Abstract

BackgroundNormative data are necessary for validation of new outcome measures. Recently, the 95th centile of stride speed was qualified by the European Medicines Agency as a valid secondary outcome for clinical trials in subjects with Duchenne muscular dystrophy. This study aims to obtain normative data on spontaneous stride velocity and length in a non-controlled environment and their evolution after 12 months.MethodNinety-one healthy volunteers (50 females, 41 males), with a mean age of 16 years and 2 months, were recruited and assessed at baseline and 12 months later. The 4-stair climb, 6-min walk test, 10-m walk test and rise from floor assessments were performed. Stride length, stride velocity, and the distance walked per hour were studied in an everyday setting for one month after each evaluation.ResultsOf the 91 subjects assessed, 82 provided more than 50 h of recordings at baseline; and 73 subjects provided the same at the end of the year. We observed significant positive correlations of the stride length with age and height of participants, and a significant increase of the median stride length in children after the period. In this group, the 95th centile stride velocity was not correlated with age and was stable after one year. All measures but the 10MWT were stable in adults after a one-year period.ConclusionThis study provides with data on the influence of age, height, and gender on stride velocity and length as well as accounting for natural changes after one year in controls.

Highlights

  • Normative data are necessary for validation of new outcome measures

  • The 95th centile stride velocity was not correlated with age and was stable after one year

  • We found a similar pattern in stride length measures: the median (SL50C) was higher in adults (p = 0.001) but the 95th centile (SL95C) was higher in children (p = 0.001)

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Summary

Introduction

Normative data are necessary for validation of new outcome measures. Recently, the 95th centile of stride speed was qualified by the European Medicines Agency as a valid secondary outcome for clinical trials in subjects with Duchenne muscular dystrophy. Duchenne muscular dystrophy (DMD) is a severe, rapidly progressive neuromuscular disorder characterized by muscle weakening. It has an estimated incidence of 1 in 5000 males [1]. To accelerate clinical development and investigate in parallel several approaches without being limited by the number of patients available, it is crucial to validate more powerful outcome measures. This is the case for DMD and for numerous other rare diseases, within or outside the neuromuscular field

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