Clinical trial NCT04193085: Wearable Technology to Assess Gait Function in SMA and DMD
Official title: Wearable Technology to Assess Gait Function in Spinal Muscle Atrophy and Duchenne Muscular Dystrophy
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Spinal Muscular Atrophy Type 3
Duchenne Muscular Dystrophy
Keywords: Spinal Muscular Atrophy
SMA
Duchenne Muscular Dystrophy
DMD
instrumented insole
neuromuscular disease
six minute walk test
machine learning model
gait analysis
wearable
ambulatory
stride
foot-worn
Lead sponsor: Columbia University
Sponsor class: OTHER
Collaborators: name: Muscular Dystrophy Association
name: Stevens Institute of Technology
First posted: 2019-12-10
Enrollment: 39
Sex: ALL
Minimum age (years): 5
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Observational model: COHORT
Time perspective: CROSS_SECTIONAL
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study sample will include 13 ambulatory SMA participants, 13 ambulatory DMD participants, and 13 healthy controls.

Brief summary: The purpose of this project is to devise instrumented insoles capable of accurately measuring gait at each footfall, over multiple hours in any environment. To achieve high accuracy, the investigators will develop a new learning-based calibration framework. Features will be tested in controlled lab settings 39 during a single visit in people with SMA (13), DMD (13) and healthy controls (13) and in 15 participants in real-life environments.

Detailed description: Spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD) are genetic disorders that often result in progressive weakness and impaired function. Results from this study will help characterize how gait is affected in SMA and DMD. This novel device can serve as a more affordable and versatile measurement instrument for neuromuscular disorders that affect gait and balance. All participants will be observed and measured while wearing the instrumented insoles in the lab and in real-life environments.

Eligibility criteria: Inclusion Criteria:

1. One of the following categories:

   * Genetic confirmation of spinal muscular atrophy
   * Genetic confirmation of Duchenne or Becker muscular dystrophy or evidence on muscle biopsy with a clinical presentation consistent with DMD/BMD
   * Healthy individuals.
2. Able to walk independently at least 25 meters

Exclusion Criteria:

1. Unable to walk 25 meters independently.
2. Use of investigational medications intended for the treatment of SMA or DMD/BMD within 30 days prior to study entry.

Primary outcomes:
measure: Validation of Instrumented Insoles: Six Minute Walk Test
timeFrame: Baseline
measure: Validation of Instrumented Insoles: 10 Meter Walk/Run
timeFrame: Baseline
measure: Validation of Instrumented Insoles: Time Up and Go Test (TUG)
timeFrame: Baseline
measure: Validation of Instrumented Insoles: Straight Line Walking
timeFrame: Baseline
measure: Validation of Instrumented Insoles: Circle Walking
timeFrame: Baseline
measure: Muscle Strength Testing with Hand-held Dynamometry (HHD)
timeFrame: Baseline
Secondary outcomes:
measure: Free-living Testing of Instrumented Insoles
timeFrame: One week
Other outcomes:
Locations:
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
Study officials:
affiliation: Columbia University
name: Jacqueline Montes, PT, EdD
role: PRINCIPAL_INVESTIGATOR
References: