Clinical trial NCT05249361: Correlation Between Functional Capacity and Functional Capability in Duchenne Muscular Dystrophy
Official title: Correlation Between Functional Capacity and Functional Capability in Patients With Duchenne Muscular Dystrophy-A Longitudinal Study
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Duchenne Muscular Dystrophy
Keywords: Duchenne Muscular Dystrophy
DMD
Accelerometer
ActiGraph
DEXA
muscle ultrasound
Lead sponsor: Samsung Medical Center
Sponsor class: OTHER
First posted: 2022-02-21
Enrollment: 48
Sex: ALL
Minimum age (years): 5
Maximum age (years): 18
Standard ages: CHILD
ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Asian patients of Duchenne muscular dystrophy, who live in South Korea and visited the Samsung medical center in Gangnam-gu, Seoul.

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.

Eligibility criteria: Inclusion Criteria:

1. Children aged 5 to 18 years diagnosed with Duchenne muscular dystrophy through genetic testing
2. Children who understand the contents of this research and can properly conduct the research

Exclusion Criteria:

1. Requiring daytime ventilator assistance or using invasive mechanical ventilation through tracheostomy (Non-invasive mechanical ventilation such as positive pressure ventilation at night is allowed)
2. History of peripheral nerve damage
3. History of major surgery within 12 weeks or if major surgery is expected during the test period
4. History of central nervous system disorders (eg, cerebral infarction, spinal cord injury)
5. Having difficulties in conducting this study due to cognitive decline.

Primary outcomes:
measure: Correlation between physical activity(VM) and muscle quantitative index
timeFrame: Baseline up to Week 48
Secondary outcomes:
measure: Correlation between physical activity and Vignos scale
timeFrame: Baseline up to Week 48
measure: Correlation between physical activity and Brooke scale
timeFrame: Baseline up to Week 48
measure: Correlation between physical activity and 6MWT(6-minute walking test)
timeFrame: Baseline up to Week 48
measure: Correlation between physical activity and NSAA(The North Star Ambulatory Assessment)
timeFrame: Baseline up to Week 48
measure: Correlation between physical activity and PUL(Performance of Upper Limb module for DMD)
timeFrame: Baseline up to Week 48
measure: Correlation between physical activity and PEDI-CAT(the Pediatric Evaluation of Disability Inventory-Computer Adaptive Test)
timeFrame: Baseline up to Week 48
measure: Correlation between physical activity and EQ-5D(EuroQol-5D)
timeFrame: Baseline up to Week 48
measure: Correlation between physical activity and CHQ-PF50(Child Health Questionnaire - Parent form 50 )
timeFrame: Baseline up to Week 48
measure: Correlation between physical activity and quantitative muscle ultrasonography
timeFrame: Baseline up to Week 48
measure: Correlation between physical activity and DMD upper limb PROM
timeFrame: Baseline up to Week 48
measure: Correlation between physical activity and Pulmonary function test
timeFrame: Baseline up to Week 48
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
Study officials:
affiliation: Samsung Medical Center
name: Jeong-yi Kwon, MD,Ph.D
role: PRINCIPAL_INVESTIGATOR
References: