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Trial record 29 of 103 for:    Recruiting, Not yet recruiting, Available Studies | "Muscular Dystrophies"

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 : March 13, 2018
Sponsor:
Collaborator:
Cure Duchenne
Information provided by (Responsible Party):
University of Florida

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.

Condition or disease Intervention/treatment
Muscular Dystrophy, Duchenne Other: Magnetic Resonance Imaging (MRI) Other: Pulmonary Function Testing (PFT) Other: Metabolic Exercise Testing using stationary bicycle Other: Echocardiogram

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.

Study Type : Observational
Estimated Enrollment : 12 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy
Study Start Date : December 2013
Estimated Primary Completion Date : March 2019
Estimated Study Completion Date : March 2019


Group/Cohort Intervention/treatment
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.

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




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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.

  2. 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).

  3. 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.



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Ages Eligible for Study:   5 Years to 15 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
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.
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02195999


Contacts
Contact: Tina Cousins, B.S. 3522739615 couscm@peds.ufl.edu

Locations
United States, Florida
Clinical and Translational Research Building Recruiting
Gainesville, Florida, United States, 32610
Contact: Tina Cousins, BS, RDCS    352-273-9615    couscm@peds.ufl.edu   
Principal Investigator: Barry Byrne, M.D., Ph.D.         
Sub-Investigator: Barbara Smith, PhD, PT         
Sub-Investigator: Anatole Martin, PhD, PT         
Sponsors and Collaborators
University of Florida
Cure Duchenne
Investigators
Principal Investigator: Barry Byrne, MD, PhD University of Florida

Responsible Party: University of Florida
ClinicalTrials.gov Identifier: NCT02195999     History of Changes
Other Study ID Numbers: IRB201300420
First Posted: July 21, 2014    Key Record Dates
Last Update Posted: March 13, 2018
Last Verified: March 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked