Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age5+
SponsorColumbia University
About this trial
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 criteria
Qualifiers
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.
Disqualifiers
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
Trial design
Treatments tested in this trial
- Not listed
Trial groups
106 Participants
are grouped into 3 trial groupsLocations
3Stanford University94304, Palo AltoCalifornia, United States
Boston Children's Hospital02115, BostonMassachusetts, United States
Columbia University Irving Medical Center10032, New YorkNew York, United States
Sponsors and collaborators
Columbia University
Lead sponsor
Stevens Institute of Technology
Collaborator
Stanford University
Collaborator
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Collaborator
Boston Children's Hospital, Boston, MA, USA
Collaborator
Source ClinicalTrials.gov