Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders

Launched by COLUMBIA UNIVERSITY · Feb 17, 2025

Keywords

ClinConnect Summary

This clinical trial is looking to understand how walking patterns can help us learn more about the severity of two rare childhood muscle disorders: Spinal Muscular Atrophy Type 3 and Duchenne Muscular Dystrophy (DMD). Researchers want to find specific "digital biomarkers," which are measurable signs related to walking, that can show how the disease affects children. By tracking these walking patterns over time in everyday settings, they hope to see how the conditions change and progress.

To participate in the study, you should be between the ages of 18 and 26 and be able to walk independently for at least 25 meters. You should also have a confirmed diagnosis of either DMD or Spinal Muscular Atrophy, or be a healthy control participant. Participants will be monitored while walking in their usual environments, and those currently taking specific treatments for their conditions will be included. It’s important to note that if you use any walking aids or have had recent injuries or surgeries that affect your walking, you may not be eligible for this trial. Overall, this study aims to gather valuable information that could improve understanding and treatment of these conditions in young adults.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• One of the following categories:
• • Genetic confirmation of disease (DMD, SMA) or healthy control Able to walk independently at least 25 meters
• Cohort specific inclusion criteria include:
• • 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