NCT07286565 Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn Screening
| NCT ID | NCT07286565 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Centre Hospitalier Universitaire de Liege |
| Condition | Spinal Muscular Atrophy (SMA) |
| Study Type | INTERVENTIONAL |
| Enrollment | 100 participants |
| Start Date | 2025-12-01 |
| Primary Completion | 2028-08 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
This trial targets 100 participants in total. It began in 2025-12-01 with a primary completion date of 2028-08.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility 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.
Contact & Investigator
Tamara Dangouloff, PhD
PRINCIPAL INVESTIGATOR
University of Liege
Frequently Asked Questions
Who can join the NCT07286565 clinical trial?
This trial is open to participants of all sexes, aged 4 Months or older, studying Spinal Muscular Atrophy (SMA). Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT07286565 currently recruiting?
Yes, NCT07286565 is actively recruiting participants. Contact the research team at tamara.dangouloff@uliege.be for enrollment information.
Where is the NCT07286565 trial being conducted?
This trial is being conducted at Liège, Belgium.
Who is sponsoring the NCT07286565 clinical trial?
NCT07286565 is sponsored by Centre Hospitalier Universitaire de Liege. The principal investigator is Tamara Dangouloff, PhD at University of Liege. The trial plans to enroll 100 participants.