Characterization of potential outcome measures for future clinical trials in fragile X syndrome

Elizabeth Berry-Kravis, Allison Sumis, Ok Kyung Kim, Rebecca Lara, Joanne Wuu

Research output: Contribution to journalArticlepeer-review

19 Scopus citations

Abstract

Clinical trials targeting recently elucidated synaptic defects in fragile X syndrome (FXS) will require outcome measures capable of assessing short-term changes in cognitive functioning. Potentially useful measures for FXS were evaluated here in a test-retest setting in males and females with FXS (N = 46). Good reproducibility, determined by an interclass correlation (ICC) or weighted kappa (κ) of 0.7-0.9 was seen for RBANS List and Story Memory, NEPSY Tower, Woodcock-Johnson Spatial Relations and the commissions score from the Carolina Fragile X Project Continuous Performance Test (CPT). This study demonstrates the feasibility of generating test profiles containing reliability data, ability levels required for test performance, and refusal rates to assist with choice of outcome measures in FXS and other cohorts with cognitive disability.

Original languageEnglish (US)
Pages (from-to)1751-1757
Number of pages7
JournalJournal of Autism and Developmental Disorders
Volume38
Issue number9
DOIs
StatePublished - Oct 1 2008
Externally publishedYes

Keywords

  • Clinical trials
  • fMR1
  • Fragile X syndrome
  • Outcome measures

ASJC Scopus subject areas

  • Developmental and Educational Psychology

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