Correlation Between Functional Capacity and Functional Capability in Duchenne Muscular Dystrophy

NCT05249361 | Unknown

• 1 other identifier: 2022-05-136
• Study Type: observational
• Target: 48
• Locations: 1 country
• Sites: 2

Brief Summary

This study investigates the correlation between assessments measuring functional capacity and functional capability in patients with Duchenne muscular dystrophy.

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne Muscular Dystrophy; DMD; Accelerometer; ActiGraph; DEXA; muscle ultrasound

Outcome Measures

Primary Outcomes (1)

• Correlation between physical activity(VM) and muscle quantitative index

  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.

  Baseline up to Week 48

Secondary Outcomes (11)

• Correlation between physical activity and Vignos scale

  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 NSAA(The North Star Ambulatory Assessment)

  Baseline up to Week 48

• Correlation between physical activity and PUL(Performance of Upper Limb module for DMD)

  Baseline up to Week 48

Study Arms (1)

Duchenne Muscular Dystrophy

Children aged 5 to 18 years with Duchenne muscular dystrophy

Other: no intervention

Interventions

no intervention OTHER

no intervention

Duchenne Muscular Dystrophy

Eligibility Criteria

• Age: 5 Years - 18 Years
• Sex: all
• Age Groups: Child (0-17), Adult (18-64)
• 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.

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Samsung Medical Center: lead

Study Sites (2)

Samsung Medical Center

Seoul, 06135, South Korea

RECRUITING

Samsung Medical Center

Seoul, 06351, South Korea

RECRUITING

Related Publications (1)

• Im YJ, Choe Y, Lee J, Lee J, Do JG, Kwon JY. Quantitative Muscle Ultrasound: A Non-Invasive Biomarker for Monitoring Duchenne Muscular Dystrophy. Muscle Nerve. 2025 Oct;72(4):606-615. doi: 10.1002/mus.28469. Epub 2025 Jul 16.

  PMID: 40671379 DERIVED

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Jeong-yi Kwon, MD,Ph.D

  Samsung Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Yumi Choe

CONTACT

82-010-3173-2671; yumi.choe@sbri.co.kr

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: February 10, 2022
• First Posted: February 21, 2022
• Study Start: May 1, 2023
• Primary Completion: April 1, 2025
• Study Completion: October 1, 2025
• Last Updated: November 29, 2023

Record last verified: 2023-11

Locations

KR (2)