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

Study Identifier:
AAAU9807
ClinicalTrials.gov Identifier:
NCT06839469
EudraCT Identifier:
N/A
EU CT Number:
N/A
Sponsor:
Columbia University
Recruiting

Study Details

Medical Condition
  • Spinal Muscular Atrophy Type III
  • All Spinal Muscular Atrophies
Study Drug
    Date
    May 2024 - Dec 2027
    Patient Requirements
    Sex: Female & Male
    Age: 5+ years
    Requirements Information
    Inclusion and 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

    Protocol 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.

    Trial Locations

    Location
    Status
    Location
    Stanford University
    Palo Alto, California, United States, 94304
    Status
    Recruiting
    Location
    Boston Children's Hospital
    Boston, Massachusetts, United States, 02115
    Status
    Recruiting
    Location
    Columbia University Irving Medical Center
    New York, New York, United States, 10032
    Status
    Recruiting

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