Annals of Clinical and Translational Neurology (Sep 2020)

Refining clinical trial inclusion criteria to optimize the standardized response mean of the CMTPedS

  • Kayla M. D. Cornett,
  • Manoj P. Menezes,
  • Paula Bray,
  • Rosemary R. Shy,
  • Isabella Moroni,
  • Emanuela Pagliano,
  • Davide Pareyson,
  • Tim Estilow,
  • Sabrina W. Yum,
  • Trupti Bhandari,
  • Francesco Muntoni,
  • Matilde Laura,
  • Mary M. Reilly,
  • Richard S. Finkel,
  • Katy J. Eichinger,
  • David N. Herrmann,
  • Michael E. Shy,
  • Joshua Burns,
  • CMTPedS Study Group

DOI
https://doi.org/10.1002/acn3.51145
Journal volume & issue
Vol. 7, no. 9
pp. 1713 – 1715

Abstract

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Abstract The CMT Pediatric Scale (CMTPedS) is a reliable, valid, and responsive clinical outcome measure of disability in children with CMT. The aim of this study was to identify the most responsive patient subset(s), based on the standardized response mean (SRM), to optimize the CMTPedS as a primary outcome measure for upcoming clinical trials. Analysis was based on a 2‐year natural history data from 187 children aged 3–20 years with a range of CMT genetic subtypes. Subsets based on age (3–8 years), disability level (CMTPedS score 0–14), and CMT type (CMT1A) increased the SRM of the CMTPedS considerably. Refining the inclusion criteria in clinical trials to younger, mildly affected cases of CMT1A optimizes the responsiveness of the CMTPedS.