Astellas STEP study V1.0
Research type
Research Study
Full title
A Non-Interventional Pilot Study of Adult Ambulant Participants with a Confirmed Diagnosis of Late Onset Pompe Disease (LOPD) to Validate the Stride Velocity 95th Centile (SV95C) Using the Syde® Device
IRAS ID
368215
Contact name
Mark Eldon Roberts
Contact email
Sponsor organisation
Astellas Pharma Global Development, Inc
Duration of Study in the UK
0 years, 9 months, 9 days
Research summary
Pompe disease is a rare inherited condition that causes progressive muscle weakness. It occurs when the body has little or none of an enzyme needed to break down glycogen, a form of stored sugar. As glycogen builds up in the muscles, they become weaker over time. Pompe disease is present from birth due to a change in a gene passed down through families. It is not contagious, and nothing a person did caused it. Symptoms can appear at any age and may include difficulty walking, climbing stairs, or breathing. In infants, it can also affect the heart. There is currently no cure, but enzyme replacement therapy (ERT) and supportive care can help manage symptoms and slow progression.
As new treatments for Pompe disease are developed, researchers need reliable ways to measure whether these treatments are effective. Currently, the 6 minute walk test (6MWT) is commonly used to assess changes in walking ability. However, this test can be challenging for some people particularly those diagnosed as adults and may not always reflect everyday movement.
This study will explore whether alternative ways of measuring activity and mobility may be as good as, or better than, the 6MWT. It will use a wearable device called the Syde device, which is designed for people with muscle conditions. The device is worn on the ankles (or, in some cases, on a wrist and ankle) throughout the day and records movement during normal activities.
The aim is to understand whether the Syde device can help detect changes in daily movement that might show whether a future treatment is working. Participants in this study will not receive any treatment; instead, their involvement will help researchers determine whether the Syde device is a useful and less burdensome tool for measuring mobility in people with Pompe disease.
REC name
Yorkshire & The Humber - Bradford Leeds Research Ethics Committee
REC reference
26/YH/0066
Date of REC Opinion
15 Apr 2026
REC opinion
Further Information Favourable Opinion