Gait and Bone Health in SMA
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Spinal Muscular Atrophy Type 3, Ambulatory Spinal Muscular Atrophy. Basic parameters: 8 years — 50 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
Identification of Gait-related Digital Biomarkers of Bone Health in Spinal Muscular Atrophy
Overview
The objective of this study is to understand how spatiotemporal and kinetic gait parameters are associated with bone health in pwSMA who are receiving DMT. We hypothesize that gait parameters are associated with BMD and can determine fracture risk in pwSMA. This is an observational study that involves one in-clinic visit (approximately 3 hours) with remote follow-up visits (approximately 15 minutes) every 3 months for 1 year to collect fracture and medical history. Each of the assessments included in this study are non-interventional and are not intended as a treatment nor are they at the level of standard of care.
Detailed description
Addressing bone health is a high priority and remains a concern for the SMA community despite the availability of DMTs. Fractures often have devastating, irrecoverable impacts on function, especially in ambulatory pwSMA. While the most recent standards of care for SMA recommend annual screening, supportive treatments are not recommended until there has been a fracture, a reactive approach which may not be enough to mitigate LBM. Implementing novel, wearable technology and machine learning (ML) models to identify fracture risk will lead to a proactive approach to manage bone health and attenuate the negative sequalae of fractures. This is an observational cross-sectional study of ambulatory children and adults with SMA. The aims of this study are to (1) Identify gait-related digital biomarkers of bone health in ambulatory pwSMA, (2) Determine the ability of digital biomarkers to differentiate between pwSMA with and without a history of fractures, and (3) Investigate the ability of risk profiles derived from the digital biomarkers to predict prospective fractures over one year.
Primary outcome measures
- Dual X-Ray Absorptiometry (DXA) [Time frame: Baseline]
- Blood collection [Time frame: Baseline]
- Fracture history [Time frame: Baseline, months 3, 6, 9, and 12]
- Fall history [Time frame: Baseline, months 3, 6, 9 and 12]
- Collection of spatiotempoal and kinetic gait parameters using instrumented insoles (AI-Sole) [Time frame: Baseline]
- Six Minute Walk Test [Time frame: Baseline]
- IPAQ-SF [Time frame: Baseline]
Secondary outcome measures (3)
- Ten Meter Walk Run (10MWR) [Time frame: Baseline]
- Timed Up and Go Test (TUG) [Time frame: Baseline]
- 30 Second Sit to Stand (30-STS) [Time frame: Baseline]
Eligibility criteria
Inclusion criteria
- Confirmed diagnosis of 5qSMA
- Between 8 and 50 years of age
- Able to walk 10 meters without support or an assistive device
- All participants who meet the inclusion criteria regardless of treatment status will be included, including those on adjuvant therapies, whether investigational or approved
Exclusion criteria
- Injury or surgery within previous 3 months that would impact ability to perform in-clinic gait assessments
- Unwilling or unable to comply with all study procedures
- Age less than 8 or over 50 years
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 1 center
- Columbia University Irving Medical Center — New York
Identifiers
NCT: NCT07400198 · AAAV3667