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

Part of paid clinical trials in Palo Alto, California.

Sponsor
Columbia University
Study ID
NCT06839469
Status
Recruiting
Apply to this trial

Conditions

  • Duchenne Muscular Dystrophy (DMD)
  • Spinal Muscular Atrophy Type 3

Eligibility Criteria

Sex
ALL
Age
5 Years - N/A
Healthy Volunteers
Accepted

Study Details

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.

Key Dates

Start date
May 1, 2024
Status verified
Apr 2026
Primary completion
Dec 30, 2027
Completion
Apr 1, 2028

Study Design

Enrollment
106 participants (estimated)

Arms

  • Arm: Spinal Muscular Atrophy
    Ambulatory children and adults at least 5 years old by the time of enrollment with genetically confirmed SMA.
  • Arm: 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.
  • Arm: 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.

Primary Outcome Measure

Six Minute Walk Test [ Time Frame: One day ]

Central Contacts

Locations (3)

FacilityCityStateZIPSite coordinators
Stanford UniversityPalo AltoCalifornia94304
Sally Dunaway Young, PT, DPT
[email protected]
650-497-0075
Constance De Monts, PT, DPT
[email protected]
6502502292
John W Day, MD (PRINCIPAL_INVESTIGATOR)
Sally Dunaway Young, PT, DPT (SUB_INVESTIGATOR)
Boston Children's HospitalBostonMassachusetts02115
Amy Pasternak, PT, DPT
[email protected]
617-355-7212
Maria Fragala Pinkham, PT, DPT, DSc
[email protected]
6173557212
Amy Pasternak, PT, DPT (PRINCIPAL_INVESTIGATOR)
Maria F Fragala Pinkham, PT, DPT, Dsc (SUB_INVESTIGATOR)
Columbia University Irving Medical CenterNew YorkNew York10032
Jacqueline Montes
[email protected]
212-305-8916
Cara Kanner, DPT
[email protected]
516-581-8150

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By research site
Stanford University· Palo Alto, CABoston Children's Hospital· Boston, MAColumbia University Irving Medical Center· New York, NY

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