<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/NCT01730755</url>
  </required_header>
  <id_info>
    <org_study_id>I-RUX-12-03</org_study_id>
    <nct_id>NCT01730755</nct_id>
  </id_info>
  <brief_title>Ruxolitinib for Chuvash Polycythemia</brief_title>
  <official_title>Ruxolitinib for Chuvash Polycythemia</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Washington University School of Medicine</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Incyte Corporation</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>Washington University School of Medicine</source>
  <brief_summary>
    <textblock>
      Chuvash polycythemia (CP) is a rare form of congenital polycythemia caused by mutations in&#xD;
      the VHL gene. Currently, there are no therapies that have proven effective for CP. Recent&#xD;
      studies have demonstrated that VHL (von Hippel-Lindau tumor suppressor) regulates the&#xD;
      activity of JAK2 (Janus kinase 2). In mouse models, inhibition of JAK2 reverses the CP&#xD;
      phenotype. Therefore, the investigators hypothesize that JAK2 inhibition may have significant&#xD;
      clinical benefits for CP patients.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Study involvement will last for 48 weeks. There will be approximately 11 visits through week&#xD;
      48. Visits may take up to 2-3 hours to complete and occur every 4 weeks for the first 24&#xD;
      weeks, then every 12 weeks until week 48.&#xD;
&#xD;
      During each study visit, any or all of the following procedures may occur:&#xD;
&#xD;
        -  List current medications and participant general health&#xD;
&#xD;
        -  Obtain blood pressure, body weight, body temperature, respiratory rate and heart rate&#xD;
&#xD;
        -  Measure Spleen by examination&#xD;
&#xD;
        -  Obtain an abdominal MRI to evaluate spleen and any pre-existing or new blood clots&#xD;
&#xD;
        -  Obtain blood samples for safety tests and to monitor kidney/liver function.&#xD;
&#xD;
        -  Questionnaires for participant to complete regarding symptoms related to disease.&#xD;
&#xD;
        -  Ruxolitinib dosing may be increased after 4 weeks if needed. The dose of the ruxolitinib&#xD;
           may be reduced related to side effects.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>No longer available</overall_status>
  <study_type>Expanded Access</study_type>
  <has_expanded_access>No</has_expanded_access>
  <condition>Erythrocytosis, Familial, 2</condition>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Ruxolitinib</intervention_name>
    <description>Ruxolitinib 10 mg tablets twice daily</description>
    <other_name>Jakafi</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Diagnosis of Chuvash polycythemia&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Unable to comprehend or unwilling to sign an informed consent form.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>N/A</maximum_age>
  </eligibility>
  <location>
    <facility>
      <name>Washington University School of Medicine</name>
      <address>
        <city>Saint Louis</city>
        <state>Missouri</state>
        <zip>63110</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>May 2018</verification_date>
  <study_first_submitted>November 7, 2012</study_first_submitted>
  <study_first_submitted_qc>November 15, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">November 21, 2012</study_first_posted>
  <last_update_submitted>May 7, 2018</last_update_submitted>
  <last_update_submitted_qc>May 7, 2018</last_update_submitted_qc>
  <last_update_posted type="Actual">May 14, 2018</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Polycythemia</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Polycythemia</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/NCT01730755
org study id: I-RUX-12-03
nct id: NCT01730755
lead sponsor: 
collaborator: 
Chuvash polycythemia (CP) is a rare form of congenital polycythemia caused by mutations in
      the VHL gene. Currently, there are no therapies that have proven effective for CP. Recent
      studies have demonstrated that VHL (von Hippel-Lindau tumor suppressor) regulates the
      activity of JAK2 (Janus kinase 2). In mouse models, inhibition of JAK2 reverses the CP
      phenotype. Therefore, the investigators hypothesize that JAK2 inhibition may have significant
      clinical benefits for CP patients.
Study involvement will last for 48 weeks. There will be approximately 11 visits through week
      48. Visits may take up to 2-3 hours to complete and occur every 4 weeks for the first 24
      weeks, then every 12 weeks until week 48.

      During each study visit, any or all of the following procedures may occur:

        -  List current medications and participant general health

        -  Obtain blood pressure, body weight, body temperature, respiratory rate and heart rate

        -  Measure Spleen by examination

        -  Obtain an abdominal MRI to evaluate spleen and any pre-existing or new blood clots

        -  Obtain blood samples for safety tests and to monitor kidney/liver function.

        -  Questionnaires for participant to complete regarding symptoms related to disease.

        -  Ruxolitinib dosing may be increased after 4 weeks if needed. The dose of the ruxolitinib
           may be reduced related to side effects.
intervention type: Drug
intervention name: Ruxolitinib
description: Ruxolitinib 10 mg tablets twice daily
other name: Jakafi
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
facility: 
country: United States
responsible party type: Sponsor
mesh term: Polycythemia