Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
Status: Recruiting
Location: See all (3) locations...
Study Type: Observational
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
Participation Requirements
Sex: All
Minimum Age: 5
Healthy Volunteers: t
View:
• 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.
Locations
United States
California
Stanford University
RECRUITING
Palo Alto
Massachusetts
Boston Children's Hospital
RECRUITING
Boston
New York
Columbia University Irving Medical Center
RECRUITING
New York
Contact Information
Primary
Jacqueline Montes, PT, EdD
jm598@cumc.columbia.edu
212-305-8916
Backup
Michael Lutzker
ml5121@cumc.columbia.edu
925-705-5133
Time Frame
Start Date: 2024-05-01
Estimated Completion Date: 2028-04-01
Participants
Target number of participants: 106
Treatments
Spinal Muscular Atrophy
Ambulatory children and adults at least 5 years old by the time of enrollment with genetically confirmed SMA.
Duchenne Muscular Dystrophy
Ambulatory children and adults ages at least 5 years old by the time of enrollment with genetically confirmed Duchenne or Becker muscular dystrophy or evidence on muscle biopsy with a clinical presentation consistent with DMD/BMD.
Healthy Control
The healthy control group will be ambulatory children and adults at least 5 years of age, age- and gender-matched to the SMA and DMD groups as best as possible.
Related Therapeutic Areas
Sponsors
Leads: Columbia University
Collaborators: Stanford University, Boston Children's Hospital, Boston, MA, USA, Stevens Institute of Technology, Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)