Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy
| Status: | Completed |
|---|---|
| Conditions: | Neurology |
| Therapuetic Areas: | Neurology |
| Healthy: | No |
| Age Range: | 5 - 15 |
| Updated: | 3/15/2019 |
| Start Date: | December 2013 |
| End Date: | March 6, 2019 |
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.
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.
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.
(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.
Inclusion Criteria:
- Male
- 5-15 years old at the time of enrollment
- Diagnosed with DMD (as defined by parent project)
- Written parental informed consent (and assent where appropriate) before any study
procedures take place
Exclusion Criteria:
- Contraindication to an MRI examination
- Presence of a secondary condition that impacts muscle function or metabolism, that
leads to developmental delay or impaired motor control, or that is not stable
- Participant is unable to comply with study requirements
- Congenital structural abnormality of the heart, repaired or unrepaired
- Clinically contraindicated participation
We found this trial at
1
site
Gainesville, Florida 32610
Principal Investigator: Barry Byrne, M.D., Ph.D.
Phone: 352-273-9615
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