<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/NCT04483531</url>
  </required_header>
  <id_info>
    <org_study_id>A4250-014</org_study_id>
    <nct_id>NCT04483531</nct_id>
  </id_info>
  <brief_title>Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis</brief_title>
  <official_title>Odevixibat (A4250) for the Treatment of Progressive Familial Intrahepatic Cholestasis (Expanded Access Program)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Albireo</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Albireo</source>
  <brief_summary>
    <textblock>
      To provide treatment access to patients with PFIC in the US who have pruritus and elevated&#xD;
      serum bile acids and who are not able to enroll in A4250-008 (PEDFIC2) for the following&#xD;
      reasons: 1) Do not meet eligibility criteria for PEDFIC 2; 2) Are not able to get to a PEDFIC&#xD;
      2 site for geographical reasons, and 3) Do meet the eligibility criteria for PEDFIC 2 after&#xD;
      recruitment has been completed&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Eligible patients will be enrolled into this expanded-access program and treated with an oral&#xD;
      dose of 120 μg/kg/day of odevixibat and evaluated on an ongoing basis.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Approved for marketing</overall_status>
  <study_type>Expanded Access</study_type>
  <has_expanded_access>No</has_expanded_access>
  <expanded_access_info>
    <expanded_access_type_treatment>Yes</expanded_access_type_treatment>
  </expanded_access_info>
  <condition>Progressive Familial Intrahepatic Cholestasis</condition>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Odevixibat</intervention_name>
    <description>A4250 is a small molecule and selective inhibitor of IBAT</description>
    <other_name>A4250</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. A male or female patient of any age, with a clinical diagnosis of PFIC, and with a&#xD;
             body weight ≥5 kg at Screening visit&#xD;
&#xD;
          2. Patient must have a clinical diagnosis of PFIC&#xD;
&#xD;
          3. Patient must have clinically confirmed pruritus&#xD;
&#xD;
          4. Patient must have elevated serum bile acid levels, specifically measured to be ≥2 ×&#xD;
             the upper limit of normal (ULN) prior to start of medication&#xD;
&#xD;
          5. Patient and/or legal guardian must sign informed consent (and assent) as appropriate.&#xD;
             Patients who turn 18 years of age (or legal age per country) during&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Patient is expected to have a liver transplant within 6 months of Screening&#xD;
&#xD;
          2. Decompensated liver disease, coagulopathy, history or presence of clinically&#xD;
             significant ascites, variceal hemorrhage, and/or encephalopathy&#xD;
&#xD;
          3. International normalized ratio (INR) &gt;1.4 (the patient may be treated with Vitamin K&#xD;
             intravenously, and if INR is ≤1.4 at resampling the patient may be started on program&#xD;
             medication)&#xD;
&#xD;
          4. Serum ALT &gt;10 × ULN at Screening&#xD;
&#xD;
          5. Serum ALT &gt;15 × ULN at any time point during the last 6 months unless an alternate&#xD;
             etiology was confirmed for the elevation&#xD;
&#xD;
          6. Total bilirubin &gt;10 × ULN at Screening&#xD;
&#xD;
          7. Any patient who is pregnant, lactating, or planning to get pregnant&#xD;
&#xD;
          8. Patients who qualify for enrollment in other Phase 2 or Phase 3 trials intended to&#xD;
             support marketing approval in PFIC&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>N/A</maximum_age>
  </eligibility>
  <location>
    <facility>
      <name>Albireo Pharma Inc.</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02109</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>July 2021</verification_date>
  <study_first_submitted>July 13, 2020</study_first_submitted>
  <study_first_submitted_qc>July 20, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 23, 2020</study_first_posted>
  <last_update_submitted>July 28, 2021</last_update_submitted>
  <last_update_submitted_qc>July 28, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">July 29, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>PFIC</keyword>
  <keyword>pediatric</keyword>
  <keyword>Cholestasis</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Cholestasis</mesh_term>
    <mesh_term>Cholestasis, Intrahepatic</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/NCT04483531
org study id: A4250-014
nct id: NCT04483531
lead sponsor: 
To provide treatment access to patients with PFIC in the US who have pruritus and elevated
      serum bile acids and who are not able to enroll in A4250-008 (PEDFIC2) for the following
      reasons: 1) Do not meet eligibility criteria for PEDFIC 2; 2) Are not able to get to a PEDFIC
      2 site for geographical reasons, and 3) Do meet the eligibility criteria for PEDFIC 2 after
      recruitment has been completed
Eligible patients will be enrolled into this expanded-access program and treated with an oral
      dose of 120 μg/kg/day of odevixibat and evaluated on an ongoing basis.
expanded access type treatment: Yes
intervention type: Drug
intervention name: Odevixibat
description: A4250 is a small molecule and selective inhibitor of IBAT
other name: A4250
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
facility: 
country: United States
responsible party type: Sponsor
mesh term: Cholestasis
mesh term: Cholestasis, Intrahepatic