RecruitingOBSERVATIONAL

A Remote Study Using Technology to Assess Outcomes in DMD

This study is exploring a new way to measure movement in young boys, aged 1 to 3 years old, who have Duchenne muscular dystrophy (DMD) and in healthy boys of the same age. DMD is a condition that causes muscles to weaken over time. Currently, it's hard to test new medicines in very young children because standard movement tests aren't suitable for toddlers. This study will use small, watch-like devices worn on the ankle to track children's steps during their daily lives. Researchers want to find out if these devices are comfortable for young boys to wear, how well they compare to other movement tests, and if they can show changes in walking ability. This could lead to a better way to test new medicines for DMD earlier, potentially making treatments more effective and reducing the need for stressful hospital visits for families.

At a glance

Status

Recruiting

Sponsor

University of Oxford

Enrolment target

Start

24 Jun 2026

Estimated completion

01 Jul 2028

Duchenne Muscular Dystrophy (DMD)

What is this study about?

Duchenne muscular dystrophy (DMD) is a rare but serious condition that affects boys, causing their muscles to gradually weaken. Sadly, there is no cure for DMD, but new medicines are being developed that might help. These medicines are likely to work best when given to very young children, before their muscles are too damaged. However, testing new medicines in children under four is difficult because the usual ways of measuring movement, like asking a child to run or stand up quickly, don't work well for toddlers who might not understand or consistently do what's asked. Tiredness or mood can also affect their results.

This study hopes to find a better way by using special watch-like devices that children wear on their ankles. These devices measure how they move during their everyday activities, like playing and walking. This kind of device is already used for older children, but this study wants to see if it works well for boys aged 1 to 3 years old. The researchers want to know if young boys are happy to wear the device, if the device measurements match up with other movement tests, and most importantly, if the device can detect small changes in their walking ability over time.

If successful, this study could make a big difference. It could give us a valuable tool to test new DMD medicines in younger children, which could lead to more effective treatments sooner. It could also mean fewer stressful visits to hospitals for families, as much of the testing could be done at home. This would also allow more children, including those with learning difficulties or living far from research centers, to take part in future studies, bringing us closer to finding better ways to manage this condition.

Key takeaways

Tests new watch-like devices for measuring movement in 1-3 year old boys.
Aims to improve how new Duchenne muscular dystrophy (DMD) medicines are tested.
Involves boys with DMD and healthy boys.
Most of the study is done at home, with no hospital visits for device wearing.
Could help make future DMD studies more accessible and less stressful.
Participation lasts six months in total, with devices worn for three 28-day periods.

Who may be eligible?

This study is looking for two groups of boys aged between 1 and 3 years old. The first group includes boys who have been diagnosed with Duchenne muscular dystrophy (DMD). For these boys to take part, they need to be able to walk by themselves for at least 10 meters, and their parent or legal guardian must be over 18, willing to give permission, and able to help with the study tasks.

The second group is healthy boys, also aged 1 to 3 years old, who can walk by themselves for at least 10 meters. Their parent or legal guardian must also be over 18, willing to give permission, and able to help. It's important for both groups that the parents can understand all the study information.

There are some reasons why a boy might not be able to join. For instance, if he's had recent surgery or an injury to his leg within the last six months, or has other serious health problems that affect his movement. Boys with DMD who are already taking certain steroid medications or are part of another treatment study would also not be able to participate. For healthy boys, any major health conditions affecting movement or delays in development would mean they couldn't join.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is your child a boy aged between 1 and 3 years old?
Can your child walk by himself for at least 10 meters?
If your child has DMD, are they currently not taking specific steroid medications for DMD?
Are you, as the parent or guardian, over 18 and able and willing to help with the study tasks?
Has your child NOT had any leg surgery or serious leg injury in the last 6 months?

Answer every question to see your result.

What does participation involve?

If you decide to take part, your child will wear small, watch-like devices on their ankles. They will wear these devices for three separate periods, each lasting 28 days, over a total of six months. Your child will wear the devices during his normal daily activities at home – there are no hospital visits for this part of the study.

For boys with DMD, a physiotherapist will visit your home at the very beginning and at the end of the six-month study. During these visits, the physiotherapist will check your child's movements using other standard tests. You, as the parent or caregiver, will also be asked to help with some remote assessments, likely involving some questionnaires or observations you can complete at home. The total duration of active participation for your child will be six months, but the device-wearing periods are spread out within that time.

Potential risks and benefits

Potential benefits of participating include helping researchers understand how to better measure movement in young children with DMD, which could lead to faster development of new medicines and less stressful testing for families in the future. It could also help children who usually can't join studies to take part. There are very few risks expected; the watch-like devices are safe to wear, and wearing them should not cause any discomfort. The home visits for boys with DMD would involve standard movement checks by a professional. You are free to withdraw your child from the study at any time, for any reason, without it affecting your child's medical care.

Locations (1)

Oxford University
Verified postcode
Oxford, United Kingdom· Recruiting

Common questions

What is Duchenne muscular dystrophy (DMD)?

DMD is a rare genetic condition that causes muscles to become weaker over time because the body can't make an important protein for muscles.

What will my child wear in the study?

Your child will wear small, watch-like devices on their ankles that measure their movements during normal daily activities.

Do we need to go to the hospital?

No, for the device-wearing part of the study, there are no hospital visits. For boys with DMD, a physiotherapist will visit your home twice.

How long does the study last for my child?

Your child will wear the devices for three separate periods of 28 days each, spread out over six months.

What is the main goal of this study?

The main goal is to find a good way to measure movement in very young boys, so new medicines for DMD can be tested earlier and more easily.

How to find out more

Fiona Moultrie

fiona.moultrie@paediatrics.ox.ac.uk +44 (0)1865 618799 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.