<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/NCT03277196</url>
  </required_header>
  <id_info>
    <org_study_id>VX15-770-126</org_study_id>
    <secondary_id>2017-001379-21</secondary_id>
    <nct_id>NCT03277196</nct_id>
  </id_info>
  <brief_title>A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation</brief_title>
  <official_title>A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Vertex Pharmaceuticals Incorporated</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Vertex Pharmaceuticals Incorporated</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an&#xD;
      observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in&#xD;
      subjects with cystic fibrosis (CF) who are &lt;24 months of age at treatment initiation and have&#xD;
      an approved Ivacaftor-Responsive mutation&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">August 16, 2017</start_date>
  <completion_date type="Anticipated">July 3, 2024</completion_date>
  <primary_completion_date type="Anticipated">July 3, 2024</primary_completion_date>
  <phase>Phase 3</phase>
  <study_type>Interventional</study_type>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Safety assessments based on the number of subjects with adverse events (AEs) and serious adverse events (SAEs)</measure>
    <time_frame>from baseline through safety follow-up (up to 24 weeks after last dose)</time_frame>
    <description>Number of subjects with AEs and SAEs will be reported.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Absolute change in sweat chloride</measure>
    <time_frame>from baseline through Week 96</time_frame>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">86</enrollment>
  <condition>Cystic Fibrosis</condition>
  <arm_group>
    <arm_group_label>Ivacaftor Arm</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>Observational Arm</arm_group_label>
    <arm_group_type>No Intervention</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Ivacaftor</intervention_name>
    <description>Ivacaftor will be administered every 12 hours (q12h) from Day 1 through the morning dose of the Week 96 Visit.</description>
    <arm_group_label>Ivacaftor Arm</arm_group_label>
    <other_name>VX-770</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        Ivacaftor Arm: Subjects From Study 124 (NCT02725567 ) Part B:&#xD;
&#xD;
          -  Subjects transitioning from Study 124 Part B must have completed the last study visit&#xD;
             of Study 124 Part B.&#xD;
&#xD;
          -  As judged by the investigator, parent or legal guardian must be able to understand&#xD;
             protocol requirements, restrictions, and instructions; and must sign the informed&#xD;
             consent form (ICF).&#xD;
&#xD;
        Ivacaftor Arm: Subjects Not From Study 124 Part B:&#xD;
&#xD;
          -  Confirmed diagnosis of CF, or 2 CF-causing mutations.&#xD;
&#xD;
          -  An ivacaftor- responsive CFTR mutation on at least 1 allele. Subjects will be eligible&#xD;
             in countries/regions where ivacaftor is approved for use in subjects 2 years of age&#xD;
             and older.&#xD;
&#xD;
          -  As judged by the investigator, parent or legal guardian must be able to understand&#xD;
             protocol requirements, restrictions, and instructions; and must sign the ICF.&#xD;
&#xD;
        Observational Arm:&#xD;
&#xD;
          -  Received ivacaftor treatment in Study 124 Part B and elected not to enroll or&#xD;
             ineligible to enroll in the ivacaftor arm of Study 126.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        Ivacaftor Arm: Subjects From Study 124 Part B:&#xD;
&#xD;
          -  History of any illness or condition that, in the opinion of the investigator, might&#xD;
             confound the results of the study or pose an additional risk in administering&#xD;
             ivacaftor to the subject.&#xD;
&#xD;
          -  Subjects receiving commercially available ivacaftor treatment&#xD;
&#xD;
        Ivacaftor Arm: Subjects Not From Study 124 Part B:&#xD;
&#xD;
          -  History of any illness or condition that, in the opinion of the investigator, might&#xD;
             confound the results of the study or pose an additional risk in administering&#xD;
             ivacaftor to the subject&#xD;
&#xD;
          -  An acute upper or lower respiratory infection, or pulmonary exacerbation, or changes&#xD;
             in therapy for pulmonary disease within 4 weeks of Day 1&#xD;
&#xD;
          -  Abnormal liver function at screening&#xD;
&#xD;
          -  Hemoglobin &lt;9.5 g/dL at screening&#xD;
&#xD;
          -  History of solid organ or hematological transplantation&#xD;
&#xD;
          -  Use of any moderate or strong inducers or inhibitors of CYP3A within 2 weeks of Day 1&#xD;
&#xD;
        Observational Arm:&#xD;
&#xD;
          -  Receiving ivacaftor treatment&#xD;
&#xD;
        Other protocol defined Inclusion/Exclusion criteria may apply.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>0 Months</minimum_age>
    <maximum_age>24 Months</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>University of Alabama at Birmingham</name>
      <address>
        <city>Birmingham</city>
        <state>Alabama</state>
        <zip>35233</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Stanford University</name>
      <address>
        <city>Palo Alto</city>
        <state>California</state>
        <zip>94034</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Alfred I DuPont Hospital for Children</name>
      <address>
        <city>Wilmington</city>
        <state>Delaware</state>
        <zip>19803</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nemours Children's Hospital</name>
      <address>
        <city>Orlando</city>
        <state>Florida</state>
        <zip>32827</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Center for Advanced Pediatrics</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30329</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Northwestern University</name>
      <address>
        <city>Chicago</city>
        <state>Illinois</state>
        <zip>60611</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Riley Hospital for Children at Indiana University Health</name>
      <address>
        <city>Indianapolis</city>
        <state>Indiana</state>
        <zip>46202</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>John Hopkins Hospital</name>
      <address>
        <city>Baltimore</city>
        <state>Maryland</state>
        <zip>21287</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Boston Children's Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Childrens's Hospitals and Clinics of Minnnesota</name>
      <address>
        <city>Minneapolis</city>
        <state>Minnesota</state>
        <zip>55404</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Mercy Hospital</name>
      <address>
        <city>Kansas City</city>
        <state>Missouri</state>
        <zip>64108</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Billings Clinic Hospital</name>
      <address>
        <city>Billings</city>
        <state>Montana</state>
        <zip>59101</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nationwide Children's Hospital</name>
      <address>
        <city>Columbus</city>
        <state>Ohio</state>
        <zip>43205</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Philadelphia</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Texas Children's Hospital</name>
      <address>
        <city>Houston</city>
        <state>Texas</state>
        <zip>77030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Seattle Children's Hospital</name>
      <address>
        <city>Seattle</city>
        <state>Washington</state>
        <zip>98105</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Wisconsin</name>
      <address>
        <city>Madison</city>
        <state>Wisconsin</state>
        <zip>53792</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Queensland Children's Hospital</name>
      <address>
        <city>South Brisbane</city>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Hospital for Sick Children</name>
      <address>
        <city>Toronto</city>
        <state>Ontario</state>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin</name>
      <address>
        <city>Heidelberg</city>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Health Ireland at Crumlin</name>
      <address>
        <city>Dublin</city>
        <country>Ireland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's University Hospital Temple Street</name>
      <address>
        <city>Dublin</city>
        <country>Ireland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Hospital Limerick</name>
      <address>
        <city>Limerick</city>
        <country>Ireland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Paediatric Clinical Research Facility</name>
      <address>
        <city>Edinburgh</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Alder Hey Children's Alder Hey Children's NHS Foundation Trust</name>
      <address>
        <city>Liverpool</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Great Ormond Street Hospital for Sick Children</name>
      <address>
        <city>London</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Royal Brompton &amp; Harefield NHS Founcation Trust, Royal BromptonHospital</name>
      <address>
        <city>London</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Royal Manchester Children's Hospital</name>
      <address>
        <city>Manchester</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Oxford University Hospitals NHS Trust, John Radcliffe Hospital</name>
      <address>
        <city>Oxford</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Australia</country>
    <country>Canada</country>
    <country>Germany</country>
    <country>Ireland</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <verification_date>September 2023</verification_date>
  <study_first_submitted>September 6, 2017</study_first_submitted>
  <study_first_submitted_qc>September 6, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">September 8, 2017</study_first_posted>
  <last_update_submitted>September 5, 2023</last_update_submitted>
  <last_update_submitted_qc>September 5, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">September 6, 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>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/NCT03277196
org study id: VX15-770-126
secondary id: 2017-001379-21
nct id: NCT03277196
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an
      observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in
      subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have
      an approved Ivacaftor-Responsive mutation
allocation: Non-Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Safety assessments based on the number of subjects with adverse events (AEs) and serious adverse events (SAEs)
time frame: from baseline through safety follow-up (up to 24 weeks after last dose)
description: Number of subjects with AEs and SAEs will be reported.
measure: Absolute change in sweat chloride
time frame: from baseline through Week 96
arm group label: Ivacaftor Arm
arm group type: Experimental
arm group label: Observational Arm
arm group type: No Intervention
intervention type: Drug
intervention name: Ivacaftor
description: Ivacaftor will be administered every 12 hours (q12h) from Day 1 through the morning dose of the Week 96 Visit.
arm group label: Ivacaftor Arm
other name: VX-770
criteria: 
gender: All
minimum age: 0 Months
maximum age: 24 Months
healthy volunteers: No
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country: Australia
country: Canada
country: Germany
country: Ireland
country: United Kingdom
country: United States
responsible party type: Sponsor
mesh term: Cystic Fibrosis
mesh term: Fibrosis
mesh term: Ivacaftor