xml: <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/NCT05982938</url>
    </required_header>
    <id_info>
      <org_study_id>D7332R00001</org_study_id>
      <nct_id>NCT05982938</nct_id>
    </id_info>
    <brief_title>Danicopan Early Access Program</brief_title>
    <official_title>Early Access Program (EAP) for Danicopan as Add-on Treatment to SOLIRIS® or ULTOMIRIS® in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Clinically Significant Extravascular Hemolysis (csEVH)</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Alexion</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Alexion</source>
    <brief_summary>
      <textblock>
        This is an EAP, designed to provide early access to danicopan for participants with PNH&#xD;
        experiencing clinically significant EVH who have failed, not tolerated, or are unable to&#xD;
        receive other approved treatments; in the Treating Physician's opinion, the participant is&#xD;
        not eligible for or is not able to participate in an ongoing clinical trial of danicopan or a&#xD;
        comparable treatment; and according to the Treating Physician, the benefit of danicopan&#xD;
        treatment outweighs the potential risks.&#xD;
      </textblock>
    </brief_summary>
    <overall_status>Available</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>Paroxysmal Nocturnal Hemoglobinuria</condition>
    <condition>PNH</condition>
    <condition>Extravascular Hemolysis</condition>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Danicopan</intervention_name>
      <description>Participants will receive 150 mg danicopan three times per day (TID) orally as an add-on to a background C5i therapy (SOLIRIS® or ULTOMIRIS®). Participants may be dose-escalated to 200 mg TID based on safety and clinical effect.</description>
      <other_name>ALXN2040</other_name>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Inclusion Criteria:&#xD;
  &#xD;
            -  Male or female, aged 18 years and older at the time of providing informed consent.&#xD;
  &#xD;
            -  Diagnosed with PNH with clinically significant EVH as defined by anemia (Hgb ≤ 9.5&#xD;
               g/dL) with absolute reticulocyte count &gt; ULN).≥ 120 × 109/L.&#xD;
  &#xD;
            -  Currently receiving treatment with SOLIRIS® or ULTOMIRIS®.® for at least 6 months&#xD;
               prior to initiation of danicopan treatment.&#xD;
  &#xD;
            -  Platelet count ≥ 30,000/µL without the need for platelet transfusions.&#xD;
  &#xD;
            -  Absolute neutrophil counts ≥ 500/µL.&#xD;
  &#xD;
            -  Have failed, not tolerated, or are unable to receive other approved treatments.&#xD;
  &#xD;
            -  In the Treating Physician's opinion, the potential benefits of treatment with&#xD;
               danicopan outweigh the potential risks for the participant.&#xD;
  &#xD;
            -  Vaccinated against N meningitidis. Participants who initiate danicopan less than 2&#xD;
               weeks after receiving a meningococcal vaccine must receive treatment with appropriate&#xD;
               prophylactic antibiotics from the first day of danicopan treatment until 2 weeks after&#xD;
               vaccination.&#xD;
  &#xD;
            -  Female participants of childbearing potential agree to use effective or highly&#xD;
               effective contraception methods during treatment with danicopan and until 3 days after&#xD;
               discontinuation.&#xD;
  &#xD;
          Exclusion Criteria:&#xD;
  &#xD;
            -  In the opinion of the Treating Physician, the participant is eligible for and able to&#xD;
               participate in an ongoing clinical trial of danicopan or a comparable treatment.&#xD;
  &#xD;
            -  Taking of disallowed medications.&#xD;
  &#xD;
            -  Participant is unwilling or unable to comply with the Treating Physician's treatment&#xD;
               plan related to this EAP for any reason.&#xD;
  &#xD;
            -  Known hypersensitivity to danicopan or any of its excipients, or any of the&#xD;
               ingredients contained in danicopan.&#xD;
  &#xD;
            -  Participants who are nursing or pregnant (or women who are planning to become pregnant&#xD;
               during treatment with danicopan).&#xD;
  &#xD;
            -  Laboratory abnormalities at screening, as follows:&#xD;
  &#xD;
            -  Alanine aminotransferase (ALT) &gt; 2 × upper limit of normal (ULN)&#xD;
  &#xD;
            -  Direct bilirubin &gt; 2 × ULN, with the exception of:&#xD;
  &#xD;
            -  participants with increased bilirubin due to EVH in the opinion of the Treating&#xD;
               Physician or&#xD;
  &#xD;
            -  participants with documented Gilbert's syndrome&#xD;
  &#xD;
            -  Active bacterial or viral infection, a body temperature &gt; 38°C on 2 consecutive daily&#xD;
               measures, evidence of other infection, or history of any febrile illness within 14&#xD;
               days prior to initiating danicopan treatment.&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>18 Years</minimum_age>
      <maximum_age>N/A</maximum_age>
      <healthy_volunteers>No</healthy_volunteers>
    </eligibility>
    <overall_contact>
      <last_name>AstraZeneca Clinical Study Information Center</last_name>
      <phone>1-877-240-9479</phone>
      <email>information.center@astrazeneca.com</email>
    </overall_contact>
    <verification_date>July 2023</verification_date>
    <study_first_submitted>August 1, 2023</study_first_submitted>
    <study_first_submitted_qc>August 1, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 9, 2023</study_first_posted>
    <last_update_submitted>August 1, 2023</last_update_submitted>
    <last_update_submitted_qc>August 1, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 9, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Sponsor</responsible_party_type>
    </responsible_party>
    <keyword>paroxysmal nocturnal hemoglobinuria</keyword>
    <keyword>PNH</keyword>
    <keyword>component 5 inhibitors</keyword>
    <keyword>C5i</keyword>
    <keyword>intravascular hemolysis</keyword>
    <keyword>IVH</keyword>
    <keyword>anemia</keyword>
    <keyword>EVH</keyword>
    <keyword>extravascular hemolysis</keyword>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Hemoglobinuria</mesh_term>
      <mesh_term>Hemoglobinuria, Paroxysmal</mesh_term>
      <mesh_term>Hemolysis</mesh_term>
    </condition_browse>
    <!-- Results have not yet been posted for this study                                          -->
  </clinical_study>
  
  
text: 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/NCT05982938
  org study id: D7332R00001
  nct id: NCT05982938
  lead sponsor: 
  This is an EAP, designed to provide early access to danicopan for participants with PNH
        experiencing clinically significant EVH who have failed, not tolerated, or are unable to
        receive other approved treatments; in the Treating Physician's opinion, the participant is
        not eligible for or is not able to participate in an ongoing clinical trial of danicopan or a
        comparable treatment; and according to the Treating Physician, the benefit of danicopan
        treatment outweighs the potential risks.
  expanded access type treatment: Yes
  intervention type: Drug
  intervention name: Danicopan
  description: Participants will receive 150 mg danicopan three times per day (TID) orally as an add-on to a background C5i therapy (SOLIRIS® or ULTOMIRIS®). Participants may be dose-escalated to 200 mg TID based on safety and clinical effect.
  other name: ALXN2040
  criteria: 
  gender: All
  minimum age: 18 Years
  maximum age: N/A
  healthy volunteers: No
  last name: AstraZeneca Clinical Study Information Center
  phone: 1-877-240-9479
  email: information.center@astrazeneca.com
  responsible party type: Sponsor
  mesh term: Hemoglobinuria
  mesh term: Hemoglobinuria, Paroxysmal
  mesh term: Hemolysis