Remote monitoring of motor development in newborn-identified children with SMA or DMD
Official title: Active-NBS Liege - Monitoring the Motor Development of Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified Through Newborn Screening
Run by Centre Hospitalier Universitaire de Liege · NCT07286565 · Data updated 2026-11-12
This project will try wearable sensors at home to see if they can track motor development in infants and toddlers diagnosed with spinal muscular atrophy or Duchenne muscular dystrophy through newborn or family screening.
Quick facts
| Study type | Interventional |
|---|---|
| Ages | 4 Months and up |
| Sex | All |
| Enrollment | 100 (estimated) |
| Sponsor | Centre Hospitalier Universitaire de Liege Academic / other |
| Locations | 1 site (Liège) |
| Status | Recruiting |
| Started | December 1, 2025 |
| Main results expected | August 2028 |
| Study ends | August 2028 |
| Registry record updated | December 16, 2025 |
| Trial ID | NCT07286565 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are infants or toddlers with genetically confirmed SMA (with documented SMN2 copy number) or DMD identified by newborn or family screening or incidental pre-symptomatic diagnosis, whose legal guardian can consent and who have home internet access.
✕Probably not a fit
Children with advanced symptomatic disease, significant comorbidities that interfere with motor development, those enrolled in therapeutic intervention trials, or families without reliable internet access are unlikely to benefit from this remote-monitoring project.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This monocentric, fully remote observational project will enroll about 100 children identified by newborn screening, family testing, or incidental genetic diagnosis and follow them for up to 30 months. Families complete consent, questionnaires, and follow-up visits by phone or video and children wear age-appropriate devices at home (MAIJU for non-ambulant infants and Syde ankle sensors for ambulant children). The team will collect continuous wearable data, standard clinical information, and PedsQL quality-of-life questionnaires to derive and validate digital motor endpoints. There are no required hospital visits and data are analysed centrally to map early motor development trajectories.
Why it matters
Potential benefit: If successful, this approach could enable earlier, less burdensome detection of motor changes and support timely treatment decisions for infants and young children with SMA or DMD.
How similar studies have gone: Previous small studies using wearable sensors in neuromuscular disorders have shown feasibility and promising signals, but validation specifically in newborn-identified, pre-symptomatic SMA and DMD cohorts is still limited.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: Genetically confirmed SMA and avalaible MSNA2 copy number: * Identified by newborn screening, * Identified by family screening, or incidental diagnosis in pre-symptomatic stage * Treated (or follow-up possible for patients with 4 SMN2 copies) Genetically confirmed DMD: * Identified by newborn screening, * Identified by family screening, or incidental diagnosis in pre-symptomatic stage * Age \< 4 years at inclusion Legal guardian able to provide informed consent Exclusion Criteria: * Any acute or chronic condition that, in the investigator's opinion, significantly interferes with assessments and/or motor development. * Participation in a therapeutic trial. * Lack of internet connection.
Where it's running
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Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle
Liège, Belgium Recruiting
Study contacts
- Principal investigator: Tamara Dangouloff, PhD — University of Liege
- Study coordinator: Tamara Dangouloff, PhD
- Email: tamara.dangouloff@uliege.be
- Phone: +33662438138
Similar studies
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.