Feasibility of using a wearable digital health technology sensor to assess ambulation in DM1 and CMT

Topic:

Clinical Trials

Poster Number: O93

Author(s):

Laurent Servais, MD, PhD, MDUK Oxford Neuromuscular Centre & NIHR Oxford Biomedical Research Centre, Oxford, UK, Guillaume Parinello, Msc, SYSNAV, Vernon, France, Margaux Poleur, MD, University department of neurology, CHR Citadelle, Liège, Belgium, Stéphanie Delstanche, MD, University department of neurology, CHR Citadelle, Liège, Belgium, Camille Bisson, SYSNAV, Vernon, France, Celine Cluzeau, PhD, SYSNAV, Vernon, France, Charline Dubois, PhD, University department of neurology, CHR Citadelle, Liège, Belgium, Manon Duclos, University department of neurology, CHR Citadelle, Liège, Belgium, Laurie Médard, University department of neurology, CHR Citadelle, Liège, Belgium, damien eggenspieler, PhD, sysnav

The European Medicines Agency qualified stride velocity 95th centile (the 5% most rapid strides) as a primary endpoint in Duchenne muscular dystrophy. This sparked interest in using this wearable digital health technology (wDHT) in diseases where ambulation is affected, like Charcot-Marie-Tooth disease (CMT) and Myotonic Dystrophy Type 1 (DM1), two inherited neuromuscular disorders characterized by progressive weakness and atrophy of the distal limb muscles. SV95C and other distal weakness-specific endpoints could provide a representative assessment of patients’ motor function in daily life, in diseases which lack objective and reliable endpoints to evaluate drug efficacy.

To determine the feasibility of using wDHT, patients with DM1 and CMT were enrolled in the 3-year ActiLiege-Next study. Patients are clinically evaluated every 6 months, and wear one sensor at each ankle daily for the first 3 months and then for 1 month every 6 months. We will investigate a portfolio of digital outcomes including SV95C and other variables (like walking perimeter, number of strides per hour…), including ones that could better evaluate distal weakness like median foot elevation during the stride.

Fourteen patients with DM1 (median age +/- standard deviation [range]: 36+/-17yo [5-71]) and 11 patients with CMT (42+/-25yo [14-84]) were enrolled so far. To date, patients were adherent to wearing ankle sensors: all but one patients recorded ≥50 hours at baseline (11/12 DM1 and 10/10 CMT), the minimum required to compute wDHT-derived variables. Data analysis is ongoing and preliminary results on reliability and construct validity of wDHT-derived variables will be presented at the congress.

Preliminary feasibility evaluation in CMT and DM1 indicates that patients can collect enough data to allow computation of gait variables, confirming that wDHT could be useful in muscle diseases and neuropathies. We will present the full baseline data and first longitudinal data in patients with 6 months of follow-up.