<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/NCT03278314</url>
  </required_header>
  <id_info>
    <org_study_id>VX17-661-901</org_study_id>
    <nct_id>NCT03278314</nct_id>
  </id_info>
  <brief_title>Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis</brief_title>
  <official_title>Tezacaftor/Ivacaftor Combination Therapy Expanded Access Program for Patients 12 Years of Age and Older With Cystic Fibrosis</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Vertex Pharmaceuticals Incorporated</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Vertex Pharmaceuticals Incorporated</source>
  <brief_summary>
    <textblock>
      To provide TEZ/IVA combination therapy to CF patients who are 12 years of age and older who&#xD;
      completed Vertex TEZ/IVA combination therapy clinical studies (NCT02565914 or NCT03150719).&#xD;
      To provide TEZ/IVA combination therapy to CF patients in critical need who are 12 years of&#xD;
      age and older, homozygous for F508del.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Approved for marketing</overall_status>
  <study_type>Expanded Access</study_type>
  <expanded_access_info>
    <expanded_access_type_treatment>Yes</expanded_access_type_treatment>
  </expanded_access_info>
  <condition>Cystic Fibrosis</condition>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>tezacaftor/ivacaftor</intervention_name>
    <description>orally administered TEZ/IVA combination therapy</description>
    <other_name>TEZ/IVA</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Males and females, 12 years of age and older with confirmed diagnosis of CF.&#xD;
&#xD;
               1. - completed Vertex clinical studies NCT02565914 or NCT03150719, or&#xD;
&#xD;
               2. - are homozygous for F508del and have (1) discontinued Orkambi within 12 weeks&#xD;
                  from start of treatment because of the onset of respiratory AEs considered to be&#xD;
                  treatment related or (2) cannot initiate Orkambi because of chronic treatment&#xD;
                  with an essential sensitive CYP3A substrate or a CYP3A substrate with a narrow&#xD;
                  therapeutic index AND who meet at least 1 of the following criteria:&#xD;
&#xD;
                    -  the highest percent predicted forced expiratory volume in 1 second (ppFEV1)&#xD;
                       is &lt;40 in the 6 months before the date of completion of the request form, or&#xD;
&#xD;
                    -  documentation of being active on a lung transplant waiting list or&#xD;
                       documentation of being evaluated for lung transplantation, but deemed&#xD;
                       unsuitable because of contraindications, or&#xD;
&#xD;
                    -  rapid and persistent loss of lung function, defined as at least a 20%&#xD;
                       relative decrease in ppFEV1 in the last 6 months and sustained for at least&#xD;
                       1 month despite appropriate treatment.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  History of any comorbidity that, in the opinion of the treating physician, might pose&#xD;
             undue risk in administering TEZ/IVA combination therapy to the patient (e.g., history&#xD;
             of advanced liver disease).&#xD;
&#xD;
          -  Ongoing or prior participation in an investigational drug study (with the exception of&#xD;
             Vertex clinical studies evaluating TEZ/IVA) within 5 terminal half-lives of the&#xD;
             previous investigational study drug or 30 days, whichever is longer, of first&#xD;
             administration of TEZ/IVA.&#xD;
&#xD;
          -  Subjects who are pregnant.&#xD;
&#xD;
          -  Patients eligible for participation in ongoing clinical studies evaluating TEZ/IVA or&#xD;
             other CFTR modulator therapies.&#xD;
&#xD;
        Other protocol defined Inclusion/Exclusion criteria may apply.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>12 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
  </eligibility>
  <verification_date>October 2018</verification_date>
  <study_first_submitted>September 6, 2017</study_first_submitted>
  <study_first_submitted_qc>September 8, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">September 11, 2017</study_first_posted>
  <last_update_submitted>October 31, 2018</last_update_submitted>
  <last_update_submitted_qc>October 31, 2018</last_update_submitted_qc>
  <last_update_posted type="Actual">November 2, 2018</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>Cystic Fibrosis</mesh_term>
    <mesh_term>Fibrosis</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Ivacaftor</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/NCT03278314
org study id: VX17-661-901
nct id: NCT03278314
lead sponsor: 
To provide TEZ/IVA combination therapy to CF patients who are 12 years of age and older who
      completed Vertex TEZ/IVA combination therapy clinical studies (NCT02565914 or NCT03150719).
      To provide TEZ/IVA combination therapy to CF patients in critical need who are 12 years of
      age and older, homozygous for F508del.
expanded access type treatment: Yes
intervention type: Drug
intervention name: tezacaftor/ivacaftor
description: orally administered TEZ/IVA combination therapy
other name: TEZ/IVA
criteria: 
gender: All
minimum age: 12 Years
maximum age: N/A
responsible party type: Sponsor
mesh term: Cystic Fibrosis
mesh term: Fibrosis
mesh term: Ivacaftor