AI Cure is a research tool showing open data. Not medical advice. Discuss with your doctor. Terms

โ† Trials
๐Ÿงช

A Remote Study Using Technology to Assess Outcomes in DMD

Recruitingn/aAges 1 Year - 3 YearsMaleUniversity of Oxford
๐Ÿ’ฌ

In plain words

What the trial is, and whether it could suit you

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 do. Tiredness and mood can also affect their scores. Luckily, there is a new way of testing how well

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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 10m independently) * Parent(s)/legal guardian Exclusion Criteria: Participant with DMD: * Limb surgery/trauma (within 6 months) * Significant comorbid chronic or acute conditions affecting motor function (within 3 weeks) * Prematurity (born \<37 weeks' gestation) * Oral corticosteroids to treat DMD (before enrolment) * Enrolment in therapeutic clinical trials * Any comorbidity which could limit their ability to complete the study assessments (according to the investigator's clinical judgement) Healthy Control participant: * Limb surgery/trauma (within 6 months) * Significant comorbid chronic condition affecting motor function * Significant acute condition affecting motor function (within 3weeks of enrolment) * Prematurity (born \<37 weeks' gestation) * Neurodevelopmental concerns or delay in acquisition of WHO developmental milestones. * Any comorbidity which could limit their ability to complete the study assessments (according to the investigator's clinical judgement).
๐Ÿ’Š

What it tests

  • Not listed
๐Ÿ“

Where

1 site
Oxford University
Oxford, United Kingdom
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
๐Ÿ›ก๏ธ

AI Cure shows public evidence (Open Targets, ClinicalTrials.gov, PubMed, openFDA, DepMap, AlphaFold) with its source, and AI summaries that cite it โ€” not medical advice and not a cure. Treatment decisions belong with your doctor. Research ideas are shared only with approved researchers and labs, who can test them. In an emergency call your local emergency number (112 in India and Europe, 911 in the US). AI Cure Terms of Use ยท Privacy