A Remote Study Using Technology to Assess Outcomes in DMD
Every year, 100 boys are born in the UK with a rare muscle disease called Duchenne muscular dystrophy. These boys cannot make an important muscle protein called dystrophin. They become weaker as they get older and lose the ability to walk as teenagers. This is a life-limiting condition. There is no cure, but medicines
| Condition(s) | Duchenne Muscular Dystrophy (DMD) |
|---|---|
| Status | Recruiting |
| Study type | Observational |
| Summary | Every year, 100 boys are born in the UK with a rare muscle disease called Duchenne muscular dystrophy. These boys cannot make an important muscle protein called dystrophin. They become weaker as they get older and lose the ability to walk as teenagers. This is a life-limiting condition. There is no cure, but medicines are being made that could help these boys make dystrophin. These medicines are most likely to work best in toddlers, before their muscles become damaged. There is no way of testing these medicines in children under four. In older children, it is possible to measure how well and how quickly a child can do movements like sitting up, standing up, and running. Unfortunately, these tests are not suitable for toddlers as they often struggle to listen and do what they are asked to d |
| Who can participate | Inclusion Criteria: Participant with DMD: * Male * Aged 1-3 years old * Ambulant (walking 10m independently) * Genetically confirmed diagnosis of DMD * Parent(s)/legal guardian(s) able and willing to provide written informed consent for the child to participate in the study * Parent(s)/legal guardian(s) able and willing to participate in the study Parent/legal guardian of participant with DMD: * Aged 18 years or more * Legal carer of the patient diagnosed with DMD * Willingness to follow study procedures and assist with remote assessments, as assessed by the research team * Willingness to sign the consent form * Ability to understand all the information with regards to the study, as assessed by the research team Healthy Control participant: * Male * Aged 1-3 years old * Ambulant (walking 1 |
| Ages | 1 Year to 3 Years |
| Sex | Male |
| Accepts healthy volunteers | Yes |
| Lead sponsor | University of Oxford |
| Locations | Oxford, United Kingdom |
| Start date | 2026-06-24 |
| NCT ID | NCT07423026 |
| Official listing | https://clinicaltrials.gov/study/NCT07423026 |