NCT07286565Recruiting
Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn Screening
Active-NBS Liege - Monitoring the Motor Development of Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified Through Newborn Screening
Source: ClinicalTrials.gov · Last updated 16 Dec 2025
Brief summary (registry, in English)
The Active NBS Liege study is a monocentric, academic, fully remote, observational study designed to validate digital measures of motor development in children with spinal muscular atrophy (SMA) or Duchenne muscular dystrophy (DMD) identified through newborn screening, family testing, or incidental diagnosis. The study will enroll 100 children and follow them longitudinally for up to 30 months. Participants are remotely recruited, and all procedures, including consent, questionnaires, and follow-up visits, are conducted by phone or video conferencing without any hospital visits. Children will use age-appropriate wearable devices at home: MAIJU®, a sensorized garment for non-ambulant infants, and Syde®, an ankle-worn sensor for ambulant children. Data collection includes digital motor endpoints, clinical information, and quality of life (PedsQL). Primary objectives are to validate digital biomarkers of motor development, while secondary objectives include early identification of motor deficits, modeling motor trajectories, and quantifying genotype-related differences. Exploratory analyses will assess gait parameters such as stride velocity 95th centile (SV95C) and compare motor outcomes across genetic profiles and treatment exposure. Risks are minimal, limited to the use of non-invasive sensors with no known side effects.
- Status (registry)
- Recruiting
- Study type
- Interventional
- Enrollment
- 100 (estimated)
- First submitted
- 5 Sept 2025
- Start date
- 1 Dec 2025
- Primary completion date
- 31 Aug 2028
- Lead sponsor
- Centre Hospitalier Universitaire de Liege
- NCT number
- NCT07286565
What you commit to
- Study type: Interventional
- Planned participants: 100
- From 1 Dec 2025 to the primary completion date, 31 Aug 2028
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- Any acute or chronic condition that, in the investigator's opinion, significantly interferes with assessments and/or motor development.
Other
- Participation in a therapeutic trial.
- Lack of internet connection.
Who the study is looking for
- 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
Age in the registry: 4 months to - · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle Coordinator of this site? Confirm its status | Liège | Belgium | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.