TODDLER Study v1.0
Research type
Research Study
Full title
Transforming Outcomes in Duchenne muscular dystrophy using DigitaL Endpoints Remotely
IRAS ID
358351
Contact name
Fiona Moultrie
Contact email
Sponsor organisation
University of Oxford Research Governance, Ethics and Assurance
Duration of Study in the UK
2 years, 6 months, 1 days
Research summary
Every year, 100 boys are born in the UK with Duchenne muscular dystrophy. These boys cannot make an important muscle protein. They become weaker as they get older and lose the ability to walk as teenagers. There is no cure, but medicines are being made that could help. These medicines will likely work better in younger boys, before muscle damage begins.
We currently have no way of testing these medicines in children under four. In older children, we test how well a children perform certain movements an skills. These tests are not really suitable for toddlers as they can struggle to listen and perform tasks on demand. We have a new way to test how well children move. Children can wear a special watch-like device on their ankles that records information about their steps as they go about their normal lives. This can tell us their maximum walking ability. It is now used to test medicines in children over four years old. Our aim is to test whether this can be used in children under four and how it compares to other movement tests.
We will invite boys with DMD (and their parent/caregiver) and healthy boys aged 1-3 years old to join the study. There are no hospital visits. Children will receive devices to wear daily at home for three blocks of 28-days over six months. At the beginning and end of the study, a physiotherapist will visit the homes of boys with DMD to assess their movements with other tests and their development.
We will find out 1) if young boys are happy to wear the device, 2) how it compares to other tests, and 3) if it can detect early changes in walking ability. This study could give us a way to test medicines in younger children with DMD.
REC name
Yorkshire & The Humber - Leeds West Research Ethics Committee
REC reference
25/YH/0261
Date of REC Opinion
18 Dec 2025
REC opinion
Favourable Opinion