Identifying walking-related digital biomarkers in rare childhood neuromuscular disorders
Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
This study is testing if special insoles with sensors can help track walking patterns in kids with spinal muscular atrophy or Duchenne muscular dystrophy to better understand their mobility.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 106 (estimated) |
| Ages | 5 Years and up |
| Sex | All |
| Sponsor | Columbia University Academic / other |
| Locations | 3 sites (Palo Alto, California and 2 other locations) |
| Trial ID | NCT06839469 on ClinicalTrials.gov |
What this trial studies
This research aims to identify specific walking-related digital biomarkers that reflect the severity of spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD). By utilizing instrumented insoles equipped with sensors, the study will monitor participants' gait parameters in both controlled lab settings and real-world environments over extended periods. The approach leverages machine learning to analyze data collected from these insoles, providing insights into functional changes in mobility. Participants will include those with genetic confirmation of DMD or SMA who can walk independently.
Who should consider this trial
Good fit: Ideal candidates are children with genetic confirmation of SMA or DMD who can walk independently for at least 25 meters.
Not a fit: Patients who use foot orthoses or assistive devices for mobility may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved monitoring and understanding of disease progression in children with SMA and DMD.
How similar studies have performed: Other studies have shown promise in using digital biomarkers for monitoring neuromuscular disorders, but this specific approach is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Genetic confirmation of disease (DMD, SMA) or healthy control * Able to walk independently at least 25 meters * Ongoing corticosteroids therapy or initiation of corticosteroid therapy in the previous 3 months for DMD * Stable dose of FDA approved SMN up-regulator therapy or in an open-label extension phase of a study treatment for at least 6 months for SMA or gene replacement at enrollment for SMA or DMD participants. Exclusion Criteria: * Use foot orthoses or assistive devices for community ambulation or a mobility device for community navigation * Use investigational medications intended for treatment of NMD within 30 days * Prior to study entry had an injury or surgery that would impact gait within the previous 3 months
Where this trial is running
Palo Alto, California and 2 other locations
- Stanford University — Palo Alto, California, United States (Recruiting)
- Boston Children's Hospital — Boston, Massachusetts, United States (Recruiting)
- Columbia University Irving Medical Center — New York, New York, United States (Recruiting)
Study contacts
- Study coordinator: Jacqueline Montes, PT, EdD
- Email: jm598@cumc.columbia.edu
- Phone: 212-305-8916
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.