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/NCT06028594</url>
    </required_header>
    <id_info>
      <org_study_id>R3918-PNH-2238</org_study_id>
      <nct_id>NCT06028594</nct_id>
    </id_info>
    <brief_title>Compassionate Use of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria</brief_title>
    <official_title>An Expanded Access Program of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria</official_title>
    <sponsors>
      <lead_sponsor>
        <agency>Regeneron Pharmaceuticals</agency>
        <agency_class>Industry</agency_class>
      </lead_sponsor>
    </sponsors>
    <source>Regeneron Pharmaceuticals</source>
    <brief_summary>
      <textblock>
        This program is designed to provide access to pozelimab and cemdisiran and document the&#xD;
        long-term safety of pozelimab and cemdisiran combination therapy in patients with paroxysmal&#xD;
        nocturnal hemoglobinuria (PNH). PNH is a rare immune disease that causes red blood cells in&#xD;
        your body to break apart.&#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>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Pozelimab</intervention_name>
      <description>Subcutaneous (SC) administration</description>
      <other_name>REGN3918</other_name>
    </intervention>
    <intervention>
      <intervention_type>Drug</intervention_type>
      <intervention_name>Cemdisiran</intervention_name>
      <description>Subcutaneous (SC) administration</description>
      <other_name>ALN-CC5</other_name>
    </intervention>
    <eligibility>
      <criteria>
        <textblock>
          Key Inclusion Criteria:&#xD;
  &#xD;
            1. Patients who have completed either the end of treatment visit of the open-label&#xD;
               treatment period or open-label extension period in one of the following parent&#xD;
               studies:&#xD;
  &#xD;
                 -  An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and&#xD;
                    Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients with&#xD;
                    Paroxysmal Nocturnal Hemoglobinuria (R3918-PNH-2050 [NCT05744921])&#xD;
  &#xD;
                 -  A Randomized, Open-Label, Two-Arm Study to Evaluate the Safety, Efficacy, and&#xD;
                    Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Treatment in&#xD;
                    Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab&#xD;
                    Monotherapy (R3918-PNH-2092 [NCT04811716])&#xD;
  &#xD;
                 -  A Single Arm, Open-Label Study to Assess the Safety, Efficacy, and&#xD;
                    Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Therapy in&#xD;
                    Patients with Paroxysmal Nocturnal Hemoglobinuria Who Switch from Eculizumab&#xD;
                    Therapy (R3918-PNH-20105 [NCT04888507]). Note: In some countries, patients that&#xD;
                    would normally enter into the R3918-PNH-2050 (NCT05744921) parent study before&#xD;
                    entering this expanded access program, may be given the opportunity to skip&#xD;
                    R3918-PNH-2050 (NCT05744921) and go straight into this expanded access program,&#xD;
                    with Regeneron's permission&#xD;
  &#xD;
            2. Patients who have been enrolled in R3918-PNH-2022 (NCT05131204), with Regeneron's&#xD;
               permission&#xD;
  &#xD;
            3. Willing and able to comply with clinic visits and related standard-of-care procedures&#xD;
  &#xD;
            4. With Regeneron's permission, patients who have been enrolled in other PNH studies as&#xD;
               appropriate&#xD;
  &#xD;
          Key Exclusion Criteria:&#xD;
  &#xD;
            1. Significant history or concerns of non-compliance that could impact the patient's&#xD;
               safety per the treating physician&#xD;
  &#xD;
            2. Any new condition or worsening of an existing condition which, in the opinion of the&#xD;
               treating physician, would make the patient unsuitable for enrollment or could&#xD;
               interfere with the patient participating in or completing the program&#xD;
  &#xD;
          NOTE: Other protocol defined inclusion / exclusion criteria apply&#xD;
        </textblock>
      </criteria>
      <gender>All</gender>
      <minimum_age>18 Years</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 31, 2023</study_first_submitted>
    <study_first_submitted_qc>August 31, 2023</study_first_submitted_qc>
    <study_first_posted type="Actual">September 8, 2023</study_first_posted>
    <last_update_submitted>August 31, 2023</last_update_submitted>
    <last_update_submitted_qc>August 31, 2023</last_update_submitted_qc>
    <last_update_posted type="Actual">September 8, 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>Hemoglobinuria</mesh_term>
      <mesh_term>Hemoglobinuria, Paroxysmal</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/NCT06028594
  org study id: R3918-PNH-2238
  nct id: NCT06028594
  lead sponsor: 
  This program is designed to provide access to pozelimab and cemdisiran and document the
        long-term safety of pozelimab and cemdisiran combination therapy in patients with paroxysmal
        nocturnal hemoglobinuria (PNH). PNH is a rare immune disease that causes red blood cells in
        your body to break apart.
  expanded access type treatment: Yes
  intervention type: Drug
  intervention name: Pozelimab
  description: Subcutaneous (SC) administration
  other name: REGN3918
  intervention type: Drug
  intervention name: Cemdisiran
  description: Subcutaneous (SC) administration
  other name: ALN-CC5
  criteria: 
  gender: All
  minimum age: 18 Years
  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: Hemoglobinuria
  mesh term: Hemoglobinuria, Paroxysmal