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Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy (CPI)

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ClinicalTrials.gov Identifier: NCT02195999
Recruitment Status : Recruiting
First Posted : July 21, 2014
Last Update Posted : November 24, 2017
Sponsor:
Information provided by (Responsible Party):

July 17, 2014
July 21, 2014
November 24, 2017
December 2013
June 2018   (Final data collection date for primary outcome measure)
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.
Same as current
Complete list of historical versions of study NCT02195999 on ClinicalTrials.gov Archive Site
  • 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.
Same as current
Not Provided
Not Provided
 
Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy
Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy
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.
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.
Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
Not Provided
Non-Probability Sample
As the focus of this study is on validating novel measures of cardiac and respiratory function in individuals with DMD, this study will utilize a single-group design with up to 60 males with DMD between 5 and 15 years old upon entry to the study. The participant selection is limited to children since the detrimental effects of DMD begin in early childhood, and the life expectancy of these boys is shortened to the early- to mid-20s. Only males will be eligible to participate in the study because DMD is an X-linked recessive genetic disorder that only leads to the characteristic disease in males. Although females may be carriers, they do not exhibit the same phenotype as males.
Muscular Dystrophy, Duchenne
  • 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.
    Other Name: Multiple-echo Dixon
Individuals with DMD

Magnetic Resonance Imaging is a non-invasive method to determine ventricular size, volumes, mass, and ejection fraction.

Pulmonary Function testing (PFT) are a series of non-invasive breathing tests that characterize respiratory muscle function, as well as lung compliance and physiology.

Metabolic exercise testing using stationary bicycle (exercise capacity and MVO2) evaluates global cardiopulmonary functional status.

Echocardiogram with multiple-echo Dixon method helps to assess cross-sectional and longitudinal variations in myocardial structure.

Interventions:
  • Other: Magnetic Resonance Imaging (MRI)
  • Other: Pulmonary Function Testing (PFT)
  • Other: Metabolic Exercise Testing using stationary bicycle
  • Other: Echocardiogram
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
12
June 2018
June 2018   (Final data collection date for primary outcome measure)

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
Sexes Eligible for Study: Male
5 Years to 15 Years   (Child)
No
Contact: Tina Cousins, B.S. 3522739615 couscm@peds.ufl.edu
United States
 
 
NCT02195999
IRB201300420
No
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Plan to Share IPD: No
University of Florida
University of Florida
Not Provided
Principal Investigator: Barry Byrne, MD, PhD University of Florida
University of Florida
November 2017