Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular… (NCT06839469) | Clinical Trial Compass
RecruitingNot Applicable
Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
United States106 participantsStarted 2024-05-01
Plain-language 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.
Who can participate
Age range
5 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Genetic confirmation of disease (DMD, SMA) or healthy control
* Able to walk independently at least 25 meters
* 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.
Exclusion Criteria:
* Use foot orthoses or assistive devices for community ambulation or a mobility device for community navigation
* Use investigational medications intended for treatment of NMD within 30 days
* Prior to study entry had an injury or surgery that would impact gait within the previous 3 months
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Six Minute Walk Test
Timeframe: One day
2
10 Meter Walk/Run
Timeframe: One day
3
Time Up and Go Test (TUG)
Timeframe: One day
4
Muscle Strength Testing with Hand-held Dynamometry (HHD)