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Neuromuscul Disord. 2019 Jul;29(7):514-516. doi: 10.1016/j.nmd.2019.06.003. Epub 2019 Jun 7.

European regulators' views on a wearable-derived performance measurement of ambulation for Duchenne muscular dystrophy regulatory trials.

Author information

1
Federal Institute of Drugs and Medical Devices, Kurt-Georg-Kiesinger-Allee 3, 53175 Bonn, Germany.
2
European Medicines Agency, Domenico Scarlattilaan 6, 1083 HS Amsterdam, the Netherlands.
3
World Duchenne Organization /UPPMD, European Organisation for Rare Diseases - Eurordis, MDA Hellas, Elpidos 6, Athina 104 34, Greece.
4
Departamento de Farmacología y Toxicología, Facultad de Medicina, Universidad Complutense, Plaza de Ramon y Cajal s/n, 28040 Madrid, Spain.
5
Medicines Evaluation Board, Graadt van Roggenweg 500, 3531 AH Utrecht, the Netherlands.
6
Laboratório de Farmacologia Clínica e Terapêutica, Faculdade de Medicina da Universidade de Lisboa, Av. Prof. Egas Moniz, 1649-028 Lisboa, Portugal.
7
National Center for Drug Research and Evaluation, Istituto Superiore di Sanità, Viale Regina Elena, 299, 00161 Roma, Italy. Electronic address: armando.magrelli@iss.it.

Abstract

Development of novel therapies for Duchenne muscular dystrophy (DMD) are driving the need for more efficient ways of detecting changes in disease- progression in DMD [1]. However, medicines' approval must be based on outcome measures that are acceptable from a regulatory perspective. In this article, European regulators provide an update on the recent regulatory consideration of a new endpoint (Stride Velocity 95th Centile (SV95C)) that could be used in therapeutic DMD trials. This new endpoint aims to quantify a patient's ambulation directly, reliably and continuously in a home environment with a wearable device.

KEYWORDS:

Clinical trial endpoints; Duchenne muscular dystrophy; European regulators; Gait Variables

PMID:
31272741
DOI:
10.1016/j.nmd.2019.06.003
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