NCT02195999

Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 9 (actual)
• Sponsor: University of Florida · Academic / Other
• Sex: Male
• Age: 5 Years – 15 Years
• Healthy volunteers: Not accepted

Summary

This study seeks to develop and validate non-invasive assessments of cardiac and respiratory muscles with magnetic resonance imaging (MRI) to better predict the natural disease progression of Duchenne muscular dystrophy (DMD) in affected individuals over time, as well as determine whether peripheral skeletal muscle dysfunction can predict cardiopulmonary dysfunction. The central hypothesis is that non-invasive MRI measures of the heart, muscle, and peripheral skeletal muscles can sensitively predict future cardiopulmonary decline.

Detailed description

Magnetic Resonance Imaging (MRI) of the heart and breathing muscles, special breathing tests (called pulmonary function testing), special exercise tests (using a stationary bike), and possibly an echocardiogram (ultrasound of the heart, commonly known as an "echo") will be completed up to 4 times per year for up to 4 years. Most participants will complete testing once or twice each year; however, some participants will be asked to repeat some of the tests twice during each visit.

Conditions

• Muscular Dystrophy, Duchenne

Interventions

Type	Name	Description
OTHER	Magnetic Resonance Imaging (MRI)	It is a non-invasive method to determine ventricular size, volumes, mass, and ejection fraction.
OTHER	Pulmonary Function Testing (PFT)	It is non-invasive breathing tests that characterize respiratory muscle function, as well as lung compliance and physiology.
OTHER	Metabolic Exercise Testing using stationary bicycle	Metabolic exercise testing, including assessment of exercise capacity and MVO2, evaluates global cardiopulmonary functional status. This is performed with the use of a stationary bicycle.
OTHER	Echocardiogram	The echocardiogram performed with the multiple-echo Dixon method helps to assess participants cross-sectionally and longitudinally for variations and changes in myocardial structure.

Timeline

• Start date: 2013-12-01
• Primary completion: 2019-03-06
• Completion: 2019-03-06
• First posted: 2014-07-21
• Last updated: 2019-07-08

Locations

1 site across 1 country: United States