<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/NCT02217332</url>
  </required_header>
  <id_info>
    <org_study_id>KNS-76704-CS201</org_study_id>
    <nct_id>NCT02217332</nct_id>
  </id_info>
  <brief_title>Study of Dexpramipexole Chronic Sinusitis With Nasal Polyps and Eosinophilia</brief_title>
  <acronym>CS201</acronym>
  <official_title>Safety and Preliminary Efficacy of Dexpramipexole in Patients With Chronic Sinusitis With Nasal Polyps and Eosinophilia (CSNP-E)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Knopp Biosciences</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Knopp Biosciences</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      Phase 2, open-label, multi-center study to evaluate the clinical effects of oral&#xD;
      administration of dexpramipexole for 6 months in subjects with chronic sinusitis with nasal&#xD;
      polyps and eosinophilia.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This open-labelled study will evaluate the safety and preliminary efficacy of dexpramipexole&#xD;
      for reducing the number of eosinophils in the peripheral blood and in improving nasal polyp&#xD;
      score when administered to 20 subjects with chronic sinusitis with nasal polyps and&#xD;
      eosinophilia.&#xD;
&#xD;
      Subjects will received dexpramipexole for up to 6 months and will have safety tests performed&#xD;
      monthly and will have efficacy evaluations performed at month 1, month 3, and month 6 after&#xD;
      beginning study drug.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>August 2014</start_date>
  <completion_date type="Actual">January 20, 2017</completion_date>
  <primary_completion_date type="Actual">December 14, 2016</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>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Ratio to Baseline in Peripheral Blood Eosinophil Counts After 6 Months of Treatment</measure>
    <time_frame>Baseline and Month 6</time_frame>
    <description>Change in peripheral blood eosinophil (measured in cells/μL) from baseline to month 6 is presented as ratio to baseline.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Change From Baseline in Total Polyp Score (TPS) After 6 Months of Treatment</measure>
    <time_frame>Baseline and Month 6</time_frame>
    <description>The change in Nasal Polyp Score (NPS) score after 6 months of treatment is presented. A blinded Central Endoscopy Rater determined the NPS for each nare on a scale of 0 to 4, and the bilateral NPS scores were added to generate the Total Polyp Score (TPS) on a scale of 0 to 8. A lower TPS corresponds to a lower polyp burden.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Number of Subjects With Potentially Clinically Significant Values in Clinical Laboratory Evaluations</measure>
    <time_frame>6 months</time_frame>
    <description>Summary of subjects from the safety population who experienced potentially clinically significant values in hand serum chemistry results</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of Subjects With Potentially Clinically Significant Values in Vital Signs and Weight</measure>
    <time_frame>6 months</time_frame>
    <description>Summary of subjects from the safety population who experienced potentially clinically significant values or changes in vital signs or weight</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of Subjects With Potentially Significant Values in Electrocardiogram Parameters</measure>
    <time_frame>6 months</time_frame>
    <description>Summary of subjects from the safety population who experienced potentially significant values in electrocardiogram parameters</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Ratio to Baseline in Peripheral Blood Eosinophil Counts After 3 Months of Treatment</measure>
    <time_frame>Baseline and Month 3</time_frame>
    <description>Change in peripheral blood eosinophil (measured in cells/μL) from baseline to month 3 is presented as ratio to baseline.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in TPS After 3 Months of Treatment</measure>
    <time_frame>Baseline and Month 3</time_frame>
    <description>The change in Nasal Polyp Score (NPS) score after 3 months of treatment is presented. A blinded Central Endoscopy Rater determined the NPS for each nare on a scale of 0 to 4, and the bilateral NPS scores were added to generate the Total Polyp Score (TPS) on a scale of 0 to 8. A lower TPS corresponds to a lower polyp burden.</description>
  </secondary_outcome>
  <other_outcome>
    <measure>Ratio to Baseline in Polyp Tissue Eosinophil Count After 6 Months of Treatment</measure>
    <time_frame>Baseline and Month 6</time_frame>
    <description>Change in polyp tissue eosinophil count measured as eosinophils/hpf (high powered field) from baseline to month 6 is presented as ratio to baseline.</description>
  </other_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">20</enrollment>
  <condition>Chronic Sinusitis With Nasal Polyps and Eosinophilia</condition>
  <arm_group>
    <arm_group_label>dexpramipexole</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>dexpramipexole 150 mg BID</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>dexpramipexole</intervention_name>
    <description>Dexpramipexole capsule-shaped film-coated tablets at the dose strength of 150 mg administered orally as 1 tablet twice daily (300 mg/day)</description>
    <arm_group_label>dexpramipexole</arm_group_label>
    <other_name>KNS-760704</other_name>
    <other_name>BIIB050</other_name>
    <other_name>RPPX</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Male or female &gt;18 or &lt;70 years of age&#xD;
&#xD;
          -  Willing to practice effective contraception during the study and be willing and able&#xD;
             to continue contraception for 1 month (females) or 3 months (males) after the last&#xD;
             dose of study treatment&#xD;
&#xD;
          -  Confirmed diagnosis of chronic sinusitis with nasal polyps&#xD;
&#xD;
          -  Documented history of nasal eosinophilia&#xD;
&#xD;
          -  Documented peripheral absolute eosinophil count &gt;300 cells/μL&#xD;
&#xD;
          -  Bilateral total polyp score of &gt;4&#xD;
&#xD;
          -  Sino-nasal outcome test (SNOT-22) score of &gt;7&#xD;
&#xD;
          -  Using an intranasal corticosteroid spray or irrigation (&lt; 1000 μg/day beclomethasone&#xD;
             or equivalent)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Acute sinusitis, concurrent nasal infection, or subjects who have had a nasal or upper&#xD;
             respiratory tract infection within 2 weeks prior to baseline&#xD;
&#xD;
          -  CT scan suggestive of allergic fungal rhinosinusitis&#xD;
&#xD;
          -  Nasal septal deviation that would occlude at least one nostril&#xD;
&#xD;
          -  Nasal surgery (including polypectomy) within 6 months prior to baseline&#xD;
&#xD;
          -  History of more than 5 sinonasal surgeries requiring general anesthesia&#xD;
&#xD;
          -  History of more than 2 sinonasal surgeries that changed the lateral wall of the nose&#xD;
&#xD;
          -  History of cystic fibrosis, primary ciliary's dysfunction or Kartagener's syndrome&#xD;
&#xD;
          -  History of diagnosis with a parasitic infection&#xD;
&#xD;
          -  Hospitalization or emergency treatment for the treatment of asthma two or more times&#xD;
             in the 12 months prior to baseline&#xD;
&#xD;
          -  Hospitalization for an acute asthmatic attack within 4 weeks prior to baseline&#xD;
&#xD;
          -  Forced expiratory volume (FEV1) of &lt;60% of predicted normal range&#xD;
&#xD;
          -  Treatment with a systemic corticosteroid or intra-polyp corticosteroid within 8 weeks&#xD;
             prior to baseline or anticipated need for systemic corticosteroids during the study&#xD;
             treatment period&#xD;
&#xD;
          -  Utilization of rescue oral corticosteroids for asthma or chronic sinusitis&#xD;
             exacerbation more than one time within the past 1 year&#xD;
&#xD;
          -  Treatment with an investigational drug in the previous 30 days or 5-half-lives,&#xD;
             whichever is longer&#xD;
&#xD;
          -  Treatment with a monoclonal antibody therapy including omalizumab (Xolair®), within&#xD;
             5-half-lives&#xD;
&#xD;
          -  Treatment with zileuton (Zyflo®) within 4 weeks of baseline&#xD;
&#xD;
          -  Treatment with pramipexole (Mirapex®) within 4 weeks of baseline&#xD;
&#xD;
          -  History of malignancy, including solid tumors and hematologic malignancies (except&#xD;
             basal cell and squamous cell cancers of the skin that have been completely excised and&#xD;
             cured)&#xD;
&#xD;
          -  History of human immunodeficiency virus (HIV) or hepatitis B or C&#xD;
&#xD;
          -  History of unstable or severe cardiac, hepatic, or renal disease, or other medically&#xD;
             significant illness&#xD;
&#xD;
          -  Medical or other condition likely to interfere with subject's ability to undergo study&#xD;
             procedures, adhere to visit schedule or comply with study requirements&#xD;
&#xD;
          -  Absolute neutrophil count &lt;2000 cells/μL at screening, or any documented history of&#xD;
             neutropenia&#xD;
&#xD;
          -  Total IgE &gt;1500 IU/ml at any visit prior to baseline&#xD;
&#xD;
          -  Renal dysfunction, defined as an estimated glomerular filtration rate (eGFR) of ≤80&#xD;
             mg/dL at screening (estimation of creatinine clearance using the MDRD formula)&#xD;
&#xD;
          -  History of long QT syndrome or arrhythmia&#xD;
&#xD;
          -  Prolongation of QT/QTc interval (e.g., repeated demonstration of a QT/QTc interval&#xD;
             &gt;450 ms) at screening or pre-dose on day 1&#xD;
&#xD;
          -  Clinically important abnormalities in resting ECG that may interfere with the&#xD;
             interpretation of QTc interval changes at screening or pre-dose on day 1, including&#xD;
             any of the following:&#xD;
&#xD;
               -  PR interval &gt;210 ms;&#xD;
&#xD;
               -  QRS &gt;110 ms;&#xD;
&#xD;
               -  Heart rate &lt;45 bpm or &gt;100 bpm (average of 3 assessments).&#xD;
&#xD;
          -  Pregnant women or women breastfeeding&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>70 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>ENT Associates of South Florida</name>
      <address>
        <city>Boca Raton</city>
        <state>Florida</state>
        <zip>33487</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Johns Hopkins University</name>
      <address>
        <city>Baltimore</city>
        <state>Maryland</state>
        <zip>21205</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Brigham and Women's Hospital</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Wake Research Associates</name>
      <address>
        <city>Raleigh</city>
        <state>North Carolina</state>
        <zip>27612</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Pennsylvania</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Pittsburgh School of Medicine</name>
      <address>
        <city>Pittsburgh</city>
        <state>Pennsylvania</state>
        <zip>15213</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Virginia</name>
      <address>
        <city>Charlottesville</city>
        <state>Virginia</state>
        <zip>22903</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>April 2021</verification_date>
  <study_first_submitted>August 12, 2014</study_first_submitted>
  <study_first_submitted_qc>August 13, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">August 15, 2014</study_first_posted>
  <results_first_submitted>March 29, 2021</results_first_submitted>
  <results_first_submitted_qc>April 26, 2021</results_first_submitted_qc>
  <results_first_posted type="Actual">May 18, 2021</results_first_posted>
  <disposition_first_submitted>December 12, 2017</disposition_first_submitted>
  <disposition_first_submitted_qc>December 12, 2017</disposition_first_submitted_qc>
  <disposition_first_posted type="Actual">December 14, 2017</disposition_first_posted>
  <last_update_submitted>April 26, 2021</last_update_submitted>
  <last_update_submitted_qc>April 26, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">May 18, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Sinusitis</keyword>
  <keyword>Rhinosinusitis</keyword>
  <keyword>CRS</keyword>
  <keyword>CRSwNP</keyword>
  <keyword>CSNP-E</keyword>
  <keyword>Nasal polyps</keyword>
  <keyword>Nasal polyposis</keyword>
  <keyword>Eosinophilia</keyword>
  <keyword>Asthma</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Sinusitis</mesh_term>
    <mesh_term>Nasal Polyps</mesh_term>
    <mesh_term>Eosinophilia</mesh_term>
    <mesh_term>Polyps</mesh_term>
    <mesh_term>Chronic Disease</mesh_term>
  </condition_browse>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Pramipexole</mesh_term>
  </intervention_browse>

  <clinical_results>

    <participant_flow>
      <pre_assignment_details>During a screening period of between 14 and 21 days, subjects were monitored symptoms of sinusitis and asthma. Subject who failed to meet the eligibility criteria during the screening period were disqualified.</pre_assignment_details>
      <group_list>
        <group group_id="P1">
          <title>Dexpramipexole</title>
          <description>dexpramipexole 150 mg BID&#xD;
dexpramipexole: Dexpramipexole capsule-shaped film-coated tablets at the dose strength of 150 mg administered orally as 1 tablet twice daily (300 mg/day)</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="20">Safety Population</participants>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="13">Subjects who continued to optional open-label treatment</participants>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="7"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Adverse Event</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Dose interruption &gt;1 month</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Protocol Violation (eligibility)</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Withdrawal by Subject</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Lost to Follow-up</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Dexpramipexole</title>
          <description>dexpramipexole 150 mg BID&#xD;
dexpramipexole: Dexpramipexole capsule-shaped film-coated tablets at the dose strength of 150 mg administered orally as 1 tablet twice daily (300 mg/day)</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="20"/>
          </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="46.1" spread="9.78"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Age, Customized</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>&lt; 50 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="14"/>
                  </measurement_list>
                </category>
                <category>
                  <title>50 to 65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="5"/>
                  </measurement_list>
                </category>
                <category>
                  <title>&gt; 65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                  </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="8"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" 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_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="19"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                  </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_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Native Hawaiian or Other Pacific Islander</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="17"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>United States</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="20"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>CSNP-E Symptoms</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <title>Anterior and/or posterior mucopurulent drainage</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="20"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Sinonasal obstruction/congestion</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="20"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Decreased sense of smell (anosmia)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="20"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Duration of Symptoms (months)</title>
          <units>months</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="112.4" spread="138.13"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Duration of symptoms</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <title>≤ 1 year</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>&gt; 1 year to ≤ 5 years</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>&gt; 5 years to ≤ 10 years</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>&gt; 10 years</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Ratio to Baseline in Peripheral Blood Eosinophil Counts After 6 Months of Treatment</title>
        <description>Change in peripheral blood eosinophil (measured in cells/μL) from baseline to month 6 is presented as ratio to baseline.</description>
        <time_frame>Baseline and Month 6</time_frame>
        <population>All subjects who had a Month 3 evaluation (on study drug Day 77 or later) for TPS and peripheral blood eosinophil count. Last observation carried forward (LOCF) was used to replace missing data due to drop out. Only Month 3 data or later (assessment date Day 77 or later and within 7 days of last dose) were used.</population>
        <group_list>
          <group group_id="O1">
            <title>Dexpramipexole</title>
            <description>dexpramipexole 150 mg BID&#xD;
dexpramipexole: Dexpramipexole capsule-shaped film-coated tablets at the dose strength of 150 mg administered orally as 1 tablet twice daily (300 mg/day)</description>
          </group>
        </group_list>
        <measure>
          <title>Ratio to Baseline in Peripheral Blood Eosinophil Counts After 6 Months of Treatment</title>
          <description>Change in peripheral blood eosinophil (measured in cells/μL) from baseline to month 6 is presented as ratio to baseline.</description>
          <population>All subjects who had a Month 3 evaluation (on study drug Day 77 or later) for TPS and peripheral blood eosinophil count. Last observation carried forward (LOCF) was used to replace missing data due to drop out. Only Month 3 data or later (assessment date Day 77 or later and within 7 days of last dose) were used.</population>
          <units>ratio to baseline</units>
          <param>Geometric Mean</param>
          <dispersion>95% Confidence Interval</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="16"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.06" lower_limit="0.023" upper_limit="0.154"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <groups_desc>Null hypothesis is the ratio of Month 6 to Baseline within the dexpramipexole group equals '1'.</groups_desc>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>&lt; 0.001</p_value>
            <method>t-test, 2 sided</method>
            <method_desc>Paired t-test comparing baseline to month 6 on log scale within the dexpramipexole treatment group.</method_desc>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Change From Baseline in Total Polyp Score (TPS) After 6 Months of Treatment</title>
        <description>The change in Nasal Polyp Score (NPS) score after 6 months of treatment is presented. A blinded Central Endoscopy Rater determined the NPS for each nare on a scale of 0 to 4, and the bilateral NPS scores were added to generate the Total Polyp Score (TPS) on a scale of 0 to 8. A lower TPS corresponds to a lower polyp burden.</description>
        <time_frame>Baseline and Month 6</time_frame>
        <population>All subjects who had a Month 3 evaluation (on study drug Day 77 or later) for TPS and peripheral blood eosinophil count. Last observation carried forward (LOCF) was used to replace missing data due to drop out. Only Month 3 data or later (assessment date Day 77 or later and within 7 days of last dose) were used.</population>
        <group_list>
          <group group_id="O1">
            <title>Dexpramipexole</title>
            <description>dexpramipexole 150 mg BID&#xD;
dexpramipexole: Dexpramipexole capsule-shaped film-coated tablets at the dose strength of 150 mg administered orally as 1 tablet twice daily (300 mg/day)</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Total Polyp Score (TPS) After 6 Months of Treatment</title>
          <description>The change in Nasal Polyp Score (NPS) score after 6 months of treatment is presented. A blinded Central Endoscopy Rater determined the NPS for each nare on a scale of 0 to 4, and the bilateral NPS scores were added to generate the Total Polyp Score (TPS) on a scale of 0 to 8. A lower TPS corresponds to a lower polyp burden.</description>
          <population>All subjects who had a Month 3 evaluation (on study drug Day 77 or later) for TPS and peripheral blood eosinophil count. Last observation carried forward (LOCF) was used to replace missing data due to drop out. Only Month 3 data or later (assessment date Day 77 or later and within 7 days of last dose) were used.</population>
          <units>units on a scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="15"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.07" spread="1.751"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.885</p_value>
            <p_value_desc>Month 6/LOCF was used for the analysis of change from Baseline to Month 6 within the dexpramipexole group.</p_value_desc>
            <method>t-test, 2 sided</method>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Number of Subjects With Potentially Clinically Significant Values in Clinical Laboratory Evaluations</title>
        <description>Summary of subjects from the safety population who experienced potentially clinically significant values in hand serum chemistry results</description>
        <time_frame>6 months</time_frame>
        <population>The safety population included all subjects who received at least 1 dose of study treatment.</population>
        <group_list>
          <group group_id="O1">
            <title>Dexpramipexole</title>
            <description>dexpramipexole 150 mg BID&#xD;
dexpramipexole: Dexpramipexole capsule-shaped film-coated tablets at the dose strength of 150 mg administered orally as 1 tablet twice daily (300 mg/day)</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Subjects With Potentially Clinically Significant Values in Clinical Laboratory Evaluations</title>
          <description>Summary of subjects from the safety population who experienced potentially clinically significant values in hand serum chemistry results</description>
          <population>The safety population included all subjects who received at least 1 dose of study treatment.</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="20"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>LOW: Albumin &lt;= 25 g/L</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="20"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>HIGH: Alanine aminotransferase (ALT) &gt;= 1.5 x ULN</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="20"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>HIGH: Aspartate aminotransferase (AST) &gt;= 1.5 x ULN</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="20"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>HIGH: Total bilirubin &gt;= 1.5 x ULN</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="20"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>HIGH: Glucose&gt;= 9.7 mmol/L</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="20"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>LOW: Glucose &lt;= 2.2 mmol/L</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="20"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>HIGH: Eosinophils &gt; 1.6 x 10(9)/L</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="20"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>HIGH: Hematocrit - Females &gt;=54%</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="8"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>LOW: Hematocrit - Females &lt;= 32%</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="8"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>HIGH: Hemoglobin - Females &gt;=175g/L</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="8"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>LOW: Hemoglobin - Females &lt;= 95g/L</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="8"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>HIGH: Lymphocytes &gt; 12 x 10(9)/L</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="20"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>LOW: Lymphocytes &lt; 0.8 x 10(9)/L</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="20"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>LOW: Neutrophils &lt; 1.5 x 10(9)/L</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="20"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Number of Subjects With Potentially Clinically Significant Values in Vital Signs and Weight</title>
        <description>Summary of subjects from the safety population who experienced potentially clinically significant values or changes in vital signs or weight</description>
        <time_frame>6 months</time_frame>
        <population>The safety population included all subjects who received at least 1 dose of study treatment.</population>
        <group_list>
          <group group_id="O1">
            <title>Dexpramipexole</title>
            <description>dexpramipexole 150 mg BID&#xD;
dexpramipexole: Dexpramipexole capsule-shaped film-coated tablets at the dose strength of 150 mg administered orally as 1 tablet twice daily (300 mg/day)</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Subjects With Potentially Clinically Significant Values in Vital Signs and Weight</title>
          <description>Summary of subjects from the safety population who experienced potentially clinically significant values or changes in vital signs or weight</description>
          <population>The safety population included all subjects who received at least 1 dose of study treatment.</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="20"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Systolic Blood Pressure: &gt;180 mmHg</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Systolic Blood Pressure: increase from pre-dosing of more than 40 mmHg</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Systolic Blood Pressure: &lt;90 mmHg</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Systolic Blood Pressure: decrease from pre-dosing of more than 30 mmHg</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Diastolic Blood Pressure: &gt;105 mmHg</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Diastolic Blood Pressure: increase from pre-dosing of more than 30 mmHg</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Diastolic Blood Pressure: &lt;50 mmHg</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Diastolic Blood Pressure: decrease from pre-dosing of more than 20 mmHg</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Heart Rate: &gt;120 beats per minute</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Heart Rate: increase from pre-dosing of more than 20 beats per minute</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Heart Rate: &lt;50 beats per minute</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Heart Rate: decrease from pre-dosing of more than 20 beats per minute</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Temperature: &gt;38ºC and an increase from pre-dosing of at least 1ºC</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Body Weight: &gt;7% increase from baseline value</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Body Weight: &lt;=7% decrease from baseline value</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Number of Subjects With Potentially Significant Values in Electrocardiogram Parameters</title>
        <description>Summary of subjects from the safety population who experienced potentially significant values in electrocardiogram parameters</description>
        <time_frame>6 months</time_frame>
        <population>The safety population included all subjects who received at least 1 dose of study treatment.</population>
        <group_list>
          <group group_id="O1">
            <title>Dexpramipexole</title>
            <description>dexpramipexole 150 mg BID&#xD;
dexpramipexole: Dexpramipexole capsule-shaped film-coated tablets at the dose strength of 150 mg administered orally as 1 tablet twice daily (300 mg/day)</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Subjects With Potentially Significant Values in Electrocardiogram Parameters</title>
          <description>Summary of subjects from the safety population who experienced potentially significant values in electrocardiogram parameters</description>
          <population>The safety population included all subjects who received at least 1 dose of study treatment.</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="20"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>QTcF: &gt;450 msec</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>QTcF: &gt;480 msec</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>QTcF: &gt; 450 msec and ≤ 450 msec at baseline</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>QTcF: &gt; 480 msec and ≤ 480 msec at baseline</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>QTcF: &gt; 450 msec and change from baseline &gt; 30 msec</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>QTcF: &gt; 480 msec and change from baseline &gt; 30 msec</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>QTcF change from baseline: &gt;30 msec</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>QTcF change from baseline: &gt;60 msec</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Rhythm abnormalities: Sinus rhythm</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="17"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Rhythm abnormalities: Sinus bradycardia</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Rhythm abnormalities: Premature atrial complexes, conducted or non-conducted sinus rhythm</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>ST-T abnormalities: Nonspecific T wave abnormality</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>ST-T abnormalities: Early repolarization</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>ST-T abnormalities: Early repolarization, considered normal variant</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>ST-T abnormalities: Nonspecific ST deviation</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>ST-T abnormalities: Nonspecific ST deviation, prolonged QTcF interval</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>ST-T abnormalities: Nonspecific T wave abnormality, prolonged QTcF interval</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>ST-T abnormalities: Prolonged QTcF interval</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Conduction abnormality: First degree AV block</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Conduction abnormality: Complete right bundle branch block</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Conduction abnormality: Complete right bundle branch block first degree AV block</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Ratio to Baseline in Peripheral Blood Eosinophil Counts After 3 Months of Treatment</title>
        <description>Change in peripheral blood eosinophil (measured in cells/μL) from baseline to month 3 is presented as ratio to baseline.</description>
        <time_frame>Baseline and Month 3</time_frame>
        <population>All subjects who had a Month 3 evaluation (on study drug Day 77 or later) for total eosinophil count</population>
        <group_list>
          <group group_id="O1">
            <title>Dexpramipexole</title>
            <description>dexpramipexole 150 mg BID&#xD;
dexpramipexole: Dexpramipexole capsule-shaped film-coated tablets at the dose strength of 150 mg administered orally as 1 tablet twice daily (300 mg/day)</description>
          </group>
        </group_list>
        <measure>
          <title>Ratio to Baseline in Peripheral Blood Eosinophil Counts After 3 Months of Treatment</title>
          <description>Change in peripheral blood eosinophil (measured in cells/μL) from baseline to month 3 is presented as ratio to baseline.</description>
          <population>All subjects who had a Month 3 evaluation (on study drug Day 77 or later) for total eosinophil count</population>
          <units>ratio</units>
          <param>Geometric Mean</param>
          <dispersion>95% Confidence Interval</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="16"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.05" lower_limit="0.019" upper_limit="0.142"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <groups_desc>Null hypothesis is the ratio of Month 3 to Baseline within the dexpramipexole group equals '1'.</groups_desc>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>&lt;.001</p_value>
            <method>t-test, 2 sided</method>
            <method_desc>Paired t-test comparing baseline to month 3 on log scale within the dexpramipexole treatment group.</method_desc>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in TPS After 3 Months of Treatment</title>
        <description>The change in Nasal Polyp Score (NPS) score after 3 months of treatment is presented. A blinded Central Endoscopy Rater determined the NPS for each nare on a scale of 0 to 4, and the bilateral NPS scores were added to generate the Total Polyp Score (TPS) on a scale of 0 to 8. A lower TPS corresponds to a lower polyp burden.</description>
        <time_frame>Baseline and Month 3</time_frame>
        <population>All subjects who had a Month 3 evaluation (on study drug Day 77 or later) for total eosinophil count and total polyp score</population>
        <group_list>
          <group group_id="O1">
            <title>Dexpramipexole</title>
            <description>dexpramipexole 150 mg BID&#xD;
dexpramipexole: Dexpramipexole capsule-shaped film-coated tablets at the dose strength of 150 mg administered orally as 1 tablet twice daily (300 mg/day)</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in TPS After 3 Months of Treatment</title>
          <description>The change in Nasal Polyp Score (NPS) score after 3 months of treatment is presented. A blinded Central Endoscopy Rater determined the NPS for each nare on a scale of 0 to 4, and the bilateral NPS scores were added to generate the Total Polyp Score (TPS) on a scale of 0 to 8. A lower TPS corresponds to a lower polyp burden.</description>
          <population>All subjects who had a Month 3 evaluation (on study drug Day 77 or later) for total eosinophil count and total polyp score</population>
          <units>units on a scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="16"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.0" spread="1.414"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>1.0</p_value>
            <p_value_desc>Month 3/LOCF was used for the analysis of change from Baseline to Month 3 within the dexpramipexole group.</p_value_desc>
            <method>t-test, 2 sided</method>
            <method_desc>Paired t-test comparing baseline to month 3 within the dexpramipexole treatment group</method_desc>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Other Pre-specified</type>
        <title>Ratio to Baseline in Polyp Tissue Eosinophil Count After 6 Months of Treatment</title>
        <description>Change in polyp tissue eosinophil count measured as eosinophils/hpf (high powered field) from baseline to month 6 is presented as ratio to baseline.</description>
        <time_frame>Baseline and Month 6</time_frame>
        <population>All subjects who had a Month 3 evaluation (on study drug Day 77 or later) for TPS and peripheral blood eosinophil count (Efficacy Population). Additionally, subjects with &lt;5 eos/hpf at screening or baseline visit are not included in this analysis.</population>
        <group_list>
          <group group_id="O1">
            <title>Dexpramipexole</title>
            <description>dexpramipexole 150 mg BID&#xD;
dexpramipexole: Dexpramipexole capsule-shaped film-coated tablets at the dose strength of 150 mg administered orally as 1 tablet twice daily (300 mg/day)</description>
          </group>
        </group_list>
        <measure>
          <title>Ratio to Baseline in Polyp Tissue Eosinophil Count After 6 Months of Treatment</title>
          <description>Change in polyp tissue eosinophil count measured as eosinophils/hpf (high powered field) from baseline to month 6 is presented as ratio to baseline.</description>
          <population>All subjects who had a Month 3 evaluation (on study drug Day 77 or later) for TPS and peripheral blood eosinophil count (Efficacy Population). Additionally, subjects with &lt;5 eos/hpf at screening or baseline visit are not included in this analysis.</population>
          <units>ratio to baseline</units>
          <param>Geometric Mean</param>
          <dispersion>68% Confidence Interval</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="12"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.03" lower_limit="0.02" upper_limit=".06"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
            </group_id_list>
            <groups_desc>Null hypothesis is the ratio of Month 6 to Baseline within the dexpramipexole group equals '1'; 68% confidence interval is equal to plus/minus 1 standard error</groups_desc>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.001</p_value>
            <method>Wilcoxon Signed Rank Test (paired)</method>
          </analysis>
        </analysis_list>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>6 months on treatment</time_frame>
      <group_list>
        <group group_id="E1">
          <title>Dexpramipexole</title>
          <description>dexpramipexole 150 mg BID&#xD;
dexpramipexole: Dexpramipexole capsule-shaped film-coated tablets at the dose strength of 150 mg administered orally as 1 tablet twice daily (300 mg/day)</description>
        </group>
      </group_list>
      <serious_events>
        <default_vocab>MedDRA 16.0</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="20"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>biliary dyskinesia</sub_title>
                <description>The investigator and the sponsor both assessed the SAE of cholecystitis as not related to the study drug.</description>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <default_vocab>MedDRA 16.0</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="18" subjects_at_risk="20"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Ear and labyrinth disorders</title>
            <event_list>
              <event>
                <sub_title>Ear pain</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Vertigo</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Abdominal distension</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Abdominal pain</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Constipation</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Diarrhoea</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Dyspepsia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Eosinophilic oesophagitis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Flatulence</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Gastrooesophageal reflux disease</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="20"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Fatigue</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Oedema</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Xerosis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Hepatobiliary disorders</title>
            <event_list>
              <event>
                <sub_title>Biliary dyskinesia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Ear infection</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Folliculitis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Nasopharyngitis</sub_title>
                <counts group_id="E1" events="4" subjects_affected="3" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Onychomycosis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Oral candidiasis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Otitis media</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Rhinitis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Sinusitis</sub_title>
                <counts group_id="E1" events="12" subjects_affected="8" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Subcutaneous abscess</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Upper respiratory tract infection</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Decreased appetite</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Hyperglycemia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Hypoglycemia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Polydipsia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Arthralgia</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Pain in extremity</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Tendonitis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Sinus headache</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>Anxiety</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Middle insomnia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Sleep disorder</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Cough</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Dyspnoea</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Epistaxis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Intranasal paraesthesia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Nasal congestion</sub_title>
                <counts group_id="E1" events="4" subjects_affected="3" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Nasal septum ulceration</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Rales</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Rhinitis allergic</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Upper-airway cough syndrome</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Wheezing</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Dermatitis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Skin hyperpigmentation</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Stasis dermatitis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </event>
              <event>
                <sub_title>Urticaria</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="20"/>
              </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>Head of Regulatory</name_or_title>
      <organization>Knopp Biosciences</organization>
      <phone>4124881776</phone>
      <email>greg@knoppbio.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/NCT02217332
org study id: KNS-76704-CS201
nct id: NCT02217332
lead sponsor: 
has dmc: No
Phase 2, open-label, multi-center study to evaluate the clinical effects of oral
      administration of dexpramipexole for 6 months in subjects with chronic sinusitis with nasal
      polyps and eosinophilia.
This open-labelled study will evaluate the safety and preliminary efficacy of dexpramipexole
      for reducing the number of eosinophils in the peripheral blood and in improving nasal polyp
      score when administered to 20 subjects with chronic sinusitis with nasal polyps and
      eosinophilia.

      Subjects will received dexpramipexole for up to 6 months and will have safety tests performed
      monthly and will have efficacy evaluations performed at month 1, month 3, and month 6 after
      beginning study drug.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Ratio to Baseline in Peripheral Blood Eosinophil Counts After 6 Months of Treatment
time frame: Baseline and Month 6
description: Change in peripheral blood eosinophil (measured in cells/μL) from baseline to month 6 is presented as ratio to baseline.
measure: Change From Baseline in Total Polyp Score (TPS) After 6 Months of Treatment
time frame: Baseline and Month 6
description: The change in Nasal Polyp Score (NPS) score after 6 months of treatment is presented. A blinded Central Endoscopy Rater determined the NPS for each nare on a scale of 0 to 4, and the bilateral NPS scores were added to generate the Total Polyp Score (TPS) on a scale of 0 to 8. A lower TPS corresponds to a lower polyp burden.
measure: Number of Subjects With Potentially Clinically Significant Values in Clinical Laboratory Evaluations
time frame: 6 months
description: Summary of subjects from the safety population who experienced potentially clinically significant values in hand serum chemistry results
measure: Number of Subjects With Potentially Clinically Significant Values in Vital Signs and Weight
time frame: 6 months
description: Summary of subjects from the safety population who experienced potentially clinically significant values or changes in vital signs or weight
measure: Number of Subjects With Potentially Significant Values in Electrocardiogram Parameters
time frame: 6 months
description: Summary of subjects from the safety population who experienced potentially significant values in electrocardiogram parameters
measure: Ratio to Baseline in Peripheral Blood Eosinophil Counts After 3 Months of Treatment
time frame: Baseline and Month 3
description: Change in peripheral blood eosinophil (measured in cells/μL) from baseline to month 3 is presented as ratio to baseline.
measure: Change From Baseline in TPS After 3 Months of Treatment
time frame: Baseline and Month 3
description: The change in Nasal Polyp Score (NPS) score after 3 months of treatment is presented. A blinded Central Endoscopy Rater determined the NPS for each nare on a scale of 0 to 4, and the bilateral NPS scores were added to generate the Total Polyp Score (TPS) on a scale of 0 to 8. A lower TPS corresponds to a lower polyp burden.
measure: Ratio to Baseline in Polyp Tissue Eosinophil Count After 6 Months of Treatment
time frame: Baseline and Month 6
description: Change in polyp tissue eosinophil count measured as eosinophils/hpf (high powered field) from baseline to month 6 is presented as ratio to baseline.
arm group label: dexpramipexole
arm group type: Experimental
description: dexpramipexole 150 mg BID
intervention type: Drug
intervention name: dexpramipexole
description: Dexpramipexole capsule-shaped film-coated tablets at the dose strength of 150 mg administered orally as 1 tablet twice daily (300 mg/day)
arm group label: dexpramipexole
other name: KNS-760704
other name: BIIB050
other name: RPPX
criteria: 
gender: All
minimum age: 18 Years
maximum age: 70 Years
healthy volunteers: No
facility: 
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country: United States
responsible party type: Sponsor
mesh term: Sinusitis
mesh term: Nasal Polyps
mesh term: Eosinophilia
mesh term: Polyps
mesh term: Chronic Disease
mesh term: Pramipexole
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 