RecruitingNCT06839469

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

Sponsor

Columbia University

Enrollment

106 participants

Start Date

May 1, 2024

Study Type

OBSERVATIONAL

Conditions

Duchenne Muscular Dystrophy (DMD)Spinal Muscular Atrophy Type 3

Summary

The purpose of this research is (1) to identify disease specific walking-related digital biomarkers of disease severity, and (2) monitor longitudinal changes in natural environments, for extended periods of time, in DMD and SMA.

Eligibility

Min Age: 5 Years

Inclusion Criteria4

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 Criteria3

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

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