<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/NCT04428632</url>
  </required_header>
  <id_info>
    <org_study_id>BCX7353-308</org_study_id>
    <nct_id>NCT04428632</nct_id>
  </id_info>
  <brief_title>Oral Berotralstat Expanded Access Program</brief_title>
  <official_title>Expanded Access Program With Oral Berotralstat for the Prevention of Attacks in Patients With Hereditary Angioedema</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>BioCryst Pharmaceuticals</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>BioCryst Pharmaceuticals</source>
  <brief_summary>
    <textblock>
      This expanded access program will provide access to berotralstat for eligible participants&#xD;
      with hereditary angioedema in the U.S.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Physicians may request access to berotralstat for eligible participants with unmet medical&#xD;
      need&#xD;
&#xD;
      Physicians can send email inquiries to access.us@inceptua.com or call 1-888-225-8677&#xD;
&#xD;
      Patients who may be interested should contact their physician about participation&#xD;
&#xD;
      Berotralstat (BCX7353) will be available through this expanded access program until it is&#xD;
      commercially available&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Approved for marketing</overall_status>
  <study_type>Expanded Access</study_type>
  <expanded_access_info>
    <expanded_access_type_intermediate>Yes</expanded_access_type_intermediate>
  </expanded_access_info>
  <condition>Hereditary Angioedema</condition>
  <condition>HAE</condition>
  <condition>Prophylaxis</condition>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Berotralstat</intervention_name>
    <description>One 150mg capsule administered orally once daily</description>
    <other_name>BCX7353</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria&#xD;
&#xD;
        Patients must meet all of the following inclusion criteria to be eligible for participation&#xD;
        in this program:&#xD;
&#xD;
          1. At least 12 years of age&#xD;
&#xD;
          2. Able to provide written, informed consent or assent&#xD;
&#xD;
          3. Patients with a clinical diagnosis of HAE Type I or II who, in the opinion of their&#xD;
             treating physician, are expected to benefit from an oral treatment for the prevention&#xD;
             of angioedema attacks, and who are not eligible or able to access a berotralstat&#xD;
             clinical trial&#xD;
&#xD;
          4. Females must use acceptable effective contraception&#xD;
&#xD;
        Exclusion Criteria&#xD;
&#xD;
        Patients must meet none of the below exclusion criteria to be eligible for participation in&#xD;
        this program:&#xD;
&#xD;
          1. Pregnancy or breast-feeding&#xD;
&#xD;
          2. Any clinically significant medical condition or medical history that, in the opinion&#xD;
             of the treating physician, would interfere with the patient's safety&#xD;
&#xD;
          3. Current infection with hepatitis B virus (HBV), hepatitis C virus (HCV), or human&#xD;
             immunodeficiency virus (HIV)&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>12 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
  </eligibility>
  <verification_date>December 2020</verification_date>
  <study_first_submitted>June 10, 2020</study_first_submitted>
  <study_first_submitted_qc>June 10, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 11, 2020</study_first_posted>
  <last_update_submitted>December 9, 2020</last_update_submitted>
  <last_update_submitted_qc>December 9, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">December 11, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Berotralstat</keyword>
  <keyword>BCX7353</keyword>
  <keyword>Hereditary Angioedema</keyword>
  <keyword>HAE</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Angioedema</mesh_term>
    <mesh_term>Angioedemas, Hereditary</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Berotralstat</mesh_term>
  </intervention_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/NCT04428632
org study id: BCX7353-308
nct id: NCT04428632
lead sponsor: 
This expanded access program will provide access to berotralstat for eligible participants
      with hereditary angioedema in the U.S.
Physicians may request access to berotralstat for eligible participants with unmet medical
      need

      Physicians can send email inquiries to access.us@inceptua.com or call 1-888-225-8677

      Patients who may be interested should contact their physician about participation

      Berotralstat (BCX7353) will be available through this expanded access program until it is
      commercially available
expanded access type intermediate: Yes
intervention type: Drug
intervention name: Berotralstat
description: One 150mg capsule administered orally once daily
other name: BCX7353
criteria: 
gender: All
minimum age: 12 Years
maximum age: N/A
responsible party type: Sponsor
mesh term: Angioedema
mesh term: Angioedemas, Hereditary
mesh term: Berotralstat