<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/NCT02979340</url>
  </required_header>
  <id_info>
    <org_study_id>CFLD-MRE</org_study_id>
    <secondary_id>U01DK062456</secondary_id>
    <nct_id>NCT02979340</nct_id>
  </id_info>
  <brief_title>MRI to Characterize and Predict CF Liver Disease in PUSH Cohort</brief_title>
  <official_title>Analysis of Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis (PUSH) Cohort Using Advanced MRI to Characterize and Predict CF Liver Disease</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)</agency>
      <agency_class>NIH</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Cystic Fibrosis Foundation</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      Specific Aim 1. Determine if valid results of non-sedated MRI based assessments of liver&#xD;
      stiffness and lipid content can be obtained in more than 90% of children and young adults&#xD;
      with cystic fibrosis.&#xD;
&#xD;
      Specific Aim 2. Determine hepatic lipid content using the HepaFat sequence and liver&#xD;
      stiffness using MRE. Investigators will compare the results obtained by MRI with PUSH study&#xD;
      grayscale ultrasounds in CF patients with normal, heterogeneous, homogeneous or nodular&#xD;
      (cirrhotic) pattern on ultrasound.&#xD;
&#xD;
      Specific Aim 3. Creation of an imaging core lab to centralize evaluation of MR imaging data,&#xD;
      allow for remote image upload, electronic data storage, and remote image&#xD;
      viewing/interpretation. This infrastructure will be utilized to standardize image post&#xD;
      processing.&#xD;
&#xD;
      Specific Aim 4. Using the longitudinal PUSH study, determine if MRI based imaging improves&#xD;
      discrimination of subjects at risk for progression to advanced CF liver disease (development&#xD;
      of cirrhosis) compared to using US imaging alone&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This proposal will be a cross sectional study of the current well-characterized PUSH cohort.&#xD;
      Patients will receive an MRI at the time of their scheduled PUSH ultrasound. Ideally, the MRI&#xD;
      will be performed the same day or a similar time point near liver ultrasound (+/-90 days, but&#xD;
      Investigators will allow a larger window due to the slow progression of fibrosis).&#xD;
&#xD;
      Blinded radiology review of imaging sequences will be performed by a local study radiologist&#xD;
      with central review performed by the Imaging Core. Analysis by the Imaging Core will include&#xD;
      calculation of the liver stiffness, liver and spleen volume, and hepatic and lipid content&#xD;
      for all MRI studies.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">December 2016</start_date>
  <completion_date type="Actual">December 31, 2019</completion_date>
  <primary_completion_date type="Actual">December 31, 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>Change over Time in Liver stiffness and Lipid content over time using MRE</measure>
    <time_frame>BSL, Year 1, Year 2</time_frame>
    <description>Analysis of variance (ANOVA) will be used to compare the distribution of liver stiffness (in kPa) and lipid content measures between all groups defined by PUSH study grayscale ultrasounds results over time.</description>
  </primary_outcome>
  <enrollment type="Actual">115</enrollment>
  <condition>Cystic Fibrosis Liver Disease</condition>
  <condition>Pancreatic Insufficiency</condition>
  <intervention>
    <intervention_type>Procedure</intervention_type>
    <intervention_name>MRI</intervention_name>
    <description>non-invasive assessment of hepatic fibrosis and steatosis</description>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Children aged 3 through 12 years of age at time of enrollment diagnosed with Cystic&#xD;
        Fibrosis and pancreatic insufficiency and enrolled in the CFLD PUSH longitudinal study.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        1. Currently enrolled in longitudinal follow up in PUSH study at a site with MR&#xD;
        elastography.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Age under 6 years (all current patients at time of opening of this study will be&#xD;
             greater than 6 years of age).&#xD;
&#xD;
          2. Internal appliance or hardware that is not compatible with MR.&#xD;
&#xD;
          3. Inability to obtain MRI within 6 months of US.&#xD;
&#xD;
          4. Inability to cooperate with MRI.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Years</minimum_age>
    <maximum_age>26 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Ed Doo, MD</last_name>
    <role>Study Director</role>
    <affiliation>National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Averell Sherker, MD</last_name>
    <role>Study Director</role>
    <affiliation>National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Children's Hospital Colorado</name>
      <address>
        <city>Aurora</city>
        <state>Colorado</state>
        <zip>80045</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Healthcare of Atlanta</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30322</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Riley Hospital for Children</name>
      <address>
        <city>Indianapolis</city>
        <state>Indiana</state>
        <zip>46202</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Johns Hopkins School of Medicine</name>
      <address>
        <city>Baltimore</city>
        <state>Maryland</state>
        <zip>21217</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Cincinnati Children's Hospital Medical</name>
      <address>
        <city>Cincinnati</city>
        <state>Ohio</state>
        <zip>45229</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Texas Children's Hospital</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Seattle Children's Hospital</name>
      <address>
        <city>Seattle</city>
        <state>Washington</state>
        <zip>98105</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>https://childrennetwork.org</url>
    <description>MRE is a study in the ChiLDReN Network</description>
  </link>
  <verification_date>July 2019</verification_date>
  <study_first_submitted>November 18, 2016</study_first_submitted>
  <study_first_submitted_qc>November 28, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 1, 2016</study_first_posted>
  <last_update_submitted>January 27, 2020</last_update_submitted>
  <last_update_submitted_qc>January 27, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">January 28, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Cystic Fibrosis Liver Disease</keyword>
  <keyword>Pancreatic Insufficiency</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Cystic Fibrosis</mesh_term>
    <mesh_term>Liver Diseases</mesh_term>
    <mesh_term>Exocrine Pancreatic Insufficiency</mesh_term>
    <mesh_term>Fibrosis</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/NCT02979340
org study id: CFLD-MRE
secondary id: U01DK062456
nct id: NCT02979340
lead sponsor: 
collaborator: 
has dmc: Yes
Specific Aim 1. Determine if valid results of non-sedated MRI based assessments of liver
      stiffness and lipid content can be obtained in more than 90% of children and young adults
      with cystic fibrosis.

      Specific Aim 2. Determine hepatic lipid content using the HepaFat sequence and liver
      stiffness using MRE. Investigators will compare the results obtained by MRI with PUSH study
      grayscale ultrasounds in CF patients with normal, heterogeneous, homogeneous or nodular
      (cirrhotic) pattern on ultrasound.

      Specific Aim 3. Creation of an imaging core lab to centralize evaluation of MR imaging data,
      allow for remote image upload, electronic data storage, and remote image
      viewing/interpretation. This infrastructure will be utilized to standardize image post
      processing.

      Specific Aim 4. Using the longitudinal PUSH study, determine if MRI based imaging improves
      discrimination of subjects at risk for progression to advanced CF liver disease (development
      of cirrhosis) compared to using US imaging alone
This proposal will be a cross sectional study of the current well-characterized PUSH cohort.
      Patients will receive an MRI at the time of their scheduled PUSH ultrasound. Ideally, the MRI
      will be performed the same day or a similar time point near liver ultrasound (+/-90 days, but
      Investigators will allow a larger window due to the slow progression of fibrosis).

      Blinded radiology review of imaging sequences will be performed by a local study radiologist
      with central review performed by the Imaging Core. Analysis by the Imaging Core will include
      calculation of the liver stiffness, liver and spleen volume, and hepatic and lipid content
      for all MRI studies.
observational model: Cohort
time perspective: Prospective
measure: Change over Time in Liver stiffness and Lipid content over time using MRE
time frame: BSL, Year 1, Year 2
description: Analysis of variance (ANOVA) will be used to compare the distribution of liver stiffness (in kPa) and lipid content measures between all groups defined by PUSH study grayscale ultrasounds results over time.
intervention type: Procedure
intervention name: MRI
description: non-invasive assessment of hepatic fibrosis and steatosis
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 6 Years
maximum age: 26 Years
healthy volunteers: No
last name: Ed Doo, MD
role: Study Director
affiliation: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
last name: Averell Sherker, MD
role: Study Director
affiliation: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: United States
url: https://childrennetwork.org
description: MRE is a study in the ChiLDReN Network
responsible party type: Sponsor
mesh term: Cystic Fibrosis
mesh term: Liver Diseases
mesh term: Exocrine Pancreatic Insufficiency
mesh term: Fibrosis
sharing ipd: No