Correlation Between Functional Capacity and Functional Capability in Patients With Duchenne Muscular Dystrophy-A Longitudinal Study
Trial Snapshot
- Phase
- Not Applicable
- Status
- Recruiting
- Sponsor
- Samsung Medical Center
- Enrollment
- 48
- Locations
- 1
- Primary Endpoint
- Correlation between physical activity(VM) and muscle quantitative index
Study Overview
Brief Summary
This study investigates the correlation between assessments measuring functional capacity and functional capability in patients with Duchenne muscular dystrophy.
Detailed Description
This study is a prospective single-arm observational study, with a total of two visits for one year follow-up.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 5 Years to 18 Years (Child, Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Children aged 5 to 18 years diagnosed with Duchenne muscular dystrophy through genetic testing
- •Children who understand the contents of this research and can properly conduct the research
Exclusion Criteria
- •Requiring daytime ventilator assistance or using invasive mechanical ventilation through tracheostomy (Non-invasive mechanical ventilation such as positive pressure ventilation at night is allowed)
- •History of peripheral nerve damage
- •History of major surgery within 12 weeks or if major surgery is expected during the test period
- •History of central nervous system disorders (eg, cerebral infarction, spinal cord injury)
- •Having difficulties in conducting this study due to cognitive decline.
Outcomes
Primary Outcomes
Correlation between physical activity(VM) and muscle quantitative index
Time Frame: Baseline up to Week 48
We analyze the correlation between the change in activity counts from baseline to 48-week and the change in muscle quantitative measures of upper and lower extremities from baseline to 48-week. The activity counts will be measured with ActiGraph wgt3x-bt worn on patient's dominant ankle and wrist. Accelerometers recorded acceleration in three orthogonal axes (x, y, z) at 30 Hz. Accelerometer recordings were uploaded to ActiLife software, integrated into 15-second epochs, and converted into an omnidirectional acceleration estimate, or vector magnitude (VM), calculated as the square root of the sum of the triaxial signals squared. The muscle quantitative will be measured with Microfet2. Muscle quantitative of upper extremity is the sum of the flexion and extension of both elbows and lower extremity is the sum of both knee flexion and extension and bilateral ankle dorsiflexion.
Secondary Outcomes
- Correlation between physical activity and Vignos scale(Baseline up to Week 48)
- Correlation between physical activity and PEDI-CAT(the Pediatric Evaluation of Disability Inventory-Computer Adaptive Test)(Baseline up to Week 48)
- Correlation between physical activity and EQ-5D(EuroQol-5D)(Baseline up to Week 48)
- Correlation between physical activity and Brooke scale(Baseline up to Week 48)
- Correlation between physical activity and 6MWT(6-minute walking test)(Baseline up to Week 48)
- Correlation between physical activity and quantitative muscle ultrasonography(Baseline up to Week 48)
- Correlation between physical activity and DMD upper limb PROM(Baseline up to Week 48)
- Correlation between physical activity and Pulmonary function test(Baseline up to Week 48)
- Correlation between physical activity and PUL(Performance of Upper Limb module for DMD)(Baseline up to Week 48)
- Correlation between physical activity and CHQ-PF50(Child Health Questionnaire - Parent form 50 )(Baseline up to Week 48)
- Correlation between physical activity and NSAA(The North Star Ambulatory Assessment)(Baseline up to Week 48)
Investigators
Jeong Yi Kwon
Professor
Samsung Medical Center