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New treatment option for Spinal Muscular Atrophy (SMA)
Official title Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne Muscular Dystrophy or Spinal Muscular Atrophy Identified by Newborn Screening
ClinicalTrials.gov ID: NCT07286565
What this study is testing
- What it's testing
- 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.
- Time commitment: about 30 months
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 and older
You may be able to join if
- 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,
You likely can't join if
- 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.
See the full eligibility 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
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Liège, Belgium
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 30 months per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 4 months and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Liège, Belgium. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.