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/NCT06003881</url>
    </required_header>
    <id_info>
      <org_study_id>R3918-PLE-21110</org_study_id>
      <nct_id>NCT06003881</nct_id>
    </id_info>
    <brief_title>Compassionate Use of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy</brief_title>
    <acronym>CHAPLE Disease</acronym>
    <official_title>An Open-Label, Expanded Access Program of Pozelimab in Patients With CD55-Deficient Protein-Losing Enteropathy (CHAPLE Disease)</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Regeneron Pharmaceuticals</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Regeneron Pharmaceuticals</source>
    <brief_summary>
      <textblock>
        The program is to provide access to an experimental drug called pozelimab and to document the&#xD;
        long-term safety of pozelimab in patients with Protein-Losing Enteropathy (PLE).&#xD;
        CD55-deficient PLE/CHAPLE disease is a rare inherited disease of the immune system. This can&#xD;
        be a life-threatening condition that is usually found when patients are children.&#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>CD55-Deficient Protein-Losing Enteropathy</condition>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Pozelimab</intervention_name>
      <description>Subcutaneous (SC) administration</description>
      <other_name>REGN3918</other_name>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Key Inclusion Criteria:&#xD;
  &#xD;
            1. Clinical diagnosis of CD55-deficient PLE/CHAPLE disease (based on a history of PLE),&#xD;
               confirmed by biallelic CD55 loss-of-function mutation detected by genotype analysis&#xD;
               (frameshift, nonsense mutations) as defined in the protocol&#xD;
  &#xD;
            2. Written informed consent from parent/guardian for minor patients&#xD;
  &#xD;
            3. Written assent from minor patients as appropriate (eg, above the age of 6 years or the&#xD;
               applicable age per local regulatory requirements)&#xD;
  &#xD;
          Key Exclusion Criteria:&#xD;
  &#xD;
            1. Patients who discontinued the prior pozelimab study due to safety or lack of efficacy&#xD;
  &#xD;
            2. Considered by the treating physician as inappropriate for this program for any reason&#xD;
               as defined in the protocol&#xD;
  &#xD;
          NOTE: Other protocol defined inclusion / exclusion criteria apply&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>1 Year</minimum_age>
      <maximum_age>N/A</maximum_age>
    </eligibility>
    <overall_contact>
      <last_name>Requests for compassionate use must be initiated by a treating physician Physicians with questions about access can contact Clinigen</last_name>
      <phone>+44(0) 1932 824 100</phone>
      <email>medicineaccess@clinigengroup.com</email>
    </overall_contact>
    <verification_date>August 2023</verification_date>
    <study_first_submitted>August 14, 2023</study_first_submitted>
    <study_first_submitted_qc>August 14, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">August 22, 2023</study_first_posted>
    <last_update_submitted>August 14, 2023</last_update_submitted>
    <last_update_submitted_qc>August 14, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">August 22, 2023</last_update_posted>
    <responsible_party>
      <responsible_party_type>Sponsor</responsible_party_type>
    </responsible_party>
    <condition_browse>
      <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
      <mesh_term>Intestinal Diseases</mesh_term>
      <mesh_term>Protein-Losing Enteropathies</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/NCT06003881
  org study id: R3918-PLE-21110
  nct id: NCT06003881
  lead sponsor: 
  The program is to provide access to an experimental drug called pozelimab and to document the
        long-term safety of pozelimab in patients with Protein-Losing Enteropathy (PLE).
        CD55-deficient PLE/CHAPLE disease is a rare inherited disease of the immune system. This can
        be a life-threatening condition that is usually found when patients are children.
  expanded access type treatment: Yes
  intervention type: Drug
  intervention name: Pozelimab
  description: Subcutaneous (SC) administration
  other name: REGN3918
  criteria: 
  gender: All
  minimum age: 1 Year
  maximum age: N/A
  last name: Requests for compassionate use must be initiated by a treating physician Physicians with questions about access can contact Clinigen
  phone: +44(0) 1932 824 100
  email: medicineaccess@clinigengroup.com
  responsible party type: Sponsor
  mesh term: Intestinal Diseases
  mesh term: Protein-Losing Enteropathies