Examining the link between functional capacity and capability in Duchenne muscular dystrophy
Correlation Between Functional Capacity and Functional Capability in Patients With Duchenne Muscular Dystrophy-A Longitudinal Study
This study looks at how physical abilities and everyday functioning are connected in children with Duchenne muscular dystrophy over the course of a year.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 48 (estimated) |
| Ages | 5 Years to 18 Years |
| Sex | All |
| Sponsor | Samsung Medical Center Academic / other |
| Locations | 2 sites (Seoul and 1 other locations) |
| Trial ID | NCT05249361 on ClinicalTrials.gov |
What this trial studies
This observational study aims to explore the relationship between functional capacity and functional capability in children diagnosed with Duchenne muscular dystrophy (DMD). It involves two visits over a one-year follow-up period, where participants will undergo assessments using accelerometers and DEXA scans. The study does not involve any interventions, focusing instead on collecting and analyzing data to better understand the condition's impact on physical abilities.
Who should consider this trial
Good fit: Ideal candidates are children aged 5 to 18 years diagnosed with Duchenne muscular dystrophy through genetic testing.
Not a fit: Patients requiring daytime ventilator assistance or with significant cognitive decline may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could enhance understanding of DMD, leading to improved assessment methods and care strategies for affected children.
How similar studies have performed: While this study focuses on a specific correlation, similar observational studies have shown promise in understanding functional capabilities in neuromuscular disorders.
Eligibility criteria
Show full inclusion / exclusion 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.
Where this trial is running
Seoul and 1 other locations
- Samsung Medical Center — Seoul, Korea, Republic of (Recruiting)
- Samsung Medical Center — Seoul, Korea, Republic of (Recruiting)
Study contacts
- Principal investigator: Jeong-yi Kwon, MD,Ph.D — Samsung Medical Center
- Study coordinator: Yumi Choe
- Email: yumi.choe@sbri.co.kr
- Phone: 82-010-3173-2671
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.