Identifying walking-related digital biomarkers in rare childhood neuromuscular disorders

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 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.

Why it matters

Who can join: the full rules

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 it's running

• 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

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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.