Journal of Autism and Developmental Disorders

, Volume 38, Issue 9, pp 1751–1757

Characterization of Potential Outcome Measures for Future Clinical Trials in Fragile X Syndrome

Authors

    • Department of PediatricsRush University Medical Center
    • Department of Neurological SciencesRush University Medical Center
    • Department of BiochemistryRush University Medical Center
  • Allison Sumis
    • Department of PediatricsRush University Medical Center
    • Department of Molecular and Cellular Oncology
  • Ok-Kyung Kim
    • Rush University School of Medicine
  • Rebecca Lara
    • Department of PediatricsRush University Medical Center
    • Pritzer School of MedicineChicago University
  • Joanne Wuu
    • Department of Neurological SciencesRush University Medical Center
    • Department of NeurologyEmory University
Original Paper

DOI: 10.1007/s10803-008-0564-8

Cite this article as:
Berry-Kravis, E., Sumis, A., Kim, O. et al. J Autism Dev Disord (2008) 38: 1751. doi:10.1007/s10803-008-0564-8

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.

Keywords

Fragile X syndrome Clinical trials Outcome measures FMR1

Copyright information

© Springer Science+Business Media, LLC 2008