<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/NCT03229252</url>
  </required_header>
  <id_info>
    <org_study_id>SPX-101-CF-201</org_study_id>
    <nct_id>NCT03229252</nct_id>
  </id_info>
  <brief_title>An Efficacy and Safety Study of SPX-101 Inhalation Solution in Subjects With Cystic Fibrosis</brief_title>
  <acronym>(HOPE-1)</acronym>
  <official_title>A Randomized, Double-blind, Placebo-controlled Phase II Study to Evaluate the Efficacy and Safety of SPX-101 Inhalation Solution in Subjects With Cystic Fibrosis (HOPE-1 Study: Hydration for Optimal Pulmonary Effectiveness)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Spyryx Biosciences, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Spyryx Biosciences, Inc.</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      28-Day double-blinded efficacy and safety trial of SPX-101 Inhalation Solution in adult&#xD;
      subjects with cystic fibrosis.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">August 1, 2017</start_date>
  <completion_date type="Actual">June 20, 2019</completion_date>
  <primary_completion_date type="Actual">June 20, 2019</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)</masking>
    <masking_description>Double-Blind</masking_description>
  </study_design_info>
  <primary_outcome>
    <measure>Change in Percent Predicted FEV1</measure>
    <time_frame>Baseline and Day 28</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Number of Participants With Adverse Events</measure>
    <time_frame>Day 1 through Day 28</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline Through Day 28 in Clinical Laboratory Tests</measure>
    <time_frame>Day 1 through Day 28</time_frame>
    <description>Chemistry, Hematology, Urinalysis</description>
  </secondary_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Actual">91</enrollment>
  <condition>Cystic Fibrosis</condition>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Placebo Inhalation solution twice daily for 28 days.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>SPX-101 Low Dose</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Inhalation solution twice daily for 28 days.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>SPX-101 High Dose</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Inhalation solution twice daily for 28 days.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo Inhalation Solution</intervention_name>
    <description>Normal Saline Inhalation Solution</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>SPX-101</intervention_name>
    <description>SPX-101 Inhalation Solution</description>
    <arm_group_label>SPX-101 High Dose</arm_group_label>
    <arm_group_label>SPX-101 Low Dose</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Confirmed diagnosis of cystic fibrosis&#xD;
&#xD;
          -  ppFEV1 (percent predicted FEV1) between 50.0% and 80.0%&#xD;
&#xD;
          -  Stable CF Lung Disease&#xD;
&#xD;
          -  Males and non-pregnant, non-lactating females&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Significant unstable co-morbidities within 28 days of screening&#xD;
&#xD;
          -  Has received an investigational drug within 28 days of screening&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>50 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>University of Calgary Heritage Medical Research Center</name>
      <address>
        <city>Calgary</city>
        <state>Alberta</state>
        <zip>T2N 1N4</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ottawa Hospital Research Institute/Institut de Recherche de l'Hospital d'Ottawa</name>
      <address>
        <city>Ottawa</city>
        <state>Ontario</state>
        <zip>K1H 8L6</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Saint Michael's Hospital</name>
      <address>
        <city>Toronto</city>
        <state>Ontario</state>
        <zip>M5B1W8</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Centre Hospitalier Universitaire Brest</name>
      <address>
        <city>Roscoff</city>
        <state>Bretagne</state>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>CHU de Rouen</name>
      <address>
        <city>Rouen</city>
        <state>Haute-Normandie</state>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>CHU de Montpellier</name>
      <address>
        <city>Montpellier</city>
        <state>Languedoc-Roussillon</state>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>CHU de Angers</name>
      <address>
        <city>Angers</city>
        <state>Pays De La Loire</state>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hopital Pasteur</name>
      <address>
        <city>Nice</city>
        <state>Provence Alpes Cote D'azur</state>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospices Civils de Lyon (HCL)</name>
      <address>
        <city>Pierre Bénite</city>
        <state>Rhone-Alpes</state>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Assistance Publique-Hôpitaux de Paris Hôpital Cochin</name>
      <address>
        <city>Paris</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Istituto Giannina Gaslini Ospedale Pediatrico</name>
      <address>
        <city>Genoa</city>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital de Santa Maria</name>
      <address>
        <city>Lisbon</city>
        <country>Portugal</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Belfast Health and Social Care Trust</name>
      <address>
        <city>Belfast</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Heart of England NHS Foundation Trust</name>
      <address>
        <city>Birmingham</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Hospitals Bristol NHS Foundation Trust</name>
      <address>
        <city>Bristol</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Western General Hospital - NHS Lothian</name>
      <address>
        <city>Edinburgh</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Royal Devon and Exeter NHS Foundation Trust</name>
      <address>
        <city>Exeter</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>NHS Greater Glasgow and Clyde</name>
      <address>
        <city>Glasgow</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>The Leeds Teaching Hospitals NHS Trust</name>
      <address>
        <city>Leeds</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Liverpool Heart and Chest Hospital NHS Foundation Trust</name>
      <address>
        <city>Liverpool</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Barts Health NHS Trust Saint Bartholomews Hospital</name>
      <address>
        <city>London</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Royal Brompton and Harefield NHS Foundation Trust</name>
      <address>
        <city>London</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Hospital of South Manchester NHS Foundation Trust</name>
      <address>
        <city>Manchester</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Newcastle Upon Tyne Hospitals</name>
      <address>
        <city>Newcastle-upon-Tyne</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nottingham University Hospitals NHS Trust</name>
      <address>
        <city>Nottingham</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Canada</country>
    <country>France</country>
    <country>Italy</country>
    <country>Portugal</country>
    <country>United Kingdom</country>
  </location_countries>
  <verification_date>December 2019</verification_date>
  <study_first_submitted>July 14, 2017</study_first_submitted>
  <study_first_submitted_qc>July 24, 2017</study_first_submitted_qc>
  <study_first_posted type="Actual">July 25, 2017</study_first_posted>
  <results_first_submitted>August 26, 2019</results_first_submitted>
  <results_first_submitted_qc>December 6, 2019</results_first_submitted_qc>
  <results_first_posted type="Actual">December 30, 2019</results_first_posted>
  <last_update_submitted>December 6, 2019</last_update_submitted>
  <last_update_submitted_qc>December 6, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">December 30, 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>Cystic Fibrosis</mesh_term>
    <mesh_term>Fibrosis</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>No</document_has_sap>
      <document_date>May 14, 2018</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/52/NCT03229252/Prot_000.pdf</document_url>
    </provided_document>
    <provided_document>
      <document_type>Statistical Analysis Plan</document_type>
      <document_has_protocol>No</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>July 9, 2019</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/52/NCT03229252/SAP_001.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>Placebo</title>
          <description>Placebo Inhalation solution twice daily for 28 days.&#xD;
Placebo Inhalation Solution: Normal Saline Inhalation Solution</description>
        </group>
        <group group_id="P2">
          <title>SPX-101 Low Dose (60mg BID)</title>
          <description>Inhalation solution twice daily for 28 days.&#xD;
SPX-101: SPX-101 Inhalation Solution</description>
        </group>
        <group group_id="P3">
          <title>SPX-101 High Dose (120mg BID)</title>
          <description>Inhalation solution twice daily for 28 days.&#xD;
SPX-101: SPX-101 Inhalation Solution</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="31"/>
                <participants group_id="P2" count="15"/>
                <participants group_id="P3" count="45"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="29"/>
                <participants group_id="P2" count="15"/>
                <participants group_id="P3" count="39"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
                <participants group_id="P2" count="0"/>
                <participants group_id="P3" count="6"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Placebo</title>
          <description>Placebo Inhalation solution twice daily for 28 days.&#xD;
Placebo Inhalation Solution: Normal Saline Inhalation Solution</description>
        </group>
        <group group_id="B2">
          <title>SPX-101 Low Dose (60mg BID)</title>
          <description>Inhalation solution twice daily for 28 days.&#xD;
SPX-101: SPX-101 Inhalation Solution</description>
        </group>
        <group group_id="B3">
          <title>SPX-101 High Dose (120mg BID)</title>
          <description>Inhalation solution twice daily for 28 days.&#xD;
SPX-101: SPX-101 Inhalation Solution</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="31"/>
            <count group_id="B2" value="15"/>
            <count group_id="B3" value="45"/>
            <count group_id="B4" value="91"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>&lt;=18 years</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>Between 18 and 65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="31"/>
                    <measurement group_id="B2" value="15"/>
                    <measurement group_id="B3" value="45"/>
                    <measurement group_id="B4" value="91"/>
                  </measurement_list>
                </category>
                <category>
                  <title>&gt;=65 years</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>
          <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="30.8" spread="7.82"/>
                    <measurement group_id="B2" value="33.7" spread="8.62"/>
                    <measurement group_id="B3" value="31.9" spread="8.49"/>
                    <measurement group_id="B4" value="31.8" spread="8.25"/>
                  </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="11"/>
                    <measurement group_id="B2" value="4"/>
                    <measurement group_id="B3" value="22"/>
                    <measurement group_id="B4" value="37"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="20"/>
                    <measurement group_id="B2" value="11"/>
                    <measurement group_id="B3" value="23"/>
                    <measurement group_id="B4" value="54"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Ethnicity (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="2"/>
                    <measurement group_id="B4" value="3"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="29"/>
                    <measurement group_id="B2" value="14"/>
                    <measurement group_id="B3" value="43"/>
                    <measurement group_id="B4" value="86"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="2"/>
                  </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="1"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="26"/>
                    <measurement group_id="B2" value="12"/>
                    <measurement group_id="B3" value="44"/>
                    <measurement group_id="B4" value="82"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                    <measurement group_id="B2" value="3"/>
                    <measurement group_id="B3" value="1"/>
                    <measurement group_id="B4" value="7"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                    <measurement group_id="B4" value="1"/>
                  </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>Canada</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                    <measurement group_id="B2" value="5"/>
                    <measurement group_id="B3" value="8"/>
                    <measurement group_id="B4" value="16"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Italy</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="3"/>
                    <measurement group_id="B4" value="4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>United Kingdom</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="14"/>
                    <measurement group_id="B2" value="7"/>
                    <measurement group_id="B3" value="15"/>
                    <measurement group_id="B4" value="36"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>France</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="12"/>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="13"/>
                    <measurement group_id="B4" value="27"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Portugal</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="6"/>
                    <measurement group_id="B4" value="8"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Change in Percent Predicted FEV1</title>
        <time_frame>Baseline and Day 28</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Placebo</title>
            <description>Placebo Inhalation solution twice daily for 28 days.&#xD;
Placebo Inhalation Solution: Normal Saline Inhalation Solution</description>
          </group>
          <group group_id="O2">
            <title>SPX-101 Low Dose (60mg BID)</title>
            <description>Inhalation solution twice daily for 28 days.&#xD;
SPX-101: SPX-101 Inhalation Solution</description>
          </group>
          <group group_id="O3">
            <title>SPX-101 High Dose (120mg BID)</title>
            <description>Inhalation solution twice daily for 28 days.&#xD;
SPX-101: SPX-101 Inhalation Solution</description>
          </group>
        </group_list>
        <measure>
          <title>Change in Percent Predicted FEV1</title>
          <units>change from baseline in ppFEV1</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="31"/>
                <count group_id="O2" value="15"/>
                <count group_id="O3" value="45"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.633" spread="6.3302"/>
                    <measurement group_id="O2" value="0.800" spread="6.5049"/>
                    <measurement group_id="O3" value="0.890" spread="6.7061"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Number of Participants With Adverse Events</title>
        <time_frame>Day 1 through Day 28</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Placebo</title>
            <description>Placebo Inhalation solution twice daily for 28 days.&#xD;
Placebo Inhalation Solution: Normal Saline Inhalation Solution</description>
          </group>
          <group group_id="O2">
            <title>SPX-101 Low Dose (60mg BID)</title>
            <description>Inhalation solution twice daily for 28 days.&#xD;
SPX-101: SPX-101 Inhalation Solution</description>
          </group>
          <group group_id="O3">
            <title>SPX-101 High Dose (120mg BID)</title>
            <description>Inhalation solution twice daily for 28 days.&#xD;
SPX-101: SPX-101 Inhalation Solution</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With Adverse Events</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="31"/>
                <count group_id="O2" value="15"/>
                <count group_id="O3" value="45"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="20"/>
                    <measurement group_id="O2" value="11"/>
                    <measurement group_id="O3" value="30"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline Through Day 28 in Clinical Laboratory Tests</title>
        <description>Chemistry, Hematology, Urinalysis</description>
        <time_frame>Day 1 through Day 28</time_frame>
        <group_list>
          <group group_id="O1">
            <title>Placebo</title>
            <description>Placebo Inhalation solution twice daily for 28 days.&#xD;
Placebo Inhalation Solution: Normal Saline Inhalation Solution</description>
          </group>
          <group group_id="O2">
            <title>SPX-101 Low Dose (60mg BID)</title>
            <description>Inhalation solution twice daily for 28 days.&#xD;
SPX-101: SPX-101 Inhalation Solution</description>
          </group>
          <group group_id="O3">
            <title>SPX-101 High Dose (120mg BID)</title>
            <description>Inhalation solution twice daily for 28 days.&#xD;
SPX-101: SPX-101 Inhalation Solution</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline Through Day 28 in Clinical Laboratory Tests</title>
          <description>Chemistry, Hematology, Urinalysis</description>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="31"/>
                <count group_id="O2" value="15"/>
                <count group_id="O3" value="45"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Blood Electrolytes (Potassium) Low</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="0"/>
                    <measurement group_id="O3" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Blood Electrolytes (Potassium) Normal</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="29"/>
                    <measurement group_id="O2" value="13"/>
                    <measurement group_id="O3" value="40"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Blood Electrolytes (Potassium) High</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="1"/>
                    <measurement group_id="O3" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>28 Days</time_frame>
      <group_list>
        <group group_id="E1">
          <title>Placebo</title>
          <description>Placebo Inhalation solution twice daily for 28 days.&#xD;
Placebo Inhalation Solution: Normal Saline Inhalation Solution</description>
        </group>
        <group group_id="E2">
          <title>SPX-101 Low Dose (60mg BID)</title>
          <description>Inhalation solution twice daily for 28 days.&#xD;
SPX-101: SPX-101 Inhalation Solution</description>
        </group>
        <group group_id="E3">
          <title>SPX-101 High Dose (120mg BID)</title>
          <description>Inhalation solution twice daily for 28 days.&#xD;
SPX-101: SPX-101 Inhalation Solution</description>
        </group>
      </group_list>
      <serious_events>
        <default_assessment>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="31"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="15"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="31"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="15"/>
                <counts group_id="E3" subjects_affected="3" subjects_at_risk="45"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Cardiac disorders</title>
            <event_list>
              <event>
                <sub_title>Tachycardia</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="31"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="45"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Intestinal Obstuction</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="31"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="45"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Infective Pulmonary Exacerbation of Cystic Fibrosis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="31"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="45"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Cough and Dyspnea</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="31"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
                <counts group_id="E3" events="1" subjects_affected="1" subjects_at_risk="45"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <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="20" subjects_at_risk="31"/>
                <counts group_id="E2" subjects_affected="10" subjects_at_risk="15"/>
                <counts group_id="E3" subjects_affected="29" subjects_at_risk="45"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Diarrhea</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="31"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="15"/>
                <counts group_id="E3" events="2" subjects_affected="2" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Constipation</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="31"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="15"/>
                <counts group_id="E3" events="2" subjects_affected="2" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Frequent Bowel Movements</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="31"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="15"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Tongue Coated</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="31"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="15"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="45"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Chest Discomfort</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="31"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="15"/>
                <counts group_id="E3" events="3" subjects_affected="2" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Fatigue</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="31"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Pyrexia</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="31"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="15"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="45"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Forced Expiratory Volume Decreased</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="31"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
                <counts group_id="E3" events="4" subjects_affected="4" subjects_at_risk="45"/>
              </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="31"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
                <counts group_id="E3" events="4" subjects_affected="4" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Dysgeusia</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="31"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Migraine</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="31"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="15"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="45"/>
              </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="6" subjects_affected="6" subjects_at_risk="31"/>
                <counts group_id="E2" events="4" subjects_affected="4" subjects_at_risk="15"/>
                <counts group_id="E3" events="6" subjects_affected="6" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Sputum Increased</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="31"/>
                <counts group_id="E2" events="3" subjects_affected="3" subjects_at_risk="15"/>
                <counts group_id="E3" events="4" subjects_affected="4" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Wheezing</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="31"/>
                <counts group_id="E2" events="2" subjects_affected="1" subjects_at_risk="15"/>
                <counts group_id="E3" events="7" subjects_affected="4" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Bronchospasm</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="31"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
                <counts group_id="E3" events="5" subjects_affected="5" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Dyspnea</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="31"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
                <counts group_id="E3" events="4" subjects_affected="4" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Epistaxis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="31"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="15"/>
                <counts group_id="E3" events="2" subjects_affected="2" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Nasal Congestion</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="31"/>
                <counts group_id="E2" events="3" subjects_affected="3" subjects_at_risk="15"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Lower Respiratory Tract Congestion</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="31"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Increased Viscosity of Bronchial Secretion</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="31"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="15"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Sputum Discolored</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="31"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="15"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Infective Pulmonary Exacerbation of Cystic Fibrosis</sub_title>
                <counts group_id="E1" events="4" subjects_affected="4" subjects_at_risk="31"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="15"/>
                <counts group_id="E3" events="3" subjects_affected="3" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Nasopharyngitis</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="31"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="15"/>
                <counts group_id="E3" events="3" subjects_affected="3" subjects_at_risk="45"/>
              </event>
              <event>
                <sub_title>Upper Respiratory Tract Infection</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="31"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="15"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="45"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Eczema</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="31"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="15"/>
                <counts group_id="E3" events="0" subjects_affected="0" subjects_at_risk="45"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed. </restrictive_agreement>
    </certain_agreements>
    <limitations_and_caveats>No Limitations or Caveats</limitations_and_caveats>
    <point_of_contact>
      <name_or_title>Dr. Rob Tarran</name_or_title>
      <organization>Spyryx Biosciences</organization>
      <phone>919-966-7052</phone>
      <email>rtarran@spyryxbio.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/NCT03229252
org study id: SPX-101-CF-201
nct id: NCT03229252
lead sponsor: 
has dmc: Yes
is fda regulated drug: No
is fda regulated device: No
28-Day double-blinded efficacy and safety trial of SPX-101 Inhalation Solution in adult
      subjects with cystic fibrosis.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
masking description: Double-Blind
measure: Change in Percent Predicted FEV1
time frame: Baseline and Day 28
measure: Number of Participants With Adverse Events
time frame: Day 1 through Day 28
measure: Change From Baseline Through Day 28 in Clinical Laboratory Tests
time frame: Day 1 through Day 28
description: Chemistry, Hematology, Urinalysis
arm group label: Placebo
arm group type: Placebo Comparator
description: Placebo Inhalation solution twice daily for 28 days.
arm group label: SPX-101 Low Dose
arm group type: Experimental
description: Inhalation solution twice daily for 28 days.
arm group label: SPX-101 High Dose
arm group type: Experimental
description: Inhalation solution twice daily for 28 days.
intervention type: Drug
intervention name: Placebo Inhalation Solution
description: Normal Saline Inhalation Solution
arm group label: Placebo
intervention type: Drug
intervention name: SPX-101
description: SPX-101 Inhalation Solution
arm group label: SPX-101 High Dose
arm group label: SPX-101 Low Dose
criteria: 
gender: All
minimum age: 18 Years
maximum age: 50 Years
healthy volunteers: No
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
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country: Canada
country: France
country: Italy
country: Portugal
country: United Kingdom
responsible party type: Sponsor
mesh term: Cystic Fibrosis
mesh term: Fibrosis
sharing ipd: No
provided document: 
provided document: 
participant flow: 
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outcome list: 
reported events: 
certain agreements: 
limitations and caveats: No Limitations or Caveats
point of contact: 