A Remote Study Using Technology to Assess Outcomes in DMD

NCT07423026 | Recruiting

• 1 other identifier: TODDLER
• Study Type: observational
• Target: 60
• Locations: 1 country
• Sites: 1

Brief 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 do. Tiredness and mood can also affect their scores. Luckily, there is a new way of testing how well children move. They can wear special watch-like devices on their ankles that record information about their steps as they go about their normal lives. This is a good way of testing how well a child walks. It is now used to test medicines in children over four years old. Our aim is to test whether this device works well in children under four. This study will invite 30 boys with DMD (and their parent/caregiver) and 30 boys without DMD aged 1-3 years old from across the country to join the study. There are no hospital visits. Children will receive the watch-like devices to wear for three blocks of 28-days over six months during their normal daily activities. At the start and end of the study, a physiotherapist will visit the homes of boys with DMD. They will check their movements using other tests. The investigators 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 changes in walking ability. This study could give us a way to test medicines in younger children. Wearable devices could cut down the travel and stress of tests for boys and their families. Children with learning or behavioural difficulties, and children living far from research centres could now also take part in studies of new medicines. This study could bring us a step closer to treating this life-limiting disease.

Conditions

Duchenne Muscular Dystrophy (DMD)

Keywords

DMD; Devices; children

Outcome Measures

Primary Outcomes (4)

• To assess the within-patient variability in SV95C using the Syde device to remotely assess motor function in boys with DMD under the age of four.

  Within-patient variability in SV95C will be measured over 2 periods of 28 days using the Syde device on both ankles.

  Syde recording periods of 28 days at baseline, 1 month and 6 months.

• To assess the test-retest reliability of the Syde device to remotely assess motor function in boys with DMD under the age of four.

  Test-retest reliability will be measured using intra-class correlation coefficient (ICC) of consecutive measurements of SV95C.

  Two consecutive 28-day Syde recording periods at baseline and 1 month.

• To assess compliance with the Syde device in boys with DMD under the age of four.

  Compliance will be measured by percentage of participants completing minimum recording period of the Syde device.

  Syde recording periods of 28 days at baseline, 1 month and 6 months.

• To assess the acceptability of using the Syde device in boys with DMD under the age of four.

  Acceptability will be measured using the device acceptability questionnaire for Syde device.

  End of baseline and 6 month Syde recording periods.

Secondary Outcomes (5)

• To investigate whether SV95C recordings from the Syde device can distinguish boys with DMD from controls.

  28-day Syde recording periods at baseline, 1 month and 6 months.

• To assess correlation between SV95C as measure by the Syde device and other motor assessment and parent-reported outcomes.

  Month 1 and month 6 assessments.

• To assess impact of cognitive, behavioural, and language impairment on SV95C and other motor assessments.

  Month 1 and Month 6 assessments.

• To determine the sensitivity of SV95C to change.

  Baseline and Month 6 Syde recording periods.

• To assess the validity of remote online functional motor assessment using NSAA.

  Remote and in-person NSAA assessments at 1 month and 6 months.

Other Outcomes (2)

• To assess the reliability of other outcome measures from Syde device.

  Syde recording periods at baseline, 1 month and 6 months.

• To investigate the economic impact of DMD diagnosis, care and assessments on families of children under four for a future health economic analysis.

  Assessments at baseline and 6 months.

Study Arms (1)

Participant with DMD or Healthy Control

Cohort observation study of two groups undergoing the same procedures.

Eligibility Criteria

• Age: 1 Year - 3 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Participants with DMD and healthy controls within the UK, identified via advertisements and through clinic lists.

You may qualify if:

• 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

You may not qualify if:

• 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).

Sponsors & Collaborators

• University of Oxford: lead

Study Sites (1)

Oxford University

Oxford, United Kingdom

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Fiona Moultrie

CONTACT

+44 (0)1865 618799; fiona.moultrie@paediatrics.ox.ac.uk

Laurent Servais

CONTACT

+44 (0)1865 618799; laurent.servais@paediatrics.ox.ac.uk

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 5, 2026
• First Posted: February 20, 2026
• Study Start (Estimated): June 24, 2026
• Primary Completion (Estimated): October 1, 2027
• Study Completion (Estimated): July 1, 2028
• Last Updated: June 10, 2026

Record last verified: 2025-12

Locations

GB (1)