , Jamie Edgin  1 , Xavier Liogier d'Ardhuy  1 , George Capone  1 , Frances A Conners  1 , Carolyn B Mervis  1 , Leonard Abbeduto  1 , Michael Rafii  1 , Sharon J Krinsky-McHale  1 , Tiina Urv  1 ; Outcome Measures Working Group
Affiliations

Outcome Measures for Clinical Trials in Down Syndrome

Anna J Esbensen et al. Am J Intellect Dev Disabil. 2017 May.

Abstract

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.

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