Clinical Trial: Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

Official Title: Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy

Brief 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 Summary: 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.
Sponsor: University of Florida

Current Primary Outcome: Magnetic Resonance (MRI) T2 and Magnetic Resonance Spectroscopy (MRS) [ Time Frame: up to 4 years ]

The MRI T2 and MRS will be used as a noninvasive marker of myocardial damage/inflammation of participants of this study as an early detection for DMD.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Pulmonary Function Testing (PFT) [ Time Frame: up to 4 years ]
    Non-invasive breathing tests that characterize respiratory muscle function, as well as lung compliance and physiology.
  • Metabolic Exercise Testing (exercise capacity and MVO2) [ Time Frame: up to 4 years ]
    With the use of metabolic exercise testing, the aim is to correlate changes in cardiopulmonary function with decline in peripheral skeletal muscle function in individuals with DMD. Metabolic exercise testing includes measuring exercise capacity and maximum oxygen consumption (MVO2).
  • Multiple-echo Dixon [ Time Frame: up to 4 years ]
    The echocardiogram performed with the multiple-echo Dixon method helps to assess participants cross-sectionally and longitudinally for variations and changes in myocardial structure. This method and MRS will also be used for fat fraction determination.


Original Secondary Outcome: Same as current

Information By: University of Florida

Dates:
Date Received: July 17, 2014
Date Started: December 2013
Date Completion: December 2017
Last Updated: November 29, 2016
Last Verified: November 2016