Home-based gait analysis as an exploratory endpoint during a multicenter phase 1 trial in limb girdle muscular dystrophy type R2 and facioscapulohumeral muscular dystrophy
| Autor: | Erwan Gasnier, David Vissiere, Laurent Servais, Tahseen Mozaffar, Gennyne Walker, Kathryn R. Wagner, John Vissing, Teresa Gidaro, M. Annoussamy, Sanjay S. Shukla, Stanley Iyadurai, Shahram Attarian |
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| Rok vydání: | 2021 |
| Předmět: |
musculoskeletal diseases
medicine.medical_specialty Physiology STRIDE Walking Cellular and Molecular Neuroscience Physical medicine and rehabilitation Physiology (medical) medicine Facioscapulohumeral muscular dystrophy Humans Gait business.industry Stride length medicine.disease Home based Muscular Dystrophy Facioscapulohumeral Clinical trial Muscular Dystrophies Limb-Girdle Gait analysis Neurology (clinical) business Gait Analysis human activities Limb-girdle muscular dystrophy |
| Zdroj: | MusclenerveREFERENCES. 65(2) |
| ISSN: | 1097-4598 |
| Popis: | INTRODUCTION/AIMS Limb girdle muscular dystrophy type 2B (LGMDR2) and facioscapulohumeral muscular dystrophy (FSHD) are genetic muscular dystrophies with an increasing number of potential therapeutic approaches. The aim of this study is to report the data of exploratory digital outcomes extracted from wearable magneto-inertial sensors used in a non-controlled environment for ambulant patients with FSHD and LGMDR2 in a short-term, multicenter clinical study. METHODS Digital outcomes (stride length, stride speed, and walk parameters in a non-controlled environment) were used as exploratory outcomes in the open-label study ATYR1940-C-004 in ambulant patients during the 3 mo of ATYR1940 treatment and 1 mo of follow-up. Activity and gait variables were calculated from the data recorded in 30-day sub-periods using the sensors. For each sub-period, activity and gait parameters were compared between FSHD and LGMDR2 patients. Change from baseline over the 4-mo study period was assessed. RESULTS Ten patients (5 FSHD, 5 LGMDR2) were ambulant and compliant for analysis. Gait parameters, but not activity variables, were significantly lower in LGMDR2 compared to FSHD patients at baseline. Longitudinal analyses showed a slight but significant decrease in stride speed at month 4 for all subjects. Activity variables such as total number of strides per day were highly variable from month to month in individual patients, and no visit effects were found for this variable. DISCUSSION The present study suggests that home-recorded stride speed constitutes a precise and sensitive outcome in ambulant patients with FSHD and LGMDR2. |
| Databáze: | OpenAIRE |
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