<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/NCT03001180</url>
  </required_header>
  <id_info>
    <org_study_id>CCHMC-LMI</org_study_id>
    <nct_id>NCT03001180</nct_id>
  </id_info>
  <brief_title>Identification of Biomarkers for Patients With Vascular Anomalies</brief_title>
  <official_title>Identification of Biomarkers for Patients With Vascular Anomalies</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Children's Hospital Medical Center, Cincinnati</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Lymphangiomatosis and Gorham's Disease Alliance (LGDA)</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>Klippel Trenaunay (KT) Support Group</agency>
      <agency_class>Other</agency_class>
    </collaborator>
    <collaborator>
      <agency>CLOVES Syndrome Community Support Group</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Children's Hospital Medical Center, Cincinnati</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The study will use blood (serum and plasma) and tissue obtained from participants undergoing&#xD;
      prescribed surgical resection of vascular anomalies of interest proposed in this study. The&#xD;
      study will also use blood (serum and plasma) and tissue collected and stored in a tissue bank&#xD;
      maintained by the Department of Hematology/Oncology.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      While vascular anomalies are rare diseases, they can be life-threatening and devastating to&#xD;
      affected children and their families. Advances in diagnosis, monitoring and therapies will be&#xD;
      significantly improved if non-invasive biomarkers that are sensitive and specific can be&#xD;
      identified. Obtaining a tissue biopsy to help in diagnosis can actually worsen the disease&#xD;
      and so identification of specific blood biomarkers is highly desirable. Studies will measure&#xD;
      angiogenic factors in serum and plasma samples at baseline and on therapy. Tissue removed&#xD;
      during surgical resection or blood removed prior to sclerotherapy will be used to obtain&#xD;
      cells and tissue for the assessment of where biomarkers are coming from and to identify&#xD;
      disease-causing pathways for new therapeutic targeting.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date>April 2015</start_date>
  <completion_date type="Anticipated">December 2050</completion_date>
  <primary_completion_date type="Anticipated">December 2050</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Case-Control</observational_model>
    <time_perspective>Cross-Sectional</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Correlation of Biomarkers with Differential Diagnosis</measure>
    <time_frame>An Average of Every 2 Years</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Correlation of Biomarkers with Disease Severity</measure>
    <time_frame>An Average of Every 2 Years</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Correlation of Biomarkers with Response to Therapies</measure>
    <time_frame>An Average of Every 2 Years</time_frame>
  </primary_outcome>
  <enrollment type="Anticipated">1000</enrollment>
  <condition>Vascular Anomaly</condition>
  <condition>Generalized Lymphatic Anomaly</condition>
  <condition>Kaposiform Hemangioendothelioma</condition>
  <condition>Kaposiform Lymphangiomatosis</condition>
  <condition>Gorham-Stout Disease</condition>
  <condition>Klippel Trenaunay Syndrome</condition>
  <condition>Congenital Lipomatous Overgrowth, Vascular Malformations, and Epidermal Nevi</condition>
  <biospec_retention>Samples With DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      Tissue, Blood, Plasma, and Serum Samples&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Any participant having labs drawn as standard of care will have blood drawn for the study&#xD;
        if consented/assented. All participants who are undergoing a surgical procedure or&#xD;
        sclerotherapy are currently consented for participation in the tissue bank. The principle&#xD;
        investigator will be looking at tissue from these participants. There is no further&#xD;
        recruitment for the study.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Any patient having labs drawn as standard of care will have blood drawn for the study&#xD;
             if consented/ assented.&#xD;
&#xD;
          -  All patients who are undergoing a surgical procedure or sclerotherapy are currently&#xD;
             consented for participation in the tissue bank.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  N/A&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>1 Day</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Timothy LeCras, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Children's Hospital Medical Center, Cincinnati</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Timothy LeCras, PhD</last_name>
    <phone>5138034862</phone>
    <email>hvmcresearch@cchmc.org</email>
  </overall_contact>
  <location>
    <facility>
      <name>Boston Children's Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Terminated</status>
  </location>
  <location>
    <facility>
      <name>Cincinnati Children's Hospital Medical Center</name>
      <address>
        <city>Cincinnati</city>
        <state>Ohio</state>
        <zip>45229</zip>
        <country>United States</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Megan Metcalf</last_name>
      <phone>513-803-2606</phone>
      <email>hvmcresearch@cchmc.org</email>
    </contact>
    <investigator>
      <last_name>Timothy LeCras, PhD</last_name>
      <role>Principal Investigator</role>
    </investigator>
    <investigator>
      <last_name>Adrienne Hammill, MD, PhD</last_name>
      <role>Sub-Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>March 2023</verification_date>
  <study_first_submitted>December 15, 2016</study_first_submitted>
  <study_first_submitted_qc>December 20, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 22, 2016</study_first_posted>
  <last_update_submitted>March 9, 2023</last_update_submitted>
  <last_update_submitted_qc>March 9, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">March 13, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Generalized Lymphatic Anomaly</keyword>
  <keyword>Vascular Anomaly</keyword>
  <keyword>Kaposiform Hemangioendothelioma</keyword>
  <keyword>Kaposiform Lymphangiomatosis</keyword>
  <keyword>Vascular Endothelial Growth Factor</keyword>
  <keyword>Biomarkers</keyword>
  <keyword>GLA</keyword>
  <keyword>KLA</keyword>
  <keyword>KHE</keyword>
  <keyword>GSD</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Hemangioendothelioma</mesh_term>
    <mesh_term>Osteolysis, Essential</mesh_term>
    <mesh_term>Vascular Malformations</mesh_term>
    <mesh_term>Klippel-Trenaunay-Weber Syndrome</mesh_term>
    <mesh_term>Congenital Abnormalities</mesh_term>
  </condition_browse>
  <!-- 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/NCT03001180
org study id: CCHMC-LMI
nct id: NCT03001180
lead sponsor: 
collaborator: 
collaborator: 
collaborator: 
has dmc: No
The study will use blood (serum and plasma) and tissue obtained from participants undergoing
      prescribed surgical resection of vascular anomalies of interest proposed in this study. The
      study will also use blood (serum and plasma) and tissue collected and stored in a tissue bank
      maintained by the Department of Hematology/Oncology.
While vascular anomalies are rare diseases, they can be life-threatening and devastating to
      affected children and their families. Advances in diagnosis, monitoring and therapies will be
      significantly improved if non-invasive biomarkers that are sensitive and specific can be
      identified. Obtaining a tissue biopsy to help in diagnosis can actually worsen the disease
      and so identification of specific blood biomarkers is highly desirable. Studies will measure
      angiogenic factors in serum and plasma samples at baseline and on therapy. Tissue removed
      during surgical resection or blood removed prior to sclerotherapy will be used to obtain
      cells and tissue for the assessment of where biomarkers are coming from and to identify
      disease-causing pathways for new therapeutic targeting.
observational model: Case-Control
time perspective: Cross-Sectional
measure: Correlation of Biomarkers with Differential Diagnosis
time frame: An Average of Every 2 Years
measure: Correlation of Biomarkers with Disease Severity
time frame: An Average of Every 2 Years
measure: Correlation of Biomarkers with Response to Therapies
time frame: An Average of Every 2 Years
Tissue, Blood, Plasma, and Serum Samples
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 1 Day
maximum age: N/A
healthy volunteers: No
last name: Timothy LeCras, PhD
role: Principal Investigator
affiliation: Children's Hospital Medical Center, Cincinnati
last name: Timothy LeCras, PhD
phone: 5138034862
email: hvmcresearch@cchmc.org
facility: 
status: Terminated
facility: 
status: Recruiting
contact: 
investigator: 
investigator: 
country: United States
responsible party type: Sponsor
mesh term: Hemangioendothelioma
mesh term: Osteolysis, Essential
mesh term: Vascular Malformations
mesh term: Klippel-Trenaunay-Weber Syndrome
mesh term: Congenital Abnormalities