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Completed

NCT Number: NCT02195999

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.

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Key information

Conditions

Muscular Dystrophy, Duchenne Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Muscular Diseases Muscular Disorders, Atrophic Muscular Dystrophies Musculoskeletal Diseases Nervous System Diseases Neuromuscular Diseases

Age range

5 year–15 year

Sex eligibility

Male

Study type

Observational

Primary location

Clinical and Translational Research Building

Gainesville, Florida, 32610, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

magnetic resonance imaging (MRI)

Other

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

Pulmonary Function Testing (PFT)

Other

It is non-invasive breathing tests that characterize respiratory muscle function, as well as lung compliance and physiology.

Metabolic Exercise Testing using stationary bicycle

Other

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

Other

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 names: Multiple-echo Dixon

Primary outcomes

  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 outcomes

  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.

Sponsors and collaborators

Lead sponsor

University of Florida

Other

Collaborators

  • CureDuchenne

Registry information

Acronym: CPI

Important dates

Study start
2013
Primary completion
2019
Study completion
2019
First posted
Jul 21, 2014
Registry last updated
Jul 8, 2019

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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