Walking-related Digital Biomarkers in Childhood Neuromuscular Disorders
This observational study aims to understand how walking patterns, measured by special insoles, can show changes in Duchenne Muscular Dystrophy (DMD) and Spinal Muscular Atrophy Type 3 (SMA Type 3). Researchers want to find digital biomarkers (measurable indicators) that show how severe the disease is and how it changes over time in everyday life. You might be able to join if you have a confirmed diagnosis of DMD or SMA, or are a healthy control, and can walk independently for at least 25 meters. For DMD, you need to be on corticosteroids or have started them recently. For SMA, you need to be on a stable dose of an FDA-approved SMN up-regulator therapy or in an open-label extension for at least 6 months. The study will measure walking ability using tests like the Six Minute Walk Test, 10 Meter Walk/Run, and Time Up and Go Test. The current recruitment status is unclear.
- Study design
- This is an observational study with a planned enrollment of 106 participants. It is not a drug trial, but rather aims to understand disease progression through walking patterns.
- What's involved
- Participants will wear instrumented insoles in a lab setting and in their daily lives. Walking tests like the Six Minute Walk Test will be performed on one day.
- Compensation
- Not stated in the trial record.
- Follow-up
- Walking tests are measured on one day. The study aims to monitor longitudinal changes, but specific follow-up duration is not detailed.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
At a glance
Conditions
NCT06839469
Where you'd take part
This study runs at 3 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.
Boston Children's Hospital
Boston, Massachusettsstudy coordinator listed
Recruiting
Columbia University Irving Medical Center
New York, New Yorkstudy coordinator listed
Recruiting
Stanford University
Palo Alto, Californiastudy coordinator listed
Recruiting
Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.
Who to contact
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Inclusion
Exclusion
What this trial measures
- Six Minute Walk TestOne day
Participants will complete the six minute walk test in a corridor which includes the instrumented walkway, while wearing the insoles. This is to measure spatial parameters, kinetic parameters, temporal parameters, and metrics of cumulative activity in order to compare against the gold standard instrumented walkway. The six minute walk test is an objective evaluation of functional exercise capacity, and measures the maximum distance a person can walk in six minutes over a 25 meter course.
- 10 Meter Walk/RunOne day
Participants will walk, or run if able to, for 10 meters on the instrumented walkway while also wearing the insoles. This will be done to measure spatial parameters, kinetic parameters, temporal parameters, and metrics of cumulative activity in order to compare against the gold standard instrumented walkway. This outcome is reported as the time it takes for the participant to complete 10 meters as fast as possible.
- Time Up and Go Test (TUG)One day
Participants will complete the TUG test. This is a timed test of mobility, requiring the participant to stand up from a seated position, walk 3 meters and return to sitting in the chair. The outcome reported is the time it takes for the participant to complete the sequence.
- Muscle Strength Testing with Hand-held Dynamometry (HHD)One day
HHD is used to assess the strength of muscles and is reported as the maximum strength exerted in pounds (lbs).
- 30 Second Sit to Stand Test (30STS)One day
30STS test is used to basic muscle power and function. The participant begins seated with their arms at their sides, feet flat on the floor and back against the chair. They are instructed to rise up to a full standing position and sit back down as many times as possible in 30 seconds, with or without using their hands for support.
- Ankle Range of MotionOne day
Ankle range of motion (ROM) will be assessed on both the left right side using standardized goniometers. The procedures used will be standardized and performed by trained physical therapists who have attended in-person training. ROM will be recorded in degrees for plantar flexion and dorsiflexion.
- Knee Extension Range of MotionOne day
Knee extension range of motion (ROM) will be assessed on both the left right side using standardized goniometers. The procedures used will be standardized and performed by trained physical therapists who have attended in-person training. ROM will be recorded in degrees of knee extension.
- Hip Extension Range of MotionOne day
Hip extension range of motion (ROM) will be assessed on both the left right side using standardized goniometers. The procedures used will be standardized and performed by trained physical therapists who have attended in-person training. ROM will be recorded in degrees of hip extension.