The importance and use of Syde® wearable technology and Stride Velocity 95th Centile (SV95C) endpoint in Duchenne muscular dystrophy

    Principal Author: Paul Strijbos

    Several studies in ambulant patients with Duchenne, including studies of the gene therapy delandistrogene moxeparvovec (SRP-9001) (EMBARK; Study 301), anti-myostatin RG6206 (SPITFIRE), gene therapy SGT-001 (IGNITE) and oligonucleotide suvodirsen (DYSTANCE 51), use(d) Stride Velocity 95th Centile (SV95C) or the minimal velocity of the 5% most rapid strides taken by a patient as a secondary efficacy endpoint.

    In April 2019, SV95C was qualified by the European Medicines Agency as an acceptable secondary efficacy endpoint in clinical trials of ambulant patients with Duchenne aged ≥5 years when measured using a validated and suitable wearable device (such as the ankle-worn movement sensor Syde®, formerly ActiMyo) that can quantify ambulation directly and continuously in a real-world environment. Development of SV95C as an endpoint for Duchenne is an example of the synergy garnered from the collaboration of multiple stakeholders, including the key perspective of patients.

    Designing clinical trials based on patient needs and preferences can improve participation and retention, which can in turn bolster the development of new therapies. The perspectives of patient advocacy group (PAG) representatives, patients and caregivers were considered during the development of Syde® and SV95C. We present how SV95C is used in EMBARK and detail activities (including community advisory boards, collaborative events, PAG workshops and PAG authorship on SV95C-related publications) between 2018 and 2022 that highlight the close collaboration with PAG groups to develop Syde® and SV95C. This type of partnership can streamline the development of “fit-for-purpose” and real-world use devices that balance precision, accuracy and reliability with user-friendliness for patients.

    Click here to download full poster.