<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/NCT03001388</url>
  </required_header>
  <id_info>
    <org_study_id>ELASTIC-CF</org_study_id>
    <secondary_id>U01DK062456</secondary_id>
    <nct_id>NCT03001388</nct_id>
  </id_info>
  <brief_title>Longitudinal Assessment of Transient Elastography in Cystic Fibrosis</brief_title>
  <official_title>Longitudinal Assessment of Transient Elastography in Cystic Fibrosis</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>
      To determine if transient elastography (TE), when combined with ultrasound (US) pattern&#xD;
      characterization can improve the prediction of progression to a nodular pattern on US.&#xD;
&#xD;
      To confirm the feasibility of obtaining TE measurements in children with Cystic Fibrosis (CF)&#xD;
      To prospectively assess whether TE data are associated with conventional laboratory markers&#xD;
      of hepatic fibrosis To determine the variability of TE measurements taken at different sites&#xD;
      in the same patient&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      A noninvasive assessment of hepatic fibrosis is desperately needed to advance the care of&#xD;
      children with CF significant liver disease and to provide for measurements during clinical&#xD;
      trials. That global assessment might serve as both a predictor/descriptor of disease course&#xD;
      but also as a critical biomarker for clinical research. FibroScan® measurement of liver&#xD;
      stiffness has great potential to fill this void. The underlying hypothesis of this proposal&#xD;
      is that elastography in addition to US can improved the prediction of the development of a&#xD;
      nodular liver on US and development of portal hypertension over time in children and young&#xD;
      adults with CF.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">April 1, 2017</start_date>
  <completion_date type="Anticipated">June 2023</completion_date>
  <primary_completion_date type="Anticipated">June 2023</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>Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM</measure>
    <time_frame>Baseline</time_frame>
    <description>Liver stiffness&quot; quantifies liver fibrosis and is measured in kPa (median of 10 subsequent valid measurements and are deemed acceptable if the ratio of interquartile range and median is &lt;30% and success rate is &gt;60%, meaning 10 valid measurements are obtained within 16 attempts).</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM</measure>
    <time_frame>Year 1</time_frame>
    <description>Liver stiffness&quot; quantifies liver fibrosis and is measured in kPa (median of 10 subsequent valid measurements and are deemed acceptable if the ratio of interquartile range and median is &lt;30% and success rate is &gt;60%, meaning 10 valid measurements are obtained within 16 attempts).</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM</measure>
    <time_frame>Year 2</time_frame>
    <description>Liver stiffness&quot; quantifies liver fibrosis and is measured in kPa (median of 10 subsequent</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Liver steatosis obtained via transient elastography</measure>
    <time_frame>Baseline, Year 1, Year 2</time_frame>
    <description>Controlled Attenuation Parameter (CAP)&quot; quantifies liver steatosis and is measured in dB</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Comparison of liver stiffness and liver steatosis measurements at each time point to grayscale ultrasound grades</measure>
    <time_frame>Baseline, Year 1 and Year 2</time_frame>
    <description>Grayscale ultrasound grades will be obtained from the parent study (PUSH: Clinical Trials: NCT01144507)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Comparison of liver stiffness and liver steatosis measurements at each time point to clinical findings of portal hypertension</measure>
    <time_frame>Baseline, Year 1 and Year 2</time_frame>
    <description>Grayscale ultrasound grades will be obtained from the parent study (PUSH: Clinical Trials: NCT01144507)</description>
  </secondary_outcome>
  <other_outcome>
    <measure>Invalid measurements and success rate on liver stiffness and liver steatosis measurements</measure>
    <time_frame>Baseline, Year 1 and Year 2</time_frame>
  </other_outcome>
  <other_outcome>
    <measure>Inter quartile range (IQR) (kPa or dB/m) of all valid measurements within the examination</measure>
    <time_frame>Baseline, Year 1 and Year 2</time_frame>
    <description>To reflect the dispersion of stiffness and steatosis</description>
  </other_outcome>
  <other_outcome>
    <measure>IQR/median ratio of all valid measurements within the examination</measure>
    <time_frame>Baseline, Year 1 and Year 2</time_frame>
    <description>This quantity should remain as low as possible to ensure reliable results (goal &lt; 30%)</description>
  </other_outcome>
  <enrollment type="Actual">141</enrollment>
  <condition>Cystic Fibrosis Liver Disease</condition>
  <intervention>
    <intervention_type>Procedure</intervention_type>
    <intervention_name>Transient Elastography (TE)</intervention_name>
    <description>Participants enrolled in Cystic Fibrosis Liver Disease Network (CFLD NET)Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis (PUSH) study in longitudinal follow up at centers with Fibroscan available (currently 8/11 centers)</description>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Participants enrolled in CFLD NET PUSH study in longitudinal follow up at centers with&#xD;
        Fibroscan available (currently 8/11 centers) All racial and ethnic groups will be included.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Participants enrolled in CFLD NET PUSH study in longitudinal follow up at centers with&#xD;
             Fibroscan available (currently 8/11 centers)&#xD;
&#xD;
          -  Entry criteria for that study were:&#xD;
&#xD;
          -  CF as determined by sweat chloride &gt;60 meq/l&#xD;
&#xD;
          -  Pancreatic insufficiency&#xD;
&#xD;
          -  Age 3-12 years old at entry&#xD;
&#xD;
          -  For entry into the longitudinal follow up subjects were in one of two groups&#xD;
&#xD;
               -  A screening US pattern of nodular liver (CIR), heterogeneous increased&#xD;
                  echogenicity (HTG) or homogeneous increased echogenicity (HMG)&#xD;
&#xD;
               -  A screening US pattern of normal (NL) matched to a HTG subject (2 NL:1HTG) by&#xD;
                  age, center and pseudomonas status&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Exited from the PUSH Study&#xD;
&#xD;
          -  Unable / unwilling to sign consent&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>6 Years</minimum_age>
    <maximum_age>26 Years</maximum_age>
    <healthy_volunteers>Accepts Healthy Volunteers</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>Ann &amp; Robert H. Lurie Children's</name>
      <address>
        <city>Chicago</city>
        <state>Illinois</state>
        <zip>60611</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>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>
    <facility>
      <name>The Hospital for Sick Children</name>
      <address>
        <city>Toronto</city>
        <state>Ontario</state>
        <zip>M5G 1X8</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Canada</country>
    <country>United States</country>
  </location_countries>
  <link>
    <url>https://childrennetwork.org</url>
    <description>ChiLDReN Website</description>
  </link>
  <verification_date>March 2022</verification_date>
  <study_first_submitted>November 18, 2016</study_first_submitted>
  <study_first_submitted_qc>December 19, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 23, 2016</study_first_posted>
  <last_update_submitted>March 23, 2022</last_update_submitted>
  <last_update_submitted_qc>March 23, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">March 24, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Cystic Fibrosis Liver Disease</keyword>
  <keyword>Pancreatic insufficiency</keyword>
  <keyword>Cystic Fibrosis</keyword>
  <keyword>Transient Elastography</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>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/NCT03001388
org study id: ELASTIC-CF
secondary id: U01DK062456
nct id: NCT03001388
lead sponsor: 
collaborator: 
has dmc: Yes
To determine if transient elastography (TE), when combined with ultrasound (US) pattern
      characterization can improve the prediction of progression to a nodular pattern on US.

      To confirm the feasibility of obtaining TE measurements in children with Cystic Fibrosis (CF)
      To prospectively assess whether TE data are associated with conventional laboratory markers
      of hepatic fibrosis To determine the variability of TE measurements taken at different sites
      in the same patient
A noninvasive assessment of hepatic fibrosis is desperately needed to advance the care of
      children with CF significant liver disease and to provide for measurements during clinical
      trials. That global assessment might serve as both a predictor/descriptor of disease course
      but also as a critical biomarker for clinical research. FibroScan® measurement of liver
      stiffness has great potential to fill this void. The underlying hypothesis of this proposal
      is that elastography in addition to US can improved the prediction of the development of a
      nodular liver on US and development of portal hypertension over time in children and young
      adults with CF.
observational model: Cohort
time perspective: Prospective
measure: Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM
time frame: Baseline
description: Liver stiffness" quantifies liver fibrosis and is measured in kPa (median of 10 subsequent valid measurements and are deemed acceptable if the ratio of interquartile range and median is <30% and success rate is >60%, meaning 10 valid measurements are obtained within 16 attempts).
measure: Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM
time frame: Year 1
description: Liver stiffness" quantifies liver fibrosis and is measured in kPa (median of 10 subsequent valid measurements and are deemed acceptable if the ratio of interquartile range and median is <30% and success rate is >60%, meaning 10 valid measurements are obtained within 16 attempts).
measure: Liver Stiffness Measurement (LSM) obtained via transient elastography using FibroScanTM
time frame: Year 2
description: Liver stiffness" quantifies liver fibrosis and is measured in kPa (median of 10 subsequent
measure: Liver steatosis obtained via transient elastography
time frame: Baseline, Year 1, Year 2
description: Controlled Attenuation Parameter (CAP)" quantifies liver steatosis and is measured in dB
measure: Comparison of liver stiffness and liver steatosis measurements at each time point to grayscale ultrasound grades
time frame: Baseline, Year 1 and Year 2
description: Grayscale ultrasound grades will be obtained from the parent study (PUSH: Clinical Trials: NCT01144507)
measure: Comparison of liver stiffness and liver steatosis measurements at each time point to clinical findings of portal hypertension
time frame: Baseline, Year 1 and Year 2
description: Grayscale ultrasound grades will be obtained from the parent study (PUSH: Clinical Trials: NCT01144507)
measure: Invalid measurements and success rate on liver stiffness and liver steatosis measurements
time frame: Baseline, Year 1 and Year 2
measure: Inter quartile range (IQR) (kPa or dB/m) of all valid measurements within the examination
time frame: Baseline, Year 1 and Year 2
description: To reflect the dispersion of stiffness and steatosis
measure: IQR/median ratio of all valid measurements within the examination
time frame: Baseline, Year 1 and Year 2
description: This quantity should remain as low as possible to ensure reliable results (goal < 30%)
intervention type: Procedure
intervention name: Transient Elastography (TE)
description: Participants enrolled in Cystic Fibrosis Liver Disease Network (CFLD NET)Prediction by Ultrasound of the Risk of Hepatic Cirrhosis in Cystic Fibrosis (PUSH) study in longitudinal follow up at centers with Fibroscan available (currently 8/11 centers)
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 6 Years
maximum age: 26 Years
healthy volunteers: Accepts Healthy Volunteers
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: 
facility: 
country: Canada
country: United States
url: https://childrennetwork.org
description: ChiLDReN Website
responsible party type: Sponsor
mesh term: Cystic Fibrosis
mesh term: Liver Diseases
mesh term: Fibrosis
sharing ipd: No