Refining clinical trial inclusion criteria to optimize the standardized response mean of the CMTPedS
Kayla M D Cornett1, Manoj P Menezes2, Paula Bray1
1School of Health Sciences, University of Sydney, The Children's Hospital at Westmead, Sydney, New South Wales, Australia.
Insights
The CMT Pediatric Scale (CMTPedS) effectively measures disability in children with Charcot-Marie-Tooth disease (CMT). Optimizing trial criteria for younger, mildly affected CMT1A patients enhances CMTPedS responsiveness.
Area of Science:
- Neurology
- Clinical Trials
- Pediatric Research
Background:
- The CMT Pediatric Scale (CMTPedS) is an established measure for childhood CMT disability.
- Optimizing outcome measures is crucial for efficient clinical trials in rare pediatric diseases.
Purpose of the Study:
- To identify patient subsets demonstrating maximal responsiveness using the standardized response mean (SRM).
- To refine CMTPedS application as a primary outcome measure in future CMT clinical trials.
Main Methods:
- Analysis of 2-year natural history data from 187 children (aged 3-20 years) with diverse CMT subtypes.
- Calculation of SRM for CMTPedS across various patient demographic and clinical strata.
Main Results:
- Subgroup analysis revealed significantly increased CMTPedS responsiveness in younger children (3-8 years).
- Mildly affected patients (CMTPedS score 0-14) and those with CMT1A showed heightened responsiveness.
- These specific subsets considerably improved the SRM of the CMTPedS.
Conclusions:
- Refining clinical trial inclusion criteria to focus on younger, less affected CMT1A patients optimizes CMTPedS responsiveness.
- This optimization enhances the utility of CMTPedS as a primary outcome measure for pediatric CMT trials.
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.
