Spinal Muscular Atrophy Type 3NCT04193085
Wearable Technology to Assess Gait Function in SMA and DMD
An observational study of Spinal Muscular Atrophy Type 3 and Duchenne Muscular Dystrophy, run by Columbia University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 5 years and olderHealthy volunteers can take part
- Conditions the study is for
- Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy
- What is being tested?
- Observational
- How many people
- 39 participants
- Where
- 1 siteUnited States
- Study dates
- 25 Nov 2019to 31 Aug 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: The study sample will include 13 ambulatory SMA participants, 13 ambulatory DMD participants, and 13 healthy controls.
Who may be able to join
- 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.
- Able to walk independently at least 25 meters
Who cannot join
- Unable to walk 25 meters independently.
- Use of investigational medications intended for the treatment of SMA or DMD/BMD within 30 days prior to study entry.
Trial locations and contacts
1 site in 1 country.
United States1 site
Columbia University Medical Center
New York, New York, 10032
About this study
Official title: Wearable Technology to Assess Gait Function in Spinal Muscle Atrophy and Duchenne Muscular Dystrophy
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.
Read the 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.
- Being tested
- Observational
- 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
- Medical subject headings
- Spinal Muscular Atrophies of Childhood, Muscular Dystrophy, Duchenne, Muscular Atrophy, Spinal, Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ObservationalOther
Observational
| Study group | Role | What they receive |
|---|---|---|
| Spinal Muscular Atrophy (SMA) | ambulatory children and adults at least 5 years old by the time of enrollment with genetically confirmed SMA Receives: Other: Observational | |
| Duchenne / Becker Muscular Dystrophy (DMD/BMD) | 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. Receives: Other: Observational | |
| Healthy Control | The healthy control group will be age and gender-matched to the SMA and DMD groups as best as possible Receives: Other: Observational |
What is being measured
The primary outcome is the main question the study is designed to answer.
Validation of Instrumented Insoles: Six Minute Walk Test
To measure spatial parameters, kinetic parameters, temporal parameters, and metrics of cumulative activity in order to compare against the gold standard instrumented walkway participants will complete the Six Minute Walk Test. This test is an objective evaluation of functional exercise capacity, measures the maximum distance a person can walk in six minutes over a 25 meter course. This will be performed in a corridor and include the instrumented walkway.
Measured: Baseline
Validation of Instrumented Insoles: 10 Meter Walk/Run
To measure spatial parameters, kinetic parameters, temporal parameters, and metrics of cumulative activity in order to compare against the gold standard instrumented walkway, participants will walk, or run if able to, for 10 meters on the instrumented walkway.
Measured: Baseline
Validation of Instrumented Insoles: Time Up and Go Test (TUG)
To measure spatial parameters, kinetic parameters, temporal parameters, and metrics of cumulative activity in order to compare against the gold standard instrumented walkway, participants will complete the TUG test. This test is designed to test mobility after a person stands up from a seated position. This will be completed on the instrumented walkway.
Measured: Baseline
Validation of Instrumented Insoles: Straight Line Walking
To measure spatial parameters, kinetic parameters, temporal parameters, and metrics of cumulative activity in order to compare against the gold standard instrumented walkway, participants will be asked to walk in a straight line multiple times over the instrumented walkway.
Measured: Baseline
Validation of Instrumented Insoles: Circle Walking
To measure spatial parameters, kinetic parameters, temporal parameters, and metrics of cumulative activity in order to compare against the gold standard instrumented walkway participants will be asked to walk a series of half-circles on the instrumented walkway.
Measured: Baseline
Muscle Strength Testing with Hand-held Dynamometry (HHD)
HHD is used to assess strength of selected muscles.
Measured: Baseline
Secondary outcomes (1)
Free-living Testing of Instrumented Insoles
To determine spatial parameters, kinetic parameters, temporal parameters, and metrics of cumulative activity using instrumented insoles, 5 participants with SMA, 5 participants with DMD, and 5 healthy controls will be asked to wear the insoles along with a validated activity tracker. Participants will be asked to wear the insoles during a visit to the lab, and then for one week at home in a real-life environment. Participants will be asked to wear the insoles and activity tracker for at least 4 hours per day when at home.
Measured: One week
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Dec 2019
- Study start25 Nov 2019
- Primary completion31 Aug 2023
- Study completion31 Aug 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Spinal Muscular Atrophy Type 3Studies of the same condition, open and under way.
- More trials run by Columbia UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Enrolment
- 39 participants (actual)
Sponsor and investigators
- Lead sponsor
- Columbia University
- Sponsor type
- University, hospital or other
- Collaborators
- Muscular Dystrophy Association, Stevens Institute of Technology
- Responsible party
- Principal investigator, Jacqueline Montes, Columbia University
- Investigators
- Jacqueline Montes, PT, EdD, Principal investigator, Columbia University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04193085
- Sponsor's protocol ID
- AAAS5641
- Status last verified
- Feb 2024
- First submitted
- 11 Nov 2019
- Last updated
- 23 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Montes J, Kanner CH, Duong T, Rodriguez-Torres R, Uher D, Young SD, Farooquee R, Druffner A, Pasternak A, Fragala-Pinkham M, Zanotto D. Wearable-Derived Patterns of Performance Fatigability During Gait in Spinal Muscular Atrophy. Muscle Nerve. 2025 Sep;72(3):493-501. doi: 10.1002/mus.28461. Epub 2025 Jun 25.
PubMed 40557477
Study at a glance
From the registry record, last updated by the sponsor on 23 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Wearable Technology to Assess Gait Function in Spinal Muscle Atrophy and Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT04193085
- Acronym / sponsor ID
- AAAS5641
- Keywords
- Spinal Muscular Atrophy, SMA, Duchenne Muscular Dystrophy, DMD, instrumented insole, neuromuscular disease, six minute walk test, machine learning model
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 39 participants (actual)
- Lead sponsor
- Columbia UniversityUniversity, hospital or other
- Collaborators
- Muscular Dystrophy Association, Stevens Institute of Technology
- Study start
- 25 Nov 2019 (actual)
- Primary completion
- 31 Aug 2023 (actual)
- Study completion
- 31 Aug 2023 (actual)
- First posted
- 10 Dec 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Spinal Muscular Atrophy (SMA) | – | Other: Observationalambulatory children and adults at least 5 years old by the time of enrollment with genetically confirmed SMA |
| Duchenne / Becker Muscular Dystrophy (DMD/BMD) | – | Other: Observationalambulatory 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 | – | Other: ObservationalThe healthy control group will be age and gender-matched to the SMA and DMD groups as best as possible |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The study sample will include 13 ambulatory SMA participants, 13 ambulatory DMD participants, and 13 healthy controls.
Key inclusion criteria (5 listed)
- One of the following categories:
- Able to walk independently at least 25 meters
Key exclusion criteria (2 listed)
- Unable to walk 25 meters independently.
- Use of investigational medications intended for the treatment of SMA or DMD/BMD within 30 days prior to study entry.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ObservationalObservational | Other | Duchenne / Becker Muscular Dystrophy (DMD/BMD), Healthy Control, Spinal Muscular Atrophy (SMA) |
Primary outcome
Validation of Instrumented Insoles: Six Minute Walk Test
Time frame: Baseline
To measure spatial parameters, kinetic parameters, temporal parameters, and metrics of cumulative activity in order to compare against the gold standard instrumented walkway participants will complete the Six Minute Walk Test. This test is an objective evaluation of functional exercise capacity, measures the maximum distance a person can walk in six minutes over a 25 meter course. This will be performed in a corridor and include the instrumented walkway.
Validation of Instrumented Insoles: 10 Meter Walk/Run
Time frame: Baseline
To measure spatial parameters, kinetic parameters, temporal parameters, and metrics of cumulative activity in order to compare against the gold standard instrumented walkway, participants will walk, or run if able to, for 10 meters on the instrumented walkway.
Validation of Instrumented Insoles: Time Up and Go Test (TUG)
Time frame: Baseline
To measure spatial parameters, kinetic parameters, temporal parameters, and metrics of cumulative activity in order to compare against the gold standard instrumented walkway, participants will complete the TUG test. This test is designed to test mobility after a person stands up from a seated position. This will be completed on the instrumented walkway.
Validation of Instrumented Insoles: Straight Line Walking
Time frame: Baseline
To measure spatial parameters, kinetic parameters, temporal parameters, and metrics of cumulative activity in order to compare against the gold standard instrumented walkway, participants will be asked to walk in a straight line multiple times over the instrumented walkway.
Validation of Instrumented Insoles: Circle Walking
Time frame: Baseline
To measure spatial parameters, kinetic parameters, temporal parameters, and metrics of cumulative activity in order to compare against the gold standard instrumented walkway participants will be asked to walk a series of half-circles on the instrumented walkway.
Muscle Strength Testing with Hand-held Dynamometry (HHD)
Time frame: Baseline
HHD is used to assess strength of selected muscles.
1 secondary outcome
Free-living Testing of Instrumented Insoles
Time frame: One week
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Sep 2024.
- Derived from this study: PubMed 40557477Montes J, Kanner CH, Duong T, Rodriguez-Torres R, Uher D, Young SD, Farooquee R, Druffner A, Pasternak A, Fragala-Pinkham M, Zanotto D. Wearable-Derived Patterns of Performance Fatigability During Gait in Spinal Muscular Atrophy. Muscle Nerve. 2025 Sep;72(3):493-501. doi: 10.1002/mus.28461. Epub 2025 Jun 25.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jacqueline Montes, PT, EdD principal investigator
Columbia University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Spinal Muscular Atrophy Type 3 and Duchenne Muscular Dystrophy, run by Columbia University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Validation of Instrumented Insoles: Six Minute Walk Test (time frame: Baseline). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04193085, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Sep 2024.