<clinical_study>
  <!-- This xml conforms to an XML Schema at:
    https://clinicaltrials.gov/ct2/html/images/info/public.xsd -->
  <required_header>
    <download_date>ClinicalTrials.gov processed this data on September 20, 2023</download_date>
    <link_text>Link to the current ClinicalTrials.gov record.</link_text>
    <url>https://clinicaltrials.gov/ct2/show/NCT02195999</url>
  </required_header>
  <id_info>
    <org_study_id>IRB201300420</org_study_id>
    <nct_id>NCT02195999</nct_id>
  </id_info>
  <brief_title>Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy</brief_title>
  <acronym>CPI</acronym>
  <official_title>Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>University of Florida</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>CureDuchenne</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>University of Florida</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This study seeks to develop and validate non-invasive assessments of cardiac and respiratory&#xD;
      muscles with magnetic resonance imaging (MRI) to better predict the natural disease&#xD;
      progression of Duchenne muscular dystrophy (DMD) in affected individuals over time, as well&#xD;
      as determine whether peripheral skeletal muscle dysfunction can predict cardiopulmonary&#xD;
      dysfunction. The central hypothesis is that non-invasive MRI measures of the heart, muscle,&#xD;
      and peripheral skeletal muscles can sensitively predict future cardiopulmonary decline.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Magnetic Resonance Imaging (MRI) of the heart and breathing muscles, special breathing tests&#xD;
      (called pulmonary function testing), special exercise tests (using a stationary bike), and&#xD;
      possibly an echocardiogram (ultrasound of the heart, commonly known as an &quot;echo&quot;) will be&#xD;
      completed up to 4 times per year for up to 4 years. Most participants will complete testing&#xD;
      once or twice each year; however, some participants will be asked to repeat some of the tests&#xD;
      twice during each visit.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>December 2013</start_date>
  <completion_date type="Actual">March 6, 2019</completion_date>
  <primary_completion_date type="Actual">March 6, 2019</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Magnetic Resonance (MRI) T2 and Magnetic Resonance Spectroscopy (MRS)</measure>
    <time_frame>up to 4 years</time_frame>
    <description>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.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Pulmonary Function Testing (PFT)</measure>
    <time_frame>up to 4 years</time_frame>
    <description>Non-invasive breathing tests that characterize respiratory muscle function, as well as lung compliance and physiology.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Metabolic Exercise Testing (exercise capacity and MVO2)</measure>
    <time_frame>up to 4 years</time_frame>
    <description>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).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Multiple-echo Dixon</measure>
    <time_frame>up to 4 years</time_frame>
    <description>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.</description>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">9</enrollment>
  <condition>Muscular Dystrophy, Duchenne</condition>
  <arm_group>
    <arm_group_label>Individuals with DMD</arm_group_label>
    <description>Magnetic Resonance Imaging is a non-invasive method to determine ventricular size, volumes, mass, and ejection fraction.&#xD;
Pulmonary Function testing (PFT) are a series of non-invasive breathing tests that characterize respiratory muscle function, as well as lung compliance and physiology.&#xD;
Metabolic exercise testing using stationary bicycle (exercise capacity and MVO2) evaluates global cardiopulmonary functional status.&#xD;
Echocardiogram with multiple-echo Dixon method helps to assess cross-sectional and longitudinal variations in myocardial structure.</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Magnetic Resonance Imaging (MRI)</intervention_name>
    <description>It is a non-invasive method to determine ventricular size, volumes, mass, and ejection fraction.</description>
    <arm_group_label>Individuals with DMD</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Pulmonary Function Testing (PFT)</intervention_name>
    <description>It is non-invasive breathing tests that characterize respiratory muscle function, as well as lung compliance and physiology.</description>
    <arm_group_label>Individuals with DMD</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Metabolic Exercise Testing using stationary bicycle</intervention_name>
    <description>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.</description>
    <arm_group_label>Individuals with DMD</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Echocardiogram</intervention_name>
    <description>The echocardiogram performed with the multiple-echo Dixon method helps to assess participants cross-sectionally and longitudinally for variations and changes in myocardial structure.</description>
    <arm_group_label>Individuals with DMD</arm_group_label>
    <other_name>Multiple-echo Dixon</other_name>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        As the focus of this study is on validating novel measures of cardiac and respiratory&#xD;
        function in individuals with DMD, this study will utilize a single-group design with up to&#xD;
        60 males with DMD between 5 and 15 years old upon entry to the study. The participant&#xD;
        selection is limited to children since the detrimental effects of DMD begin in early&#xD;
        childhood, and the life expectancy of these boys is shortened to the early- to mid-20s.&#xD;
        Only males will be eligible to participate in the study because DMD is an X-linked&#xD;
        recessive genetic disorder that only leads to the characteristic disease in males. Although&#xD;
        females may be carriers, they do not exhibit the same phenotype as males.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Male&#xD;
&#xD;
          -  5-15 years old at the time of enrollment&#xD;
&#xD;
          -  Diagnosed with DMD (as defined by parent project)&#xD;
&#xD;
          -  Written parental informed consent (and assent where appropriate) before any study&#xD;
             procedures take place&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Contraindication to an MRI examination&#xD;
&#xD;
          -  Presence of a secondary condition that impacts muscle function or metabolism, that&#xD;
             leads to developmental delay or impaired motor control, or that is not stable&#xD;
&#xD;
          -  Participant is unable to comply with study requirements&#xD;
&#xD;
          -  Congenital structural abnormality of the heart, repaired or unrepaired&#xD;
&#xD;
          -  Clinically contraindicated participation&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <minimum_age>5 Years</minimum_age>
    <maximum_age>15 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Barry Byrne, MD, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>University of Florida</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Clinical and Translational Research Building</name>
      <address>
        <city>Gainesville</city>
        <state>Florida</state>
        <zip>32610</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>July 2019</verification_date>
  <study_first_submitted>July 17, 2014</study_first_submitted>
  <study_first_submitted_qc>July 17, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">July 21, 2014</study_first_posted>
  <last_update_submitted>July 3, 2019</last_update_submitted>
  <last_update_submitted_qc>July 3, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">July 8, 2019</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Muscular Dystrophies</mesh_term>
    <mesh_term>Muscular Dystrophy, Duchenne</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT02195999
org study id: IRB201300420
nct id: NCT02195999
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
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 model: Cohort
time perspective: Prospective
measure: Magnetic Resonance (MRI) T2 and Magnetic Resonance Spectroscopy (MRS)
time frame: up to 4 years
description: 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.
measure: Pulmonary Function Testing (PFT)
time frame: up to 4 years
description: Non-invasive breathing tests that characterize respiratory muscle function, as well as lung compliance and physiology.
measure: Metabolic Exercise Testing (exercise capacity and MVO2)
time frame: up to 4 years
description: 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).
measure: Multiple-echo Dixon
time frame: up to 4 years
description: 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.
arm group label: Individuals with DMD
description: 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.
intervention type: Other
intervention name: Magnetic Resonance Imaging (MRI)
description: It is a non-invasive method to determine ventricular size, volumes, mass, and ejection fraction.
arm group label: Individuals with DMD
intervention type: Other
intervention name: Pulmonary Function Testing (PFT)
description: It is non-invasive breathing tests that characterize respiratory muscle function, as well as lung compliance and physiology.
arm group label: Individuals with DMD
intervention type: Other
intervention name: Metabolic Exercise Testing using stationary bicycle
description: 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.
arm group label: Individuals with DMD
intervention type: Other
intervention name: Echocardiogram
description: The echocardiogram performed with the multiple-echo Dixon method helps to assess participants cross-sectionally and longitudinally for variations and changes in myocardial structure.
arm group label: Individuals with DMD
other name: Multiple-echo Dixon
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: Male
minimum age: 5 Years
maximum age: 15 Years
healthy volunteers: No
last name: Barry Byrne, MD, PhD
role: Principal Investigator
affiliation: University of Florida
facility: 
country: United States
responsible party type: Sponsor
mesh term: Muscular Dystrophies
mesh term: Muscular Dystrophy, Duchenne
sharing ipd: No