<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/NCT02971839</url>
  </required_header>
  <id_info>
    <org_study_id>CP656.2001</org_study_id>
    <nct_id>NCT02971839</nct_id>
  </id_info>
  <brief_title>Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations</brief_title>
  <official_title>A Phase 2, Randomized, Parallel-Group, Double-Blind, Placebo Controlled Study to Evaluate the Safety and Efficacy of CTP-656 With an Open-Label Active Comparator in Patients With Cystic Fibrosis With CFTR Gating Mutations.</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>
  </oversight_info>
  <brief_summary>
    <textblock>
      This study will evaluate the efficacy and safety of CTP-656 in patients with cystic fibrosis&#xD;
      (CF) who have a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a randomized, parallel-group, double-blind, placebo controlled multicenter study to&#xD;
      evaluate the safety and efficacy of CTP-656 in CF patients with CFTR gating mutations,&#xD;
      compared to Kalydeco, for a total of 28 days. Subjects will be randomized to receive either&#xD;
      double-blind CTP-656 or placebo, or open-label Kalydeco.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    Decision by Sponsor.&#xD;
  </why_stopped>
  <start_date type="Actual">December 2016</start_date>
  <completion_date type="Actual">August 2017</completion_date>
  <primary_completion_date type="Actual">August 2017</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Change From Baseline in Sweat Chloride at Day 28</measure>
    <time_frame>From baseline at Day 28</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28</measure>
    <time_frame>From baseline at Day 28</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Cystic Fibrosis Questionnaire-Respiratory Domain (CFQ-R) at Day 28</measure>
    <time_frame>From baseline at Day 28</time_frame>
  </secondary_outcome>
  <number_of_arms>5</number_of_arms>
  <enrollment type="Actual">11</enrollment>
  <condition>Cystic Fibrosis</condition>
  <arm_group>
    <arm_group_label>VX-561 20 mg</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>VX-561 100 mg</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>VX-561 150 mg</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>Ivacaftor</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>VX-561</intervention_name>
    <arm_group_label>VX-561 100 mg</arm_group_label>
    <arm_group_label>VX-561 150 mg</arm_group_label>
    <arm_group_label>VX-561 20 mg</arm_group_label>
    <other_name>CTP-656</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>IVA</intervention_name>
    <arm_group_label>Ivacaftor</arm_group_label>
    <other_name>ivacaftor; VX-770</other_name>
    <other_name>Kalydeco</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  18 years of age or older&#xD;
&#xD;
          -  Has a confirmed diagnosis of CF with at least one allele of the following CFTR gating&#xD;
             mutations: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, and S549R.&#xD;
&#xD;
          -  Has been stable on Kalydeco therapy for at least 3 months prior to screening&#xD;
&#xD;
          -  Has FEV1 ≥ 60% of predicted normal for age, sex, and height at screening and baseline&#xD;
             (Day 1) assessments&#xD;
&#xD;
          -  Weighs at least 40 kg at screening&#xD;
&#xD;
          -  Patients of either gender and women of childbearing potential must be willing to use a&#xD;
             medically highly effective form of birth control during the treatment period and 30&#xD;
             days after the last dose of study treatment.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Acute upper respiratory infection or lower respiratory infection, pulmonary&#xD;
             exacerbation, or changes in therapy within 4 weeks of study treatment&#xD;
&#xD;
          -  Uncontrolled type 2 diabetes, or uncontrolled CF-related diabetes&#xD;
&#xD;
          -  History of hepatitis C or chronic active hepatitis B infection&#xD;
&#xD;
          -  History of pulmonary tuberculosis, non-tuberculosis mycobacterial infections or&#xD;
             allergic bronchopulmonary aspergillosis (ABPA) treated during screening or within 2&#xD;
             years prior to screening&#xD;
&#xD;
          -  Colonization with B. cenocepacia, B. dolosa, B. multivorans, and/or M. abcessus within&#xD;
             2 years prior to Screening&#xD;
&#xD;
          -  Abnormal liver function&#xD;
&#xD;
          -  History of abnormal renal function&#xD;
&#xD;
          -  History of prolonged QTcF &gt; 450 msec for males or QTcF &gt; 470 msec for females&#xD;
&#xD;
          -  History of solid organ or hematological transplantation&#xD;
&#xD;
          -  Using any inhibitor or inducer of cytochrome P450/3A during the study or within 30&#xD;
             days of screening&#xD;
&#xD;
          -  Women who are pregnant or lactating, or have plans to become pregnant during the study&#xD;
             or within 1 month following the last dose&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>University of Southern California</name>
      <address>
        <city>Los Angeles</city>
        <state>California</state>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Stanford Hospital</name>
      <address>
        <city>Palo Alto</city>
        <state>California</state>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's National Health</name>
      <address>
        <city>Washington</city>
        <state>District of Columbia</state>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Miami</name>
      <address>
        <city>Miami</city>
        <state>Florida</state>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Rush University</name>
      <address>
        <city>Chicago</city>
        <state>Illinois</state>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Indiana University</name>
      <address>
        <city>Indianapolis</city>
        <state>Indiana</state>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Boston Children's Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Massachusetts</name>
      <address>
        <city>Worcester</city>
        <state>Massachusetts</state>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Washington University</name>
      <address>
        <city>Saint Louis</city>
        <state>Missouri</state>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Atlantic Health</name>
      <address>
        <city>Morristown</city>
        <state>New Jersey</state>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>New York Medical College</name>
      <address>
        <city>Valhalla</city>
        <state>New York</state>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Cincinnati Children's Hospital</name>
      <address>
        <city>Cincinnati</city>
        <state>Ohio</state>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nationwide Children's Hospital</name>
      <address>
        <city>Columbus</city>
        <state>Ohio</state>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Hospital of Philadelphia</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>August 2020</verification_date>
  <study_first_submitted>November 21, 2016</study_first_submitted>
  <study_first_submitted_qc>November 21, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">November 23, 2016</study_first_posted>
  <results_first_submitted>August 11, 2020</results_first_submitted>
  <results_first_submitted_qc>August 11, 2020</results_first_submitted_qc>
  <results_first_posted type="Actual">August 26, 2020</results_first_posted>
  <disposition_first_submitted>August 13, 2018</disposition_first_submitted>
  <disposition_first_submitted_qc>August 13, 2018</disposition_first_submitted_qc>
  <disposition_first_posted type="Actual">August 15, 2018</disposition_first_posted>
  <last_update_submitted>August 11, 2020</last_update_submitted>
  <last_update_submitted_qc>August 11, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">August 26, 2020</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>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>October 24, 2016</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/39/NCT02971839/Prot_000.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Statistical Analysis Plan</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>September 22, 2017</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/39/NCT02971839/SAP_001.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>VX-561 20 mg</title>
          <description>Participants received VX-561 20 mg orally once daily for 28 days.</description>
        </group>
        <group group_id="P2">
          <title>VX-561 100 mg</title>
          <description>Participants received VX-561 100 mg orally once daily for 28 days.</description>
        </group>
        <group group_id="P3">
          <title>VX-561 150 mg</title>
          <description>Participants received VX-561 150 mg orally once daily for 28 days.</description>
        </group>
        <group group_id="P4">
          <title>Placebo</title>
          <description>Participants received placebo matched to VX-561 orally once daily for 28 days.</description>
        </group>
        <group group_id="P5">
          <title>Ivacaftor</title>
          <description>Participants received Ivacaftor 150 mg orally every 12 hours for 28 days.</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
                <participants group_id="P2" count="2"/>
                <participants group_id="P3" count="2"/>
                <participants group_id="P4" count="2"/>
                <participants group_id="P5" count="3"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
                <participants group_id="P2" count="2"/>
                <participants group_id="P3" count="2"/>
                <participants group_id="P4" count="2"/>
                <participants group_id="P5" count="3"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="0"/>
                <participants group_id="P3" count="0"/>
                <participants group_id="P4" count="0"/>
                <participants group_id="P5" count="0"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>VX-561 20 mg</title>
          <description>Participants received VX-561 20 mg orally once daily for 28 days.</description>
        </group>
        <group group_id="B2">
          <title>VX-561 100 mg</title>
          <description>Participants received VX-561 100 mg orally once daily for 28 days.</description>
        </group>
        <group group_id="B3">
          <title>VX-561 150 mg</title>
          <description>Participants received VX-561 150 mg orally once daily for 28 days.</description>
        </group>
        <group group_id="B4">
          <title>Placebo</title>
          <description>Participants received placebo matched to VX-561 orally once daily for 28 days.</description>
        </group>
        <group group_id="B5">
          <title>Ivacaftor</title>
          <description>Participants received Ivacaftor 150 mg orally every 12 hours for 28 days.</description>
        </group>
        <group group_id="B6">
          <title>Total</title>
          <description>Total of all reporting groups</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="2"/>
            <count group_id="B2" value="2"/>
            <count group_id="B3" value="2"/>
            <count group_id="B4" value="2"/>
            <count group_id="B5" value="3"/>
            <count group_id="B6" value="11"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>years</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="24.0" spread="1.41"/>
                    <measurement group_id="B2" value="20.0" spread="1.41"/>
                    <measurement group_id="B3" value="19.5" spread="2.12"/>
                    <measurement group_id="B4" value="29.0" spread="2.83"/>
                    <measurement group_id="B5" value="19.7" spread="0.58"/>
                    <measurement group_id="B6" value="22.2" spread="4.00"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="2"/>
                    <measurement group_id="B4" value="2"/>
                    <measurement group_id="B5" value="2"/>
                    <measurement group_id="B6" value="9"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="1"/>
                    <measurement group_id="B6" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Ethnicity (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="1"/>
                    <measurement group_id="B5" value="0"/>
                    <measurement group_id="B6" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="2"/>
                    <measurement group_id="B4" value="1"/>
                    <measurement group_id="B5" value="3"/>
                    <measurement group_id="B6" value="10"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                    <measurement group_id="B6" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>American Indian or Alaska Native</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                    <measurement group_id="B6" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                    <measurement group_id="B6" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Native Hawaiian or Other Pacific Islander</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                    <measurement group_id="B6" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                    <measurement group_id="B6" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="2"/>
                    <measurement group_id="B4" value="2"/>
                    <measurement group_id="B5" value="3"/>
                    <measurement group_id="B6" value="11"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                    <measurement group_id="B6" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="0"/>
                    <measurement group_id="B5" value="0"/>
                    <measurement group_id="B6" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sweat Chloride</title>
          <units>millimole per liter (mmol/L)</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="59.25" spread="16.617"/>
                    <measurement group_id="B2" value="82.25" spread="27.931"/>
                    <measurement group_id="B3" value="61.50" spread="32.527"/>
                    <measurement group_id="B4" value="36.50" spread="29.698"/>
                    <measurement group_id="B5" value="71.25" spread="37.830"/>
                    <measurement group_id="B6" value="62.15" spread="27.355"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Change From Baseline in Sweat Chloride at Day 28</title>
        <time_frame>From baseline at Day 28</time_frame>
        <population>As pre-specified in SAP section 4.13 (Changes in Conduct or Planned Analyses), due to the limited number of participants being enrolled in the study, model-based analyses and summary statistics plan were not performed. Therefore, no efficacy summary is provided for this outcome measure.</population>
        <group_list>
          <group group_id="O1">
            <title>VX-561 20 mg</title>
            <description>Participants received VX-561 20 mg orally once daily for 28 days.</description>
          </group>
          <group group_id="O2">
            <title>VX-561 100 mg</title>
            <description>Participants received VX-561 100 mg orally once daily for 28 days.</description>
          </group>
          <group group_id="O3">
            <title>VX-561 150 mg</title>
            <description>Participants received VX-561 150 mg orally once daily for 28 days.</description>
          </group>
          <group group_id="O4">
            <title>Placebo</title>
            <description>Participants received placebo matched to VX-561 orally once daily for 28 days.</description>
          </group>
          <group group_id="O5">
            <title>Ivacaftor</title>
            <description>Participants received Ivacaftor 150 mg orally every 12 hours for 28 days.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Sweat Chloride at Day 28</title>
          <population>As pre-specified in SAP section 4.13 (Changes in Conduct or Planned Analyses), due to the limited number of participants being enrolled in the study, model-based analyses and summary statistics plan were not performed. Therefore, no efficacy summary is provided for this outcome measure.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
                <count group_id="O3" value="0"/>
                <count group_id="O4" value="0"/>
                <count group_id="O5" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28</title>
        <time_frame>From baseline at Day 28</time_frame>
        <population>As pre-specified in SAP section 4.13 (Changes in Conduct or Planned Analyses), due to the limited number of participants being enrolled in the study, all model-based analyses and summary statistics are no longer applicable and are therefore removed from analysis plan. Therefore no efficacy summary is available for this outcome measure.</population>
        <group_list>
          <group group_id="O1">
            <title>VX-561 20 mg</title>
            <description>Participants received VX-561 20 mg orally once daily for 28 days.</description>
          </group>
          <group group_id="O2">
            <title>VX-561 100 mg</title>
            <description>Participants received VX-561 100 mg orally once daily for 28 days.</description>
          </group>
          <group group_id="O3">
            <title>VX-561 150 mg</title>
            <description>Participants received VX-561 150 mg orally once daily for 28 days.</description>
          </group>
          <group group_id="O4">
            <title>Placebo</title>
            <description>Participants received placebo matched to VX-561 orally once daily for 28 days.</description>
          </group>
          <group group_id="O5">
            <title>Ivacaftor</title>
            <description>Participants received Ivacaftor 150 mg orally every 12 hours for 28 days.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28</title>
          <population>As pre-specified in SAP section 4.13 (Changes in Conduct or Planned Analyses), due to the limited number of participants being enrolled in the study, all model-based analyses and summary statistics are no longer applicable and are therefore removed from analysis plan. Therefore no efficacy summary is available for this outcome measure.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
                <count group_id="O3" value="0"/>
                <count group_id="O4" value="0"/>
                <count group_id="O5" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Cystic Fibrosis Questionnaire-Respiratory Domain (CFQ-R) at Day 28</title>
        <time_frame>From baseline at Day 28</time_frame>
        <population>As pre-specified in SAP section 4.13 (Changes in Conduct or Planned Analyses), due to the limited number of participants being enrolled in the study, all model-based analyses and summary statistics are no longer applicable and are therefore removed from analysis plan. Therefore no efficacy summary is available for this outcome measure.</population>
        <group_list>
          <group group_id="O1">
            <title>VX-561 20 mg</title>
            <description>Participants received VX-561 20 mg orally once daily for 28 days.</description>
          </group>
          <group group_id="O2">
            <title>VX-561 100 mg</title>
            <description>Participants received VX-561 100 mg orally once daily for 28 days.</description>
          </group>
          <group group_id="O3">
            <title>VX-561 150 mg</title>
            <description>Participants received VX-561 150 mg orally once daily for 28 days.</description>
          </group>
          <group group_id="O4">
            <title>Placebo</title>
            <description>Participants received placebo matched to VX-561 orally once daily for 28 days.</description>
          </group>
          <group group_id="O5">
            <title>Ivacaftor</title>
            <description>Participants received Ivacaftor 150 mg orally every 12 hours for 28 days.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Cystic Fibrosis Questionnaire-Respiratory Domain (CFQ-R) at Day 28</title>
          <population>As pre-specified in SAP section 4.13 (Changes in Conduct or Planned Analyses), due to the limited number of participants being enrolled in the study, all model-based analyses and summary statistics are no longer applicable and are therefore removed from analysis plan. Therefore no efficacy summary is available for this outcome measure.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
                <count group_id="O3" value="0"/>
                <count group_id="O4" value="0"/>
                <count group_id="O5" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Day 1 up to Day 35</time_frame>
      <group_list>
        <group group_id="E1">
          <title>VX-561 20 mg</title>
          <description>Participants received VX-561 20 mg orally once daily for 28 days.</description>
        </group>
        <group group_id="E2">
          <title>VX-561 100 mg</title>
          <description>Participants received VX-561 100 mg orally once daily for 28 days.</description>
        </group>
        <group group_id="E3">
          <title>VX-561 150 mg</title>
          <description>Participants received VX-561 150 mg orally once daily for 28 days.</description>
        </group>
        <group group_id="E4">
          <title>Placebo</title>
          <description>Participants received placebo matched to VX-561 orally once daily for 28 days.</description>
        </group>
        <group group_id="E5">
          <title>Ivacaftor</title>
          <description>Participants received Ivacaftor 150 mg orally every 12 hours for 28 days.</description>
        </group>
      </group_list>
      <serious_events>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>0</frequency_threshold>
        <default_vocab>MedDRA 19.1</default_vocab>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="2" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="1" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Abdominal pain</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Abdominal pain upper</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Constipation</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Diarrhoea</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Nausea</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Vomiting</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Chest discomfort</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Fatigue</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Infective pulmonary exacerbation of cystic fibrosis</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Skin abrasion</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="1" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Blood creatine phosphokinase increased</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Alanine aminotransferase increased</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Aspartate aminotransferase increased</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Blood lactate dehydrogenase increased</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Forced expiratory volume decreased</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Neutrophil count increased</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Pulmonary function test decreased</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Weight increased</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Dizziness</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Reproductive system and breast disorders</title>
            <event_list>
              <event>
                <sub_title>Dysmenorrhoea</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Cough</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="2" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Paranasal sinus hypersecretion</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Sputum increased</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="2" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Dyspnoea</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Increased bronchial secretion</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Nasal congestion</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Nasal oedema</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Oropharyngeal pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Pharyngeal erythema</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Pharyngeal oedema</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Pulmonary congestion</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Rales</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Rhinorrhoea</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Sinus pain</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Night sweats</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E5" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Medical Monitor</name_or_title>
      <organization>Vertex Pharmaceuticals Incorporated</organization>
      <phone>617-341-6777</phone>
      <email>medicalinfo@vrtx.com</email>
    </point_of_contact>
  </clinical_results>
</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/NCT02971839
org study id: CP656.2001
nct id: NCT02971839
lead sponsor: 
has dmc: Yes
This study will evaluate the efficacy and safety of CTP-656 in patients with cystic fibrosis
      (CF) who have a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation.
This is a randomized, parallel-group, double-blind, placebo controlled multicenter study to
      evaluate the safety and efficacy of CTP-656 in CF patients with CFTR gating mutations,
      compared to Kalydeco, for a total of 28 days. Subjects will be randomized to receive either
      double-blind CTP-656 or placebo, or open-label Kalydeco.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
measure: Change From Baseline in Sweat Chloride at Day 28
time frame: From baseline at Day 28
measure: Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28
time frame: From baseline at Day 28
measure: Change From Baseline in Cystic Fibrosis Questionnaire-Respiratory Domain (CFQ-R) at Day 28
time frame: From baseline at Day 28
arm group label: VX-561 20 mg
arm group type: Experimental
arm group label: VX-561 100 mg
arm group type: Experimental
arm group label: VX-561 150 mg
arm group type: Experimental
arm group label: Ivacaftor
arm group type: Active Comparator
arm group label: Placebo
arm group type: Placebo Comparator
intervention type: Drug
intervention name: VX-561
arm group label: VX-561 100 mg
arm group label: VX-561 150 mg
arm group label: VX-561 20 mg
other name: CTP-656
intervention type: Drug
intervention name: Placebo
arm group label: Placebo
intervention type: Drug
intervention name: IVA
arm group label: Ivacaftor
other name: ivacaftor; VX-770
other name: Kalydeco
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
facility: 
facility: 
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facility: 
country: United States
responsible party type: Sponsor
mesh term: Cystic Fibrosis
mesh term: Fibrosis
mesh term: Ivacaftor
sharing ipd: No
provided document: 
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 