Outcome Measures for Clinical Trials in Individuals with Down Syndrome

April 23–24, 2015


Intellectual and Developmental Disabilities Branch (IDDB), Division of Extramural Research (DER), and Office of the Director (OD), NICHD; National Down Syndrome Society; LuMind Research Down Syndrome Foundation


Doubletree Hotel, Bethesda, Maryland


One of the persistent challenges within the field of Down syndrome research is the lack of outcome measures for clinical trials. Although there are a variety of tests for different intellectual and developmental disabilities available, it is unclear whether they are appropriate for use in clinical trials of Down syndrome.

The purpose of this meeting was to identify instruments that can assess Down syndrome clinical trial outcomes, either pharmaceutical or behavioral. The groundwork for this meeting began earlier in 2015 via teleconferences.

Goals for the meeting included:

  • Establishing working groups with members from a variety of backgrounds, including clinicians and clinical trialists, and people with varying expertise, including psychometrics, pharmaceuticals, and advocates
  • Identifying the concepts and domains to be measured
  • Identifying the currently available assessments
  • Assessing potential instruments for reliability, validity, and ability to detect change in the domain to be measured
  • Rating the existing measures and categorizing them by those that are ready to use, those that would need modification before they could be used, and those that should not be used

More Information


Dr. Tiina Urv, IDDB, DER, NICHD
Phone: 301-402-7015
Email: tu36j@nih.gov

Lisa Kaeser, OD, NICHD
Phone: 301-49g-0536
Email: kaeserl@mail.nih.gov

Please note: Views expressed during NICHD-sponsored events do not necessarily reflect the opinions or the official positions of NICHD, NIH, or HHS.
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