Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy

Muscular Dystrophy, Duchenne Clinical Trial

— CPI  

Official title:

Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02195999
• Other study ID #: IRB201300420
• Status: Completed
• Start date: December 2013
• Est. completion date: March 6, 2019

Study information

• Verified date: July 2019
• Source: University of Florida
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 9
• Est. completion date: March 6, 2019
• Est. primary completion date: March 6, 2019
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 5 Years to 15 Years

Eligibility

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

Related Conditions & MeSH terms

• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Other:
• Magnetic Resonance Imaging (MRI)
It is a non-invasive method to determine ventricular size, volumes, mass, and ejection fraction.
• Pulmonary Function Testing (PFT)
It is non-invasive breathing tests that characterize respiratory muscle function, as well as lung compliance and physiology.
• 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.
• 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.

Locations

Country	Name	City	State
United States	Clinical and Translational Research Building	Gainesville	Florida

Sponsors (2)

Lead Sponsor	Collaborator
University of Florida	CureDuchenne

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Magnetic Resonance (MRI) T2 and Magnetic Resonance Spectroscopy (MRS)	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.	up to 4 years
Secondary	Pulmonary Function Testing (PFT)	Non-invasive breathing tests that characterize respiratory muscle function, as well as lung compliance and physiology.	up to 4 years
Secondary	Metabolic Exercise Testing (exercise capacity and MVO2)	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).	up to 4 years
Secondary	Multiple-echo Dixon	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.	up to 4 years