Wearable monitoring of motor development in babies and young children with spinal muscular atrophy detected at birth

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational, longitudinal effort will follow up to 60 children identified with SMA (and healthy controls) for as long as 30 months using home-worn wearable devices (Syde and Motor Assessment) to capture motor activity. Participants will include infants and children from before 4 months up to under 4 years of age, with known SMN2 copy number and variable exposure to disease-modifying therapies. Data will be collected remotely through a federated model and analyzed to validate motor outcome measures suitable for this young age group. The goal is to determine when movement patterns diverge from typical development to inform timing of additional treatments under development.

Why it matters

Who can join: the full rules

Inclusion criteria (Test cohort):

1. Genetically confirmed SMA and number of SMN2 copies available
2. a. Patients identified by NBS and treated with disease modifying therapy (DMT)

(2)a,i 4 copies or more of SMN2 and not treated with DMT

(2)a,ii less than 4 copies of SMN2 and not treated with DMT

or

(2)b. Patients diagnosed due to a sibling or alternative means

(2)b,i 4 copies or more of SMN2 and not treated with DMT

(2)b,ii less than 4 copies of SMN2 and not treated with DMT

(3)Patients between 4 months and below 4 years at baseline. Inclusion of patients can be before 4 months of age

(4)Parent(s)/legal guardian(s) able to provide written informed consent prior to the patient's participation in the study

Exclusion Criteria (Test cohort):

(1) Any acute or chronic condition which, according to the investigator, significantly interferes with the assessments and/or the motor evolution

Inclusion criteria (Control):

1. Typically developing child
2. Participant between 6 months and 4 years at baseline
3. Parent(s)/legal guardian(s) able to provide written informed consent prior to the participation in the study

Exclusion criteria (Control):

(1)Any acute or chronic condition which, according to the investigator, significantly interferes with the assessments and/or the motor evolution

Where it's running

• University of Oxford
  Oxford, United Kingdom Recruiting

Study contacts

• Study coordinator: Charlotte Lilien
• Email: charlotte.lilien@paediatrics.ox.ac.uk
• Phone: 01865618799

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Newborn SMA Screening Service Evaluation

  This project tests newborn SMA screening to see if it works well and is acceptable to families in the UK.

  Recruiting at 2 sites University of Oxford · London and 2 other locations · NCT07888868

• Biomarker search in SMA using advanced multi-omics

  Researchers are trying to see if new biomarkers can help diagnose and monitor spinal muscular atrophy in patients and newborns.

  Recruiting at 3 sites Istituto Giannina Gaslini · Genova and 2 other locations · NCT07883655

• Effects of Different Stiffness Settings During Robotic Isokinetic Training in Children With SMA

  This study tests whether a portable knee-training robot with a compliant vs. rigid stiffness setting improves knee strength in children with spinal muscular atrophy, using each child’s other leg as a comparison.

  Recruiting Peking University Third Hospital · Beijing · NCT07822711

• HABIT-ILE plus Functional Strength Training for Children with SMA

  This study will try an intensive HABIT-ILE program combined with functional strength training for children aged 5–17 with SMA who are on stable disease-modifying therapy to see if it improves motor skills.

  Recruiting Teachers College, Columbia University · New York, New York · NCT07488338

• Long-read genetic analysis for Spinal Muscular Atrophy (LOREASI)

  This project tries a long-read DNA technique on adults with known SMN1 copy-number patterns to find hidden '2+0' carrier genotypes that standard tests miss.

  Recruiting University Hospital, Rouen · Rouen · NCT07332702

• Portable markerless video system to measure walking in adults with neuromuscular conditions or healthy volunteers.

  This project will test a portable video device that measures walking patterns to see if it can track gait changes in adults with neuromuscular diseases and in healthy volunteers.

  Recruiting Institut de Myologie, France · Paris · NCT07321977

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.