Remote Monitoring Study Using Wearable Technology to Measure Movement in Boys Aged 1 to 3 with Duchenne Muscular Dystrophy

What this Trial Is About

Duchenne muscular dystrophy DMD is a rare genetic muscle disease affecting boys who cannot produce a vital muscle protein called dystrophin. This leads to progressive muscle weakness, loss of walking ability in teenage years, and eventually premature death. While treatments exist for older children, there are currently no reliable ways to measure motor function or assess new medicines in toddlers under four years old, who may benefit most from early interventions. This study invites 30 boys with DMD and 30 healthy boys aged 1 to 3 years to wear special watch-like devices called Syde on their ankles to track their walking ability during normal daily activities. The recording periods last for three 28-day blocks over six months. Physiotherapists will visit the homes of boys with DMD at the start and end of the study to perform additional motor function tests. The study aims to evaluate how well boys tolerate wearing the device, how its measurements compare to other tests, and whether it can detect changes in walking ability over time. Participants will be monitored remotely with no hospital visits required. Assessments include continuous movement tracking, physiotherapist evaluations, and parent-reported outcomes. Researchers will analyze the devices reliability, variability, compliance, and acceptability. This approach could reduce travel and stress for families, include children who previously could not participate due to behavioral or developmental challenges, and provide a new way to measure treatment effects in younger boys with DMD. The total study duration for each participant is six months.

A Remote Study Using Technology to Assess Outcomes in DMD