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Abstract Details

Holter of Movement Provides the First Digital Outcome Qualified by a Regulatory Agency
Neuromuscular and Clinical Neurophysiology (EMG)
ES2 - Emerging Science 2 (5:30 PM-5:36 PM)
001

Quantifying movement disorders is challenging, as clinical condition of patients fluctuates and as these assesments can be subjective and poorly sensitive to change.

Magneto-inertial technology allows to reconstruct precisely strides in 3D with centimetric performance.  This allows a precise and objective quantification of ambulation and gait of patients in their environment.

 

To gather regulatory qualification as a primary endpoint of the 95th centile of stride velocity, a digital outcome obtained by a wearable device. The context of use is ambulant patients with Duchenne Muscular Dystrophy

We have developed a wearable device to measure patients’ movements continuously and accurately in the uncontrolled environments of everyday life and a platform of clinical outcomes to summarize data into clinically meaningful indicators.One of these outcomes, the 95th centile stride velocity (SV95C), represents the 5% most rapid strides performed by a patient over a span of at least 50 hours.

SV95C properties were studied in 125 ambulant patients with Duchenne, aged 5 to 14 years, from 6 different natural history studies and clinical trials and from patients attending routine clinic appointments.

The median error of stride velocity in comparison with gold standard motion capture was measured with 0.01 cm/s precision. The reliability of the SV95C measured during a  > 50h period was 0.97. Compliance rate in uncontrolled environment was over 85%. The clear and rapid sensitivity to positive change in patients given steroids, as well as sensitivity to negative changes in patients from natural history significantly outperformed hospital-based assessments such as 6 minutes-walking-test.

On January 23rd 2023, the SV95C (Stride Velocity 95th centile) received positive opinion by the European Medical Agency to become a qualified primary endpoint for ambulant patients living with Duchenne Muscular Dystrophy, opening the way to public consultation. A similar application is ongoing at the FDA.

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 $10,000-$49,999 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. 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 as a Consultant for Scholar Rock. Laurent Servais has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Sanofi. 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.