<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/NCT03041909</url>
  </required_header>
  <id_info>
    <org_study_id>GBT440-024</org_study_id>
    <nct_id>NCT03041909</nct_id>
  </id_info>
  <brief_title>An Extension Study to Further Evaluate the Safety, Tolerability of GBT440 in Patients With Sickle Cell Disease Who Participated in the Study GBT440-001</brief_title>
  <official_title>An Open Label Single Arm Extension Study to Further Evaluate the Safety, Tolerability and Treatment Response of GBT440 in Patients With Sickle Cell Disease Who Participated in the Phase 1 Study GBT440-001</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Global Blood Therapeutics</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Global Blood Therapeutics</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is an open label, single arm study which enrolled 5 subjects with SCD who previously&#xD;
      participated in the GBT440-001 study (NCT02285088).&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is an open label, single arm study which enrolled 5 subjects with SCD who previously&#xD;
      participated in the GBT440-001 study (NCT02285088).&#xD;
&#xD;
      Dosing of study drug was 2 to 6 months, depending on subject's dose assignment in the last&#xD;
      administration of study drug in GBT440-001 (NCT02285088).&#xD;
&#xD;
      The primary objective of the study was to evaluate the safety and tolerability of up to a&#xD;
      total of 6 months dosing of subjects with SCD who participated in the GBT440-001 study&#xD;
      (NCT02285088).&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>August 2016</start_date>
  <completion_date type="Actual">August 2017</completion_date>
  <primary_completion_date type="Actual">June 2017</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Other</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months.</measure>
    <time_frame>2 - 6 months</time_frame>
    <description>The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia</measure>
    <time_frame>2 - 6 months</time_frame>
    <description>Data presented are hemoglobin value collected at specific time points.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>To Observed Pharmacokinetics in Plasma and Whole Blood.</measure>
    <time_frame>2 - 6 months</time_frame>
    <description>Measure maximum plasma concentration (Cmax)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>To Characterize the Effect of GBT440 on Hemolysis.</measure>
    <time_frame>2 - 6 months</time_frame>
    <description>Data presented for unconjugated bilirubin at specific time point.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">5</enrollment>
  <condition>Sickle Cell Disease</condition>
  <arm_group>
    <arm_group_label>Single Arm</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Single Arm / open label</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>GBT440</intervention_name>
    <description>Oral drug</description>
    <arm_group_label>Single Arm</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Male or female subjects with SCD aged 18 to 60 years inclusive and &gt;50 kg who have&#xD;
             participated in the GBT440-001 study.&#xD;
&#xD;
          2. Subjects, who if female and of child bearing potential, agree to continue to use&#xD;
             highly effective methods of contraception prior to enrollment in this study and for 3&#xD;
             months after the last dose of study drug.&#xD;
&#xD;
          3. Subjects, who if male are willing to continue to use barrier methods of contraception,&#xD;
             prior to enrollment in this study to 3 months after the last dose of study drug.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Subjects requiring chronic transfusion therapy.&#xD;
&#xD;
          2. Subjects receiving a blood transfusion within 30 days of enrollment in this study.&#xD;
&#xD;
          3. Female subjects who are pregnant, trying to become pregnant or lactating.&#xD;
&#xD;
          4. Subjects who have a clinically relevant history or presence of respiratory,&#xD;
             gastrointestinal, renal, hepatic, haematological, lymphatic, neurological,&#xD;
             cardiovascular, psychiatric, musculoskeletal, genitourinary, immunological,&#xD;
             dermatological, connective tissue diseases or disorders, or additional risk factors&#xD;
             for torsades de pointe (e.g., heart failure, hypokalemia, personal or family history&#xD;
             of long QTc interval).&#xD;
&#xD;
          5. Subjects who have a significant infection or known inflammatory process on admission&#xD;
             to this study.&#xD;
&#xD;
          6. Subjects who have acute gastrointestinal symptoms at the time of admission (e.g.&#xD;
             nausea, vomiting, diarrhoea, heartburn).&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>60 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Josh Lehrer-Graiwer, MD</last_name>
    <role>Study Director</role>
    <affiliation>Global Blood Therapeutics, Inc.</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Timothy Mant, FRCP, FFPM</last_name>
    <role>Principal Investigator</role>
    <affiliation>Quintiles, Inc.</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>The BRC Research Facility, Floor 15 The Tower Wing</name>
      <address>
        <city>London</city>
        <zip>SE1 9RT</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United Kingdom</country>
  </location_countries>
  <verification_date>December 2018</verification_date>
  <study_first_submitted>January 19, 2017</study_first_submitted>
  <study_first_submitted_qc>February 1, 2017</study_first_submitted_qc>
  <study_first_posted type="Estimate">February 3, 2017</study_first_posted>
  <results_first_submitted>October 2, 2018</results_first_submitted>
  <results_first_submitted_qc>December 18, 2018</results_first_submitted_qc>
  <results_first_posted type="Actual">January 2, 2019</results_first_posted>
  <last_update_submitted>December 18, 2018</last_update_submitted>
  <last_update_submitted_qc>December 18, 2018</last_update_submitted_qc>
  <last_update_posted type="Actual">January 2, 2019</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>Anemia, Sickle Cell</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_info_type>Study Protocol</ipd_info_type>
  </patient_data>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol and Statistical Analysis Plan</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>September 28, 2016</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/09/NCT03041909/Prot_SAP_000.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <recruitment_details>Subjects enrolled in this study participated in GBT440-001 study (NCT02285088).</recruitment_details>
      <pre_assignment_details>There was no screening period as subjects transitioned directly from the GBT440-001 study (NCT02285088).into this study.</pre_assignment_details>
      <group_list>
        <group group_id="P1">
          <title>GBT440 - 2 Months</title>
          <description>These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (n=3) (NCT02285088).</description>
        </group>
        <group group_id="P2">
          <title>GBT440 - 6 Months</title>
          <description>The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (n=1) (NCT02285088).</description>
        </group>
        <group group_id="P3">
          <title>GBT440 - 4 Months</title>
          <description>This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (n=1) (NCT02285088).</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
                <participants group_id="P2" count="1"/>
                <participants group_id="P3" count="1"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
                <participants group_id="P2" count="1"/>
                <participants group_id="P3" count="1"/>
              </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_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>GBT440 - 2 Months</title>
          <description>These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (NCT02285088) (n=3).</description>
        </group>
        <group group_id="B2">
          <title>GBT440 - 6 Months</title>
          <description>The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (NCT02285088) (n=1).</description>
        </group>
        <group group_id="B3">
          <title>GBT440 - 4 Months</title>
          <description>This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (NCT02285088) (n=1).</description>
        </group>
        <group group_id="B4">
          <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="3"/>
            <count group_id="B2" value="1"/>
            <count group_id="B3" value="1"/>
            <count group_id="B4" value="5"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>Years</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="35" lower_limit="25" upper_limit="42"/>
                    <measurement group_id="B2" value="26"/>
                    <measurement group_id="B3" value="21"/>
                    <measurement group_id="B4" value="30" lower_limit="21" upper_limit="42"/>
                  </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="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="1"/>
                    <measurement group_id="B4" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="4"/>
                  </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_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_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_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="1"/>
                    <measurement group_id="B4" value="5"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</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_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_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_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months.</title>
        <description>The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events.</description>
        <time_frame>2 - 6 months</time_frame>
        <population>Safety</population>
        <group_list>
          <group group_id="O1">
            <title>GBT440 - 2 Months</title>
            <description>These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (n=3) (NCT02285088).</description>
          </group>
          <group group_id="O2">
            <title>GBT440 - 6 Months</title>
            <description>The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (n=1) (NCT02285088).</description>
          </group>
          <group group_id="O3">
            <title>GBT440 - 4 Months</title>
            <description>This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (n=1) (NCT02285088).</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months.</title>
          <description>The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events.</description>
          <population>Safety</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="3"/>
                <count group_id="O2" value="1"/>
                <count group_id="O3" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                    <measurement group_id="O2" value="1"/>
                    <measurement group_id="O3" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia</title>
        <description>Data presented are hemoglobin value collected at specific time points.</description>
        <time_frame>2 - 6 months</time_frame>
        <group_list>
          <group group_id="O1">
            <title>GBT440 - 2 Months</title>
            <description>These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (n=3) (NCT02285088).</description>
          </group>
          <group group_id="O2">
            <title>GBT440 - 6 Months</title>
            <description>The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (n=1) (NCT02285088).</description>
          </group>
          <group group_id="O3">
            <title>GBT440 - 4 Months</title>
            <description>This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (n=1) (NCT02285088).</description>
          </group>
        </group_list>
        <measure>
          <title>To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia</title>
          <description>Data presented are hemoglobin value collected at specific time points.</description>
          <units>g/dL</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="3"/>
                <count group_id="O2" value="1"/>
                <count group_id="O3" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="NA">Data is not reported due to participant privacy.</measurement>
                    <measurement group_id="O2" value="NA">Data is not reported due to participant privacy.</measurement>
                    <measurement group_id="O3" value="NA">Data is not reported due to participant privacy.</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>To Observed Pharmacokinetics in Plasma and Whole Blood.</title>
        <description>Measure maximum plasma concentration (Cmax)</description>
        <time_frame>2 - 6 months</time_frame>
        <group_list>
          <group group_id="O1">
            <title>GBT440 - 2 Months</title>
            <description>These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (n=3) (NCT02285088).</description>
          </group>
          <group group_id="O2">
            <title>GBT440 - 6 Months</title>
            <description>The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (n=1) (NCT02285088).</description>
          </group>
          <group group_id="O3">
            <title>GBT440 - 4 Months</title>
            <description>This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (n=1) (NCT02285088).</description>
          </group>
        </group_list>
        <measure>
          <title>To Observed Pharmacokinetics in Plasma and Whole Blood.</title>
          <description>Measure maximum plasma concentration (Cmax)</description>
          <units>ug/mL</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="3"/>
                <count group_id="O2" value="1"/>
                <count group_id="O3" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="NA">Data is not reported due to participant privacy.</measurement>
                    <measurement group_id="O2" value="NA">Data is not reported due to participant privacy.</measurement>
                    <measurement group_id="O3" value="NA">Data is not reported due to participant privacy.</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>To Characterize the Effect of GBT440 on Hemolysis.</title>
        <description>Data presented for unconjugated bilirubin at specific time point.</description>
        <time_frame>2 - 6 months</time_frame>
        <group_list>
          <group group_id="O1">
            <title>GBT440 - 2 Months</title>
            <description>These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (n=3) (NCT02285088).</description>
          </group>
          <group group_id="O2">
            <title>GBT440 - 6 Months</title>
            <description>The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (n=1) (NCT02285088).</description>
          </group>
          <group group_id="O3">
            <title>GBT440 - 4 Months</title>
            <description>This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (n=1) (NCT02285088).</description>
          </group>
        </group_list>
        <measure>
          <title>To Characterize the Effect of GBT440 on Hemolysis.</title>
          <description>Data presented for unconjugated bilirubin at specific time point.</description>
          <units>umol/L</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="3"/>
                <count group_id="O2" value="1"/>
                <count group_id="O3" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="NA">Data is not reported due to participant privacy.</measurement>
                    <measurement group_id="O2" value="NA">Data is not reported due to participant privacy.</measurement>
                    <measurement group_id="O3" value="NA">Data is not reported due to participant privacy.</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>10 months</time_frame>
      <group_list>
        <group group_id="E1">
          <title>GBT440 - 2 Months</title>
          <description>These subjects received GBT440 for 2 months as these subjects received GBT440 for 4 months in GBT440-001 study (NCT02285088) (n=3).</description>
        </group>
        <group group_id="E2">
          <title>GBT440 - 6 Months</title>
          <description>The subject received GBT440 for 6 months as this subject received placebo in GBT440-001 study (NCT02285088) (n=1).</description>
        </group>
        <group group_id="E3">
          <title>GBT440 - 4 Months</title>
          <description>This subject received GBT440 for 4 months as the subject received GBT440 for 2 months in GBT440-001 study (NCT02285088) (n=1).</description>
        </group>
      </group_list>
      <serious_events>
        <default_vocab>MedDRA (17.1)</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="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>Sickle Cell Anemia with Crisis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="1"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Surgical and medical procedures</title>
            <event_list>
              <event>
                <sub_title>Cholecystectomy</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <default_vocab>MedDRA (17.1)</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="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="1"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Diarrhea</sub_title>
                <assessment>Non-systematic Assessment</assessment>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>Toothache</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="1"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>Abdominal Pain</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="1"/>
              </event>
              <event>
                <sub_title>Gingival Swelling</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="1"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Upper Respiratory Tract Infection</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="1"/>
              </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>The sponsor and the PI will have to agree on any publications regarding the study.</restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Margaret Tonda, Sr. Director, Clinical Science</name_or_title>
      <organization>Global Blood Therapeutics</organization>
      <phone>650-741-7761</phone>
      <email>mtonda@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/NCT03041909
org study id: GBT440-024
nct id: NCT03041909
lead sponsor: 
has dmc: No
This is an open label, single arm study which enrolled 5 subjects with SCD who previously
      participated in the GBT440-001 study (NCT02285088).
This is an open label, single arm study which enrolled 5 subjects with SCD who previously
      participated in the GBT440-001 study (NCT02285088).

      Dosing of study drug was 2 to 6 months, depending on subject's dose assignment in the last
      administration of study drug in GBT440-001 (NCT02285088).

      The primary objective of the study was to evaluate the safety and tolerability of up to a
      total of 6 months dosing of subjects with SCD who participated in the GBT440-001 study
      (NCT02285088).
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Other
masking: None (Open Label)
measure: Number of Participants With Treatment-Emergent Adverse Events During Dosing of GBT440 for up to 6 Months.
time frame: 2 - 6 months
description: The safety evaluation will include physical examinations, blood pressure, clinical laboratory tests (hematology, serum biochemistry) and adverse events.
measure: To Assess the Efficacy of GBT440 as Measured by Improvements in Anemia
time frame: 2 - 6 months
description: Data presented are hemoglobin value collected at specific time points.
measure: To Observed Pharmacokinetics in Plasma and Whole Blood.
time frame: 2 - 6 months
description: Measure maximum plasma concentration (Cmax)
measure: To Characterize the Effect of GBT440 on Hemolysis.
time frame: 2 - 6 months
description: Data presented for unconjugated bilirubin at specific time point.
arm group label: Single Arm
arm group type: Experimental
description: Single Arm / open label
intervention type: Drug
intervention name: GBT440
description: Oral drug
arm group label: Single Arm
criteria: 
gender: All
minimum age: 18 Years
maximum age: 60 Years
healthy volunteers: No
last name: Josh Lehrer-Graiwer, MD
role: Study Director
affiliation: Global Blood Therapeutics, Inc.
last name: Timothy Mant, FRCP, FFPM
role: Principal Investigator
affiliation: Quintiles, Inc.
facility: 
country: United Kingdom
responsible party type: Sponsor
mesh term: Anemia, Sickle Cell
sharing ipd: Yes
ipd info type: Study Protocol
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 