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Abstract Details

Digital Outcome Measure Assessing the Motor Function of Patients with Amyotrophic Lateral Sclerosis
Neuromuscular and Clinical Neurophysiology (EMG)
P5 - Poster Session 5 (5:30 PM-6:30 PM)
11-011
To validate the feasibility, reliability, external validity and the sensitivity to change of Stride Velocity 95th centile (SV95C) in non-controlled environment in ambulant individuals with Amyotrophic Lateral Sclerosis (ALS)

Progressive loss of ambulation is one of the main disability causes in ALS. In Duchenne Muscular Dystrophy, the 95th centile of stride velocity (SV95C) as measured by a magneto-inertial device specifically designed for continuous assessment of patients with neurological diseases (ActiMyo®) in uncontrolled environment has been qualified as a primary endpoint by European Medical Agency and is currently considered by FDA.

Longitudinal multisite natural history study in ALS. Participants were assessed with standard evaluations including the ALS functional rating scale, six-minute walk test, Medical Research Council scores (MRC-S), Ashworth score and hand dynamometer every 3 months for 1 year. After each visit, patients were asked to wear for one month one inertial sensor at the ankle and one at the wrist for continuous assessment of motor function in real-life. These data are compared with already acquired normative data in matched healthy controls

We included 19 patients with ALS (6 females and 13 males) aged from 20 to 70 years. Eighteen of them (94%) provided a recording period over 50 hours at baseline.

On the first 8 patients aged from 44 to 68 years, reliability was excellent with an inter-class correlation of 0.98. Correlation with MRC-S was 0.46 (p<0.05). ICC was 0.98. All patients declined rapidly with a standardized response mean of -2.78 at month 3 and -2.63 at months 6. We will present the entire set of data.

If confirmed on the entire cohort- these data demonstrate the excellent metric property of SV95C in ALS, which allows to consider short clinical trials with limited number of patients.

Authors/Disclosures
Laurent Servais (John Radcliffe Hospital)
PRESENTER
Laurent Servais has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biogen. Laurent Servais has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Roche. Laurent Servais has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Novartis. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Astellas. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Pfizer. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sarepta. The institution of Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Evox. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for PTC. Laurent Servais has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Sysnav. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for BioHaven. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Dyne. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Zentech. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MitoRx. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Lupin. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Fibrogen. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Alltrana. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Illumina. Laurent Servais has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Roche. The institution of Laurent Servais has received research support from Roche. The institution of Laurent Servais has received research support from Novartis. The institution of Laurent Servais has received research support from Biogen. The institution of Laurent Servais has received research support from Zentech. The institution of Laurent Servais has received research support from BioHaven. The institution of Laurent Servais has received research support from PerkinHalmers. The institution of Laurent Servais has received research support from Scholar Rock.
Margaux Poleur (Reference Center for Neuromuscular Diseases) No disclosure on file
Ahmed Nemr (Maadi Military Hospitals) Ahmed Nemr has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Nvartis. Ahmed Nemr has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Merk. Ahmed Nemr has received personal compensation in the range of $0-$499 for serving on a Speakers Bureau for Iansen.
Léopold BANCEL (SYSNAV) No disclosure on file
Guillaume Parinello (Sysnav) No disclosure on file
Celine Cluzeau (Sysnav) No disclosure on file
Yacine Bechichi (Sysnav) No disclosure on file
Stephanie Delstanche (CHR Citadelle) No disclosure on file