<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/NCT02989168</url>
  </required_header>
  <id_info>
    <org_study_id>GBT440-026</org_study_id>
    <nct_id>NCT02989168</nct_id>
  </id_info>
  <brief_title>Study to Evaluate the Effect of GBT440 Administered to Subjects With IPF on Supplemental Oxygen at Rest</brief_title>
  <acronym>Zephyr</acronym>
  <official_title>A Phase II Open Label Study to Evaluate the Effect of GBT440 on Hypoxemia in Subjects With Idiopathic Pulmonary Fibrosis (IPF) Who Are Using Supplemental Oxygen at Rest (ZEPHYR)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Global Blood Therapeutics</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Global Blood Therapeutics</source>
  <brief_summary>
    <textblock>
      This is an open label study in which eligible IPF subjects who are using supplemental oxygen&#xD;
      at rest will receive GBT440 orally daily.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    The study was terminated due to lack of clinically meaningful benefit&#xD;
  </why_stopped>
  <start_date>November 2016</start_date>
  <completion_date type="Actual">October 23, 2017</completion_date>
  <primary_completion_date type="Actual">October 23, 2017</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Sequential Assignment</intervention_model>
    <primary_purpose>Supportive Care</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Change in Oxygen Saturation at End of Treatment Period Compared to Baseline</measure>
    <time_frame>Days 1 to 90</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Change in Supplemental Oxygen Requirement at End of Treatment Period Compared to Baseline</measure>
    <time_frame>Days 1 to 90</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evaluate the Effect of GBT440 on Resting and Post-exercise Alveolar-arterial O2 Tension Difference [P(A-a) O2] at End of Treatment Period Compared to Baseline</measure>
    <time_frame>Days 1 to 90</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evaluate the Effect of GBT440 on Performance of the 6MWT</measure>
    <time_frame>Days 1 to 90</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evaluate the Effect of GBT440 on IPF Related Symptoms Using Patient Related Outcomes</measure>
    <time_frame>Days 1 to 90</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Evaluate Pulmonary Function Using Pulmonary Function Tests (FVC and DLco)</measure>
    <time_frame>Days 1 to 90</time_frame>
    <description>Spirometry measurements will include forced vital capacity (FVC) and diffusing capacity of the lung for carbon monoxide (DLco).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0</measure>
    <time_frame>Days 1 to 90</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Pharmacokinetic Parameters of GBT440 in Plasma and Whole Blood (Minimum Concentration (Cmin))</measure>
    <time_frame>Day 1 (15 mins post-dose and 2-4h post-dose), Day 15 (pre-dose), Day 30 (pre-dose), Day 60 (pre-dose and 2-4h post-dose), Day 90 (pre-dose), Day 105 (during study visit), and Day 120 (during study visit)</time_frame>
    <description>PK parameters of GBT440 administered daily orally for 90 days in plasma and whole blood, including but not limited to, minimum concentration (Cmin), at steady state.</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">14</enrollment>
  <condition>Idiopathic Pulmonary Fibrosis</condition>
  <condition>Hypoxemia</condition>
  <arm_group>
    <arm_group_label>GBT440 900 mg Dose</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Part A, 900 mg&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
  </arm_group>
  <arm_group>
    <arm_group_label>GBT440 1500 mg Dose</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Part B , 1500 mg&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>GBT440</intervention_name>
    <description>GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
    <arm_group_label>GBT440 1500 mg Dose</arm_group_label>
    <arm_group_label>GBT440 900 mg Dose</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Documented diagnosis of IPF.&#xD;
&#xD;
          -  Receiving supplemental oxygen for use at rest.&#xD;
&#xD;
          -  Weight ≥ 40 kg.&#xD;
&#xD;
          -  Male or female of child bearing potential willing and able to use highly effective&#xD;
             methods of contraception from study start to 30 days after the last dose of study&#xD;
             drug.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  FEV1/FVC &lt; 70%&#xD;
&#xD;
          -  History of other interstitial lung diseases.&#xD;
&#xD;
          -  Subject plans to begin or has commenced pulmonary rehabilitation within 30 days of&#xD;
             screening.&#xD;
&#xD;
          -  Corticosteroid (&gt; 10 mg per day of prednisone or an equivalent) administered for 7&#xD;
             days or longer, within 30 days of screening.&#xD;
&#xD;
          -  Participated in another clinical trial of an investigational drug (or medical device)&#xD;
             within 30 days or 5-half-lives, whichever is longer, prior to screening, or is&#xD;
             currently participating in another trial of an investigational drug (or medical&#xD;
             device).&#xD;
&#xD;
          -  Female who is breast-feeding or pregnant&#xD;
&#xD;
          -  Current smoker or history of smoking within 3 months from screening&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>45 Years</minimum_age>
    <maximum_age>85 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <address>
        <city>Birmingham</city>
        <state>Alabama</state>
        <zip>35233</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Denver</city>
        <state>Colorado</state>
        <zip>80206</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>New Orleans</city>
        <state>Louisiana</state>
        <zip>70122</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19140</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Nashville</city>
        <state>Tennessee</state>
        <zip>37232</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Salt Lake City</city>
        <state>Utah</state>
        <zip>84108</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>London</city>
        <zip>SW3 6NP</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Manchester</city>
        <zip>M23 9QZ</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Southampton</city>
        <zip>SO16 6YD</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <verification_date>July 2020</verification_date>
  <study_first_submitted>November 28, 2016</study_first_submitted>
  <study_first_submitted_qc>December 7, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 12, 2016</study_first_posted>
  <results_first_submitted>May 4, 2020</results_first_submitted>
  <results_first_submitted_qc>July 31, 2020</results_first_submitted_qc>
  <results_first_posted type="Actual">August 21, 2020</results_first_posted>
  <last_update_submitted>July 31, 2020</last_update_submitted>
  <last_update_submitted_qc>July 31, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">August 21, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Idiopathic pulmonary fibrosis</keyword>
  <keyword>hypoxemia</keyword>
  <keyword>oxygen</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Pulmonary Fibrosis</mesh_term>
    <mesh_term>Idiopathic Pulmonary Fibrosis</mesh_term>
    <mesh_term>Fibrosis</mesh_term>
    <mesh_term>Hypoxia</mesh_term>
  </condition_browse>
  <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>June 5, 2017</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/68/NCT02989168/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>November 15, 2017</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/68/NCT02989168/SAP_001.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>GBT440 900 mg Dose</title>
          <description>Part A , 900 mg daily dose&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
        </group>
        <group group_id="P2">
          <title>GBT440 1500 mg Dose</title>
          <description>Part B , 1500 mg daily dose&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="11"/>
                <participants group_id="P2" count="3"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="7"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="4"/>
                <participants group_id="P2" count="3"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Withdrawal by Subject</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Physician Decision</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Adverse Event</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="2"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Sponsor stopped study.</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>GBT440 900 mg Dose</title>
          <description>Part A, 900 mg daily dose&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
        </group>
        <group group_id="B2">
          <title>GBT440 1500 mg Dose</title>
          <description>Part B, 1500 mg daily dose&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
        </group>
        <group group_id="B3">
          <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="11"/>
            <count group_id="B2" value="3"/>
            <count group_id="B3" value="14"/>
          </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="68.3" spread="6.12"/>
                    <measurement group_id="B2" value="70.3" spread="5.69"/>
                    <measurement group_id="B3" value="68.7" spread="5.88"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Age</title>
          <units>years</units>
          <param>Median</param>
          <dispersion>Full Range</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="69.0" lower_limit="59" upper_limit="77"/>
                    <measurement group_id="B2" value="72.0" lower_limit="64" upper_limit="75"/>
                    <measurement group_id="B3" value="70.0" lower_limit="59" upper_limit="77"/>
                  </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="1"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="2"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="10"/>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="12"/>
                  </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_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="11"/>
                    <measurement group_id="B2" value="3"/>
                    <measurement group_id="B3" value="14"/>
                  </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_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_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_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_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_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="10"/>
                    <measurement group_id="B2" value="3"/>
                    <measurement group_id="B3" value="13"/>
                  </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_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Change in Oxygen Saturation at End of Treatment Period Compared to Baseline</title>
        <time_frame>Days 1 to 90</time_frame>
        <population>Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented.</population>
        <group_list>
          <group group_id="O1">
            <title>GBT440 900 mg Dose</title>
            <description>Part A&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
          </group>
          <group group_id="O2">
            <title>GBT440 1500 mg Dose</title>
            <description>Part B&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
          </group>
        </group_list>
        <measure>
          <title>Change in Oxygen Saturation at End of Treatment Period Compared to Baseline</title>
          <population>Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented.</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_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change in Supplemental Oxygen Requirement at End of Treatment Period Compared to Baseline</title>
        <time_frame>Days 1 to 90</time_frame>
        <population>Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented.</population>
        <group_list>
          <group group_id="O1">
            <title>GBT440 900 mg Dose</title>
            <description>Part A&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
          </group>
          <group group_id="O2">
            <title>GBT440 1500 mg Dose</title>
            <description>Part B&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
          </group>
        </group_list>
        <measure>
          <title>Change in Supplemental Oxygen Requirement at End of Treatment Period Compared to Baseline</title>
          <population>Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented.</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_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Evaluate the Effect of GBT440 on Resting and Post-exercise Alveolar-arterial O2 Tension Difference [P(A-a) O2] at End of Treatment Period Compared to Baseline</title>
        <time_frame>Days 1 to 90</time_frame>
        <population>Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented.</population>
        <group_list>
          <group group_id="O1">
            <title>GBT440 900 mg Dose</title>
            <description>Part A&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
          </group>
          <group group_id="O2">
            <title>GBT440 1500 mg Dose</title>
            <description>Part B&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
          </group>
        </group_list>
        <measure>
          <title>Evaluate the Effect of GBT440 on Resting and Post-exercise Alveolar-arterial O2 Tension Difference [P(A-a) O2] at End of Treatment Period Compared to Baseline</title>
          <population>Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented.</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_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Evaluate the Effect of GBT440 on Performance of the 6MWT</title>
        <time_frame>Days 1 to 90</time_frame>
        <population>Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented.</population>
        <group_list>
          <group group_id="O1">
            <title>GBT440 900 mg Dose</title>
            <description>Part A&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
          </group>
          <group group_id="O2">
            <title>GBT440 1500 mg Dose</title>
            <description>Part B&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
          </group>
        </group_list>
        <measure>
          <title>Evaluate the Effect of GBT440 on Performance of the 6MWT</title>
          <population>Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented.</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_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Evaluate the Effect of GBT440 on IPF Related Symptoms Using Patient Related Outcomes</title>
        <time_frame>Days 1 to 90</time_frame>
        <population>Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented.</population>
        <group_list>
          <group group_id="O1">
            <title>GBT440 900 mg Dose</title>
            <description>Part A&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
          </group>
          <group group_id="O2">
            <title>GBT440 1500 mg Dose</title>
            <description>Part B&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
          </group>
        </group_list>
        <measure>
          <title>Evaluate the Effect of GBT440 on IPF Related Symptoms Using Patient Related Outcomes</title>
          <population>Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented.</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_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Evaluate Pulmonary Function Using Pulmonary Function Tests (FVC and DLco)</title>
        <description>Spirometry measurements will include forced vital capacity (FVC) and diffusing capacity of the lung for carbon monoxide (DLco).</description>
        <time_frame>Days 1 to 90</time_frame>
        <population>Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented.</population>
        <group_list>
          <group group_id="O1">
            <title>GBT440 900 mg Dose</title>
            <description>Part A&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
          </group>
          <group group_id="O2">
            <title>GBT440 1500 mg Dose</title>
            <description>Part B&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
          </group>
        </group_list>
        <measure>
          <title>Evaluate Pulmonary Function Using Pulmonary Function Tests (FVC and DLco)</title>
          <description>Spirometry measurements will include forced vital capacity (FVC) and diffusing capacity of the lung for carbon monoxide (DLco).</description>
          <population>Since study was stopped prematurely, complete data for all participants were not analyzable so no efficacy results are presented.</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_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0</title>
        <time_frame>Days 1 to 90</time_frame>
        <population>Participant had at least one dose of study drug</population>
        <group_list>
          <group group_id="O1">
            <title>GBT440 900 mg Dose</title>
            <description>Part A&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
          </group>
          <group group_id="O2">
            <title>GBT440 1500 mg Dose</title>
            <description>Part B&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0</title>
          <population>Participant had at least one dose of study drug</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="11"/>
                <count group_id="O2" value="3"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="11"/>
                    <measurement group_id="O2" value="3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Pharmacokinetic Parameters of GBT440 in Plasma and Whole Blood (Minimum Concentration (Cmin))</title>
        <description>PK parameters of GBT440 administered daily orally for 90 days in plasma and whole blood, including but not limited to, minimum concentration (Cmin), at steady state.</description>
        <time_frame>Day 1 (15 mins post-dose and 2-4h post-dose), Day 15 (pre-dose), Day 30 (pre-dose), Day 60 (pre-dose and 2-4h post-dose), Day 90 (pre-dose), Day 105 (during study visit), and Day 120 (during study visit)</time_frame>
        <population>Complete PK data were not analyzable for any participant.</population>
        <group_list>
          <group group_id="O1">
            <title>GBT440 900 mg Dose</title>
            <description>Part A , 900 mg daily dose&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
          </group>
          <group group_id="O2">
            <title>GBT440 1500 mg Dose</title>
            <description>Part B , 1500 mg daily dose&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
          </group>
        </group_list>
        <measure>
          <title>Pharmacokinetic Parameters of GBT440 in Plasma and Whole Blood (Minimum Concentration (Cmin))</title>
          <description>PK parameters of GBT440 administered daily orally for 90 days in plasma and whole blood, including but not limited to, minimum concentration (Cmin), at steady state.</description>
          <population>Complete PK data were not analyzable for any participant.</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_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Screening to 90 days</time_frame>
      <group_list>
        <group group_id="E1">
          <title>GBT440 900 mg Dose</title>
          <description>Part A , 900 mg daily dose&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
        </group>
        <group group_id="E2">
          <title>GBT440 1500 mg Dose</title>
          <description>Part B , 1500 mg daily dose&#xD;
GBT440: Capsules which contain GBT440 drug substance in Swedish orange</description>
        </group>
      </group_list>
      <serious_events>
        <default_vocab>MedDRA 20.0</default_vocab>
        <default_assessment>Non-systematic Assessment</default_assessment>
        <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="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Cardiac disorders</title>
            <event_list>
              <event>
                <sub_title>Pericardial effusion</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Cardiac failure congestive</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Non-cardiac chest pain</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Idiopathic pulmonary fibrosis</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Pleural effusion</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>0</frequency_threshold>
        <default_vocab>MedDRA 20.0</default_vocab>
        <default_assessment>Non-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="11" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="3" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Cardiac disorders</title>
            <event_list>
              <event>
                <sub_title>Atrial flutter</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Diarrhoea</sub_title>
                <counts group_id="E1" subjects_affected="4" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Nausea</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Fatigue</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Non-cardiac chest pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Asthenia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Oedema Peripheral</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Hepatobiliary disorders</title>
            <event_list>
              <event>
                <sub_title>Hepatic mass</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Bronchitis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Oral herpes</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Sinusitis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Viral sinusitis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Tooth fracture</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Decreased appetite</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Hyperkalaemia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Musculoskeletal pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Neoplasms benign, malignant and unspecified (incl cysts and polyps)</title>
            <event_list>
              <event>
                <sub_title>Adenocarcinoma</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" 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="2" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Dizziness</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>Insomnia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Reproductive system and breast disorders</title>
            <event_list>
              <event>
                <sub_title>Erectile dysfunction</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Dyspnoea</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Cough</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Pleural effusion</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Productive cough</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Rash</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Pruritus</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="3"/>
              </event>
              <event>
                <sub_title>Rash generalised</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="11"/>
                <counts group_id="E2" 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>
      <restrictive_agreement>There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed. </restrictive_agreement>
    </certain_agreements>
    <limitations_and_caveats>Since this study was stopped prematurely, no efficacy results will be presented.</limitations_and_caveats>
    <point_of_contact>
      <name_or_title>Nick Vlahakis</name_or_title>
      <organization>Global Blood Therapeutics</organization>
      <phone>16508228756</phone>
      <email>nvlahakis@gbt.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/NCT02989168
org study id: GBT440-026
nct id: NCT02989168
lead sponsor: 
This is an open label study in which eligible IPF subjects who are using supplemental oxygen
      at rest will receive GBT440 orally daily.
allocation: Non-Randomized
intervention model: Sequential Assignment
primary purpose: Supportive Care
masking: None (Open Label)
measure: Change in Oxygen Saturation at End of Treatment Period Compared to Baseline
time frame: Days 1 to 90
measure: Change in Supplemental Oxygen Requirement at End of Treatment Period Compared to Baseline
time frame: Days 1 to 90
measure: Evaluate the Effect of GBT440 on Resting and Post-exercise Alveolar-arterial O2 Tension Difference [P(A-a) O2] at End of Treatment Period Compared to Baseline
time frame: Days 1 to 90
measure: Evaluate the Effect of GBT440 on Performance of the 6MWT
time frame: Days 1 to 90
measure: Evaluate the Effect of GBT440 on IPF Related Symptoms Using Patient Related Outcomes
time frame: Days 1 to 90
measure: Evaluate Pulmonary Function Using Pulmonary Function Tests (FVC and DLco)
time frame: Days 1 to 90
description: Spirometry measurements will include forced vital capacity (FVC) and diffusing capacity of the lung for carbon monoxide (DLco).
measure: Number of Participants With Treatment-related Adverse Events as Assessed by CTCAE v4.0
time frame: Days 1 to 90
measure: Pharmacokinetic Parameters of GBT440 in Plasma and Whole Blood (Minimum Concentration (Cmin))
time frame: Day 1 (15 mins post-dose and 2-4h post-dose), Day 15 (pre-dose), Day 30 (pre-dose), Day 60 (pre-dose and 2-4h post-dose), Day 90 (pre-dose), Day 105 (during study visit), and Day 120 (during study visit)
description: PK parameters of GBT440 administered daily orally for 90 days in plasma and whole blood, including but not limited to, minimum concentration (Cmin), at steady state.
arm group label: GBT440 900 mg Dose
arm group type: Experimental
description: Part A, 900 mg
GBT440: Capsules which contain GBT440 drug substance in Swedish orange
arm group label: GBT440 1500 mg Dose
arm group type: Experimental
description: Part B , 1500 mg
GBT440: Capsules which contain GBT440 drug substance in Swedish orange
intervention type: Drug
intervention name: GBT440
description: GBT440: Capsules which contain GBT440 drug substance in Swedish orange
arm group label: GBT440 1500 mg Dose
arm group label: GBT440 900 mg Dose
criteria: 
gender: All
minimum age: 45 Years
maximum age: 85 Years
healthy volunteers: No
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: United Kingdom
country: United States
responsible party type: Sponsor
mesh term: Pulmonary Fibrosis
mesh term: Idiopathic Pulmonary Fibrosis
mesh term: Fibrosis
mesh term: Hypoxia
provided document: 
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
limitations and caveats: Since this study was stopped prematurely, no efficacy results will be presented.
point of contact: 