Gait and Bone Health in SMA
{ "Gait and Bone Health in Spinal Muscular Atrophy (SMA)", "This observational study aims to understand the connection between how people with Spinal Muscular Atrophy (SMA) walk and their bone health, especially for those receiving disease-modifying therapies (DMTs). Researchers want to see if walking patterns can help predict bone mineral density (BMD) and the risk of fractures. You may be eligible if you are 8 to 50 years old, have a confirmed diagnosis of 5qSMA, and can walk 10 meters without help. The study involves one in-clinic visit and remote follow-up calls for a year. This research hopes to find new ways to proactively manage bone health in people with SMA.", "design": "This is an observational study with a planned enrollment of 22 participants. It is not a treatment study, but rather aims to understand existing health patterns.", "commitments": "You would have one in-clinic visit lasting about 3 hours. After that, there will be remote follow-up visits every 3 months for one year, each lasting about 15 minutes.", "compensation": "Not stated in the trial record.", "follow_up": "Participants will have remote follow-up visits every 3 months for one year to collect fracture and medical history.", }
- Study design
- Not specified.
- What's involved
- Not specified in the trial record.
- Compensation
- Not stated in the trial record.
- Follow-up
- Not specified.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
Gait and Bone Health in SMA
At a glance
Conditions
Where it's being run
1 sites across 1 statesWho to contact
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Inclusion
Exclusion
What this trial measures
- Dual X-Ray Absorptiometry (DXA)Baseline
Bone mineral density (BMD), a measure of the mineral content in bones is assessed using dual x-ray absorptiometry (DXA). DXA can be used to identify individuals with low bone mass (LBM). DXA uses low-power x-ray and is a safe and reliable measure validated to diagnose osteoporosis. DXA provides a t-score and z-score so that based on age, low bone mass can be appropriately determined. DXA also provides a subtotal BMD score as well as scores specific to certain body regions, both of which will be utilized in the first aim.
- Blood collectionBaseline
Blood will be collected to assess for routine markers related to bone mineral density. Blood will be tested for levels of calcium, vitamin D, bone alkaline phosphatase (BALP), and C-terminal telopeptide of type I collagen (CTx). The amount of blood collected will be within hospital rules for children. The total amount of blood will be approximately 2 mL or 1/2 a teaspoon. BALP is a bone formation marker while CTx is a bone resorption marker. Calcium and vitamin D are known to be positively associated with BMD.
- Fracture historyBaseline, months 3, 6, 9, and 12
Details regarding lifetime fracture history will be collected via questioning at the in-clinic visit and during remote follow up visits, every three months for one year. The participant will be asked to describe each incident separately, including the age at fracture, mechanism of injury (how it happened, its nature, and force), any preceding events like falls or dizziness, and associated symptoms. Fractures will be characterized by location, type and treatment (surgical or conservative).
- Fall historyBaseline, months 3, 6, 9 and 12
Details regarding falls, that occurred within the past year, will be collected using a structured questionnaire at the in-clinic visit and during the one-year follow-up. Falls will be described by mechanism, environment and outcome.
- Collection of spatiotempoal and kinetic gait parameters using instrumented insoles (AI-Sole)Baseline
Stride-by-stride spatiotemporal and kinetic gait parameters will be collected with AI-Sole continuously during the 6MWT. Spatiotemporal parameters included stride length (SL), stride velocity (SV), percent time in stance phase (%St), and percent time in terminal double support (%DS). Kinetic parameters included the anteroposterior (AP-COP) and mediolateral (ML-COP) ranges of the center-of-pressure normalized by shoe size, as well as the Absolute COP-Cyclogram Asymmetry Index (\|ASI\|).
- Six Minute Walk TestBaseline
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.
- IPAQ-SFBaseline
A quantitative assessment of usual physical activity will be captured using the International Physical Activity Questionnaire Short Form (IPAQ-SF).The IPAQ-SF is a commonly used self-administered questionnaire designed to obtain internationally comparable data on health-related physical activity. The IPAQ-SF is composed of seven questions from four domains of physical activity, including vigorous and moderate physical activity, walking physical activity, and sitting time. A weighted total from vigorous physical activity, moderate physical activity, and walking physical activity is calculated to produce a total amount of physical activity in MET-min/week.