<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/NCT04415073</url>
  </required_header>
  <id_info>
    <org_study_id>SNDX-6352-0505</org_study_id>
    <nct_id>NCT04415073</nct_id>
  </id_info>
  <brief_title>A Phase 2 Study to Evaluate Axatilimab for Hospitalized Participants With Respiratory Involvement Secondary to COVID-19</brief_title>
  <official_title>A Phase 2, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Axatilimab for the Treatment of Hospitalized Patients With Respiratory Signs and Symptoms Secondary to Novel Coronavirus Disease (COVID-19)</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Syndax Pharmaceuticals</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Syndax Pharmaceuticals</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This was a randomized, double-blind, placebo-controlled, 29-day study to assess the efficacy&#xD;
      and safety of axatilimab plus standard of care (SOC), compared with placebo plus SOC, in&#xD;
      participants with respiratory signs and symptoms secondary to COVID-19.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Axatilimab (SNDX-6352) is a humanized immunoglobin G4 monoclonal antibody with high affinity&#xD;
      against colony stimulating factor-1 receptor (CSF-1R) under investigation for the prevention&#xD;
      or treatment of respiratory signs and symptoms secondary to COVID-19.&#xD;
&#xD;
      This was a Phase 2, randomized, double-blind, placebo-controlled, multicenter study to&#xD;
      evaluate the efficacy, safety and tolerability of axatilimab as an add-on to SOC therapy in&#xD;
      hospitalized participants with respiratory signs and symptoms secondary to COVID-19 compared&#xD;
      to SOC treatment.&#xD;
&#xD;
      Eligible participants were to be randomized in a 1:1 ratio to 1 of 2 treatment groups, active&#xD;
      or control. All participants were to receive axatilimab or matching placebo intravenously&#xD;
      (IV) as an add-on to SOC on Day 1, within 8 hours of randomization and on Day 15.&#xD;
      Participants were to be followed for at least 28 days (+3 days) after the first dose of study&#xD;
      intervention (Day 29).&#xD;
&#xD;
      The primary objective of the study was to assess the proportion of participants alive and&#xD;
      free of respiratory failure at Day 29.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    Given enrollment challenges, partly attributable to the constantly changing COVID-19 treatment&#xD;
    landscape, the trial has been closed.&#xD;
  </why_stopped>
  <start_date type="Actual">May 30, 2020</start_date>
  <completion_date type="Actual">July 13, 2020</completion_date>
  <primary_completion_date type="Actual">July 13, 2020</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Double (Participant, Care Provider)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Count Of Participants Alive And Free Of Respiratory Failure At Day 29</measure>
    <time_frame>Day 29</time_frame>
    <description>Respiratory failure was defined by need for mechanical ventilation, extracorporeal membrane oxygenation, non-invasive ventilation &gt;6 liters oxygen/minute, or clinical diagnosis of respiratory failure with initiation of none of these measures only when clinical decision-making was driven solely by resource limitation.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Count Of Participants With Secondary Clinical Improvement Outcomes At Day 29</measure>
    <time_frame>Day 29</time_frame>
    <description>Participants achieving a ≥2 category improvement on a 7-point ordinal score relative to the baseline on Day 28 as collected on Day 29 are reported.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Time To Clinical Improvement</measure>
    <time_frame>Baseline through Day29</time_frame>
    <description>The time to clinical improvement is defined as a national early warning score of ≤2 maintained for 24 hours.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline At Day 29 In Oxygenation In Hospitalized Adults With Respiratory Signs And Symptoms Secondary To COVID-19 Treated With Axatilimab</measure>
    <time_frame>Baseline, Day 29</time_frame>
    <description>The change from baseline at Day 29 or hospital discharge or death, if sooner, in the ratio of peripheral hemoglobin oxygen saturation to fraction of inspired oxygen is evaluated.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline At Day 15 In Biomarkers Following Treatment With Axatilimab</measure>
    <time_frame>Baseline, Day 15</time_frame>
    <description>The change from baseline at Day 15 in serum concentrations of interleukin 6 and c-reactive protein or hospital discharge or death is evaluated.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Count Of Participants Experiencing Adverse Events And Serious Adverse Events</measure>
    <time_frame>Baseline through Day 29</time_frame>
    <description>This outcome measure evaluates the safety and tolerability of axatilimab within the same population. A summary of all serious adverse events and other adverse events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Count Of Participants Requiring Ventilation Support</measure>
    <time_frame>Baseline through Day 29</time_frame>
    <description>Participants who required initiation of mechanical ventilation after study entry are analyzed.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Count Of Participants Considered Severe Acute Respiratory Syndrome Coronavirus (SARS-CoV-2) Free By Day 15</measure>
    <time_frame>Day 15</time_frame>
    <description>This outcome measure evaluates the antiviral effects of axatilimab in hospitalized adults by Day 15 or hospital discharge, whichever was sooner, with recently diagnosed SARS-CoV-2 infection.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Pharmacokinetics: Serum Concentration Of Axatilimab And Anti-drug Antibodies</measure>
    <time_frame>Day 29</time_frame>
    <description>The serum concentration of axatilimab and presence of anti-drug antibodies is evaluated.</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">1</enrollment>
  <condition>Coronavirus</condition>
  <condition>COVID</condition>
  <condition>ARDS</condition>
  <condition>Cytokine Storm</condition>
  <condition>Cytokine Release Syndrome</condition>
  <arm_group>
    <arm_group_label>Axatilimab (SNDX-6352)</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
    <description>Axatilimab on Days 1 and 15, IV + SOC</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Matching placebo on Days 1 and 15, IV + SOC</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>SNDX-6352</intervention_name>
    <description>SNDX-6352</description>
    <arm_group_label>Axatilimab (SNDX-6352)</arm_group_label>
    <other_name>Axatilimab</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>Placebo comparator</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
        Type of Participant and Disease Characteristics -&#xD;
&#xD;
          1. Documented or confirmed severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2)&#xD;
             infection by an FDA-approved polymerase chain reaction test of nasopharyngeal swab or&#xD;
             stool less than 72 hours before randomization&#xD;
&#xD;
          2. Hospitalized for COVID-19&#xD;
&#xD;
          3. Illness of any duration with at least 1 of the following:&#xD;
&#xD;
               1. Clinical assessment (evidence of rales/crackles on exam) and peripheral capillary&#xD;
                  oxygen saturation less than or equal to 94% on room air, or&#xD;
&#xD;
               2. Requiring mechanical ventilation and/or supplemental oxygen, or&#xD;
&#xD;
               3. Radiographic evidence (chest x-ray or computed tomography scan) of 1 of the&#xD;
                  following:&#xD;
&#xD;
                    -  Ground-glass opacities, or&#xD;
&#xD;
                    -  Local or bilateral patchy infiltrates, or&#xD;
&#xD;
                    -  Interstitial pulmonary infiltrates&#xD;
&#xD;
          4. If the participant was intubated, must have been intubated less than 24 hours prior to&#xD;
             randomization&#xD;
&#xD;
             Sex and Contraception Guidelines -&#xD;
&#xD;
          5. Contraceptive use by men or women should have been consistent with local regulations&#xD;
             regarding the methods of contraception for those participating in clinical studies.&#xD;
&#xD;
             Informed Consent -&#xD;
&#xD;
          6. Capable of giving signed informed consent or by a designated representative, which&#xD;
             includes compliance with the requirements and restrictions listed in the informed&#xD;
             consent form and in this protocol&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
        Medical Conditions -&#xD;
&#xD;
          1. Active bacterial pneumonia defined: based on either lobar consolidation on x-ray,&#xD;
             positive sputum cultures, or leukocytosis with a left shift&#xD;
&#xD;
          2. Known active tuberculosis&#xD;
&#xD;
          3. Participants with acquired immune deficiency syndrome&#xD;
&#xD;
          4. It is not in the best interest of the participants to participate, in the opinion of&#xD;
             the treating Investigator.&#xD;
&#xD;
          5. In the opinion of the investigator, progression to death was imminent and inevitable&#xD;
             within the next 24 hours, irrespective of the provision of treatments.&#xD;
&#xD;
          6. Female participants who were pregnant or breastfeeding or expecting to conceive within&#xD;
             the projected duration of the study, starting with the screening visit through 90 days&#xD;
             after the last dose of study intervention&#xD;
&#xD;
             Excluded Prior/Concomitant Therapy -&#xD;
&#xD;
          7. Prior treatment with other agents with actual or possible direct acting&#xD;
             anti-inflammatory activity against SARS-CoV-2 in the past 30 days (for example,&#xD;
             chloroquine, hydroxychloroquine)&#xD;
&#xD;
          8. Treatment with convalescent plasma&#xD;
&#xD;
          9. Treatment with high doses of corticosteroids (greater than 20 milligrams daily,&#xD;
             prednisone equivalent) prior to randomization&#xD;
&#xD;
         10. Treatment with immunomodulators including anti-interleukin (IL)-6, anti-IL-6 receptor&#xD;
             antagonists, or with Janus kinase inhibitors in the past 30 days or plans to receive&#xD;
             during the study period&#xD;
&#xD;
         11. Previous exposure to study intervention or any other agent targeting colony&#xD;
             stimulating factor-1 or CSF-1R or known allergy/sensitivity to study intervention&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>HonorHealth</name>
      <address>
        <city>Scottsdale</city>
        <state>Arizona</state>
        <zip>85258</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Montefiore Medical Center</name>
      <address>
        <city>Bronx</city>
        <state>New York</state>
        <zip>10467</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>June 2022</verification_date>
  <study_first_submitted>May 29, 2020</study_first_submitted>
  <study_first_submitted_qc>June 2, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 4, 2020</study_first_posted>
  <results_first_submitted>June 29, 2022</results_first_submitted>
  <results_first_submitted_qc>June 29, 2022</results_first_submitted_qc>
  <results_first_posted type="Actual">July 22, 2022</results_first_posted>
  <last_update_submitted>June 29, 2022</last_update_submitted>
  <last_update_submitted_qc>June 29, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">July 22, 2022</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>Coronavirus Infections</mesh_term>
    <mesh_term>Cytokine Release Syndrome</mesh_term>
  </condition_browse>
  <provided_document_section>
    <provided_document>
      <document_type>Study Protocol and Statistical Analysis Plan</document_type>
      <document_has_protocol>Yes</document_has_protocol>
      <document_has_icf>No</document_has_icf>
      <document_has_sap>Yes</document_has_sap>
      <document_date>April 23, 2020</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/73/NCT04415073/Prot_SAP_000.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <pre_assignment_details>This study was terminated by the Sponsor given enrollment challenges, partly attributable to the constantly changing novel coronavirus disease (COVID-19) treatment landscape. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</pre_assignment_details>
      <group_list>
        <group group_id="P1">
          <title>Axatilimab (SNDX-6352)</title>
          <description>Axatilimab on Days 1 and 15, intravenous (IV) + standard of care (SOC)</description>
        </group>
        <group group_id="P2">
          <title>Placebo</title>
          <description>Matching placebo on Days 1 and 15, IV + SOC</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
      <group_list>
        <group group_id="B1">
          <title>Axatilimab (SNDX-6352)</title>
          <description>Axatilimab on Days 1 and 15, IV + SOC</description>
        </group>
        <group group_id="B2">
          <title>Placebo</title>
          <description>Matching placebo on Days 1 and 15, IV + SOC</description>
        </group>
        <group group_id="B3">
          <title>Total</title>
          <description>Total of all reporting groups</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="0"/>
            <count group_id="B2" value="0"/>
            <count group_id="B3" value="0"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                </category>
                <category>
                  <title>Male</title>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Ethnicity (NIH/OMB)</title>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Hispanic or Latino</title>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race (NIH/OMB)</title>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>American Indian or Alaska Native</title>
                </category>
                <category>
                  <title>Asian</title>
                </category>
                <category>
                  <title>Native Hawaiian or Other Pacific Islander</title>
                </category>
                <category>
                  <title>Black or African American</title>
                </category>
                <category>
                  <title>White</title>
                </category>
                <category>
                  <title>More than one race</title>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <class_list>
            <class>
              <title>United States</title>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Count Of Participants Alive And Free Of Respiratory Failure At Day 29</title>
        <description>Respiratory failure was defined by need for mechanical ventilation, extracorporeal membrane oxygenation, non-invasive ventilation &gt;6 liters oxygen/minute, or clinical diagnosis of respiratory failure with initiation of none of these measures only when clinical decision-making was driven solely by resource limitation.</description>
        <time_frame>Day 29</time_frame>
        <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
        <group_list>
          <group group_id="O1">
            <title>Axatilimab (SNDX-6352)</title>
            <description>Axatilimab on Days 1 and 15, IV + SOC</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>Matching placebo on Days 1 and 15, IV + SOC</description>
          </group>
        </group_list>
        <measure>
          <title>Count Of Participants Alive And Free Of Respiratory Failure At Day 29</title>
          <description>Respiratory failure was defined by need for mechanical ventilation, extracorporeal membrane oxygenation, non-invasive ventilation &gt;6 liters oxygen/minute, or clinical diagnosis of respiratory failure with initiation of none of these measures only when clinical decision-making was driven solely by resource limitation.</description>
          <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Count Of Participants With Secondary Clinical Improvement Outcomes At Day 29</title>
        <description>Participants achieving a ≥2 category improvement on a 7-point ordinal score relative to the baseline on Day 28 as collected on Day 29 are reported.</description>
        <time_frame>Day 29</time_frame>
        <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
        <group_list>
          <group group_id="O1">
            <title>Axatilimab (SNDX-6352)</title>
            <description>Axatilimab on Days 1 and 15, IV + SOC</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>Matching placebo on Days 1 and 15, IV + SOC</description>
          </group>
        </group_list>
        <measure>
          <title>Count Of Participants With Secondary Clinical Improvement Outcomes At Day 29</title>
          <description>Participants achieving a ≥2 category improvement on a 7-point ordinal score relative to the baseline on Day 28 as collected on Day 29 are reported.</description>
          <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Time To Clinical Improvement</title>
        <description>The time to clinical improvement is defined as a national early warning score of ≤2 maintained for 24 hours.</description>
        <time_frame>Baseline through Day29</time_frame>
        <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
        <group_list>
          <group group_id="O1">
            <title>Axatilimab (SNDX-6352)</title>
            <description>Axatilimab on Days 1 and 15, IV + SOC</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>Matching placebo on Days 1 and 15, IV + SOC</description>
          </group>
        </group_list>
        <measure>
          <title>Time To Clinical Improvement</title>
          <description>The time to clinical improvement is defined as a national early warning score of ≤2 maintained for 24 hours.</description>
          <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline At Day 29 In Oxygenation In Hospitalized Adults With Respiratory Signs And Symptoms Secondary To COVID-19 Treated With Axatilimab</title>
        <description>The change from baseline at Day 29 or hospital discharge or death, if sooner, in the ratio of peripheral hemoglobin oxygen saturation to fraction of inspired oxygen is evaluated.</description>
        <time_frame>Baseline, Day 29</time_frame>
        <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
        <group_list>
          <group group_id="O1">
            <title>Axatilimab (SNDX-6352)</title>
            <description>Axatilimab on Days 1 and 15, IV + SOC</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>Matching placebo on Days 1 and 15, IV + SOC</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline At Day 29 In Oxygenation In Hospitalized Adults With Respiratory Signs And Symptoms Secondary To COVID-19 Treated With Axatilimab</title>
          <description>The change from baseline at Day 29 or hospital discharge or death, if sooner, in the ratio of peripheral hemoglobin oxygen saturation to fraction of inspired oxygen is evaluated.</description>
          <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline At Day 15 In Biomarkers Following Treatment With Axatilimab</title>
        <description>The change from baseline at Day 15 in serum concentrations of interleukin 6 and c-reactive protein or hospital discharge or death is evaluated.</description>
        <time_frame>Baseline, Day 15</time_frame>
        <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
        <group_list>
          <group group_id="O1">
            <title>Axatilimab (SNDX-6352)</title>
            <description>Axatilimab on Days 1 and 15, IV + SOC</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>Matching placebo on Days 1 and 15, IV + SOC</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline At Day 15 In Biomarkers Following Treatment With Axatilimab</title>
          <description>The change from baseline at Day 15 in serum concentrations of interleukin 6 and c-reactive protein or hospital discharge or death is evaluated.</description>
          <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Count Of Participants Experiencing Adverse Events And Serious Adverse Events</title>
        <description>This outcome measure evaluates the safety and tolerability of axatilimab within the same population. A summary of all serious adverse events and other adverse events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.</description>
        <time_frame>Baseline through Day 29</time_frame>
        <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
        <group_list>
          <group group_id="O1">
            <title>Axatilimab (SNDX-6352)</title>
            <description>Axatilimab on Days 1 and 15, IV + SOC</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>Matching placebo on Days 1 and 15, IV + SOC</description>
          </group>
        </group_list>
        <measure>
          <title>Count Of Participants Experiencing Adverse Events And Serious Adverse Events</title>
          <description>This outcome measure evaluates the safety and tolerability of axatilimab within the same population. A summary of all serious adverse events and other adverse events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.</description>
          <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Count Of Participants Requiring Ventilation Support</title>
        <description>Participants who required initiation of mechanical ventilation after study entry are analyzed.</description>
        <time_frame>Baseline through Day 29</time_frame>
        <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
        <group_list>
          <group group_id="O1">
            <title>Axatilimab (SNDX-6352)</title>
            <description>Axatilimab on Days 1 and 15, IV + SOC</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>Matching placebo on Days 1 and 15, IV + SOC</description>
          </group>
        </group_list>
        <measure>
          <title>Count Of Participants Requiring Ventilation Support</title>
          <description>Participants who required initiation of mechanical ventilation after study entry are analyzed.</description>
          <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Count Of Participants Considered Severe Acute Respiratory Syndrome Coronavirus (SARS-CoV-2) Free By Day 15</title>
        <description>This outcome measure evaluates the antiviral effects of axatilimab in hospitalized adults by Day 15 or hospital discharge, whichever was sooner, with recently diagnosed SARS-CoV-2 infection.</description>
        <time_frame>Day 15</time_frame>
        <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
        <group_list>
          <group group_id="O1">
            <title>Axatilimab (SNDX-6352)</title>
            <description>Axatilimab on Days 1 and 15, IV + SOC</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>Matching placebo on Days 1 and 15, IV + SOC</description>
          </group>
        </group_list>
        <measure>
          <title>Count Of Participants Considered Severe Acute Respiratory Syndrome Coronavirus (SARS-CoV-2) Free By Day 15</title>
          <description>This outcome measure evaluates the antiviral effects of axatilimab in hospitalized adults by Day 15 or hospital discharge, whichever was sooner, with recently diagnosed SARS-CoV-2 infection.</description>
          <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Pharmacokinetics: Serum Concentration Of Axatilimab And Anti-drug Antibodies</title>
        <description>The serum concentration of axatilimab and presence of anti-drug antibodies is evaluated.</description>
        <time_frame>Day 29</time_frame>
        <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
        <group_list>
          <group group_id="O1">
            <title>Axatilimab (SNDX-6352)</title>
            <description>Axatilimab on Days 1 and 15, IV + SOC</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>Matching placebo on Days 1 and 15, IV + SOC</description>
          </group>
        </group_list>
        <measure>
          <title>Pharmacokinetics: Serum Concentration Of Axatilimab And Anti-drug Antibodies</title>
          <description>The serum concentration of axatilimab and presence of anti-drug antibodies is evaluated.</description>
          <population>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</time_frame>
      <desc>This study was terminated by the Sponsor. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</desc>
      <group_list>
        <group group_id="E1">
          <title>Axatilimab (SNDX-6352)</title>
          <description>Axatilimab on Days 1 and 15, IV + SOC</description>
        </group>
        <group group_id="E2">
          <title>Placebo</title>
          <description>Matching placebo on Days 1 and 15, IV + SOC</description>
        </group>
      </group_list>
      <serious_events>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="0"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="0"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="0"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="0"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>0</frequency_threshold>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="0"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="0"/>
              </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>This study was terminated by the Sponsor given enrollment challenges, partly attributable to the constantly changing COVID-19 treatment landscape. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.</limitations_and_caveats>
    <point_of_contact>
      <name_or_title>Kate Madigan, MD</name_or_title>
      <organization>Syndax Pharmaceuticals, Inc.</organization>
      <phone>+1-781-419-1400</phone>
      <email>kmadigan@syndax.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/NCT04415073
org study id: SNDX-6352-0505
nct id: NCT04415073
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
This was a randomized, double-blind, placebo-controlled, 29-day study to assess the efficacy
      and safety of axatilimab plus standard of care (SOC), compared with placebo plus SOC, in
      participants with respiratory signs and symptoms secondary to COVID-19.
Axatilimab (SNDX-6352) is a humanized immunoglobin G4 monoclonal antibody with high affinity
      against colony stimulating factor-1 receptor (CSF-1R) under investigation for the prevention
      or treatment of respiratory signs and symptoms secondary to COVID-19.

      This was a Phase 2, randomized, double-blind, placebo-controlled, multicenter study to
      evaluate the efficacy, safety and tolerability of axatilimab as an add-on to SOC therapy in
      hospitalized participants with respiratory signs and symptoms secondary to COVID-19 compared
      to SOC treatment.

      Eligible participants were to be randomized in a 1:1 ratio to 1 of 2 treatment groups, active
      or control. All participants were to receive axatilimab or matching placebo intravenously
      (IV) as an add-on to SOC on Day 1, within 8 hours of randomization and on Day 15.
      Participants were to be followed for at least 28 days (+3 days) after the first dose of study
      intervention (Day 29).

      The primary objective of the study was to assess the proportion of participants alive and
      free of respiratory failure at Day 29.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Double (Participant, Care Provider)
measure: Count Of Participants Alive And Free Of Respiratory Failure At Day 29
time frame: Day 29
description: Respiratory failure was defined by need for mechanical ventilation, extracorporeal membrane oxygenation, non-invasive ventilation >6 liters oxygen/minute, or clinical diagnosis of respiratory failure with initiation of none of these measures only when clinical decision-making was driven solely by resource limitation.
measure: Count Of Participants With Secondary Clinical Improvement Outcomes At Day 29
time frame: Day 29
description: Participants achieving a ≥2 category improvement on a 7-point ordinal score relative to the baseline on Day 28 as collected on Day 29 are reported.
measure: Time To Clinical Improvement
time frame: Baseline through Day29
description: The time to clinical improvement is defined as a national early warning score of ≤2 maintained for 24 hours.
measure: Change From Baseline At Day 29 In Oxygenation In Hospitalized Adults With Respiratory Signs And Symptoms Secondary To COVID-19 Treated With Axatilimab
time frame: Baseline, Day 29
description: The change from baseline at Day 29 or hospital discharge or death, if sooner, in the ratio of peripheral hemoglobin oxygen saturation to fraction of inspired oxygen is evaluated.
measure: Change From Baseline At Day 15 In Biomarkers Following Treatment With Axatilimab
time frame: Baseline, Day 15
description: The change from baseline at Day 15 in serum concentrations of interleukin 6 and c-reactive protein or hospital discharge or death is evaluated.
measure: Count Of Participants Experiencing Adverse Events And Serious Adverse Events
time frame: Baseline through Day 29
description: This outcome measure evaluates the safety and tolerability of axatilimab within the same population. A summary of all serious adverse events and other adverse events (nonserious) regardless of causality is located in the 'Reported Adverse Events' section.
measure: Count Of Participants Requiring Ventilation Support
time frame: Baseline through Day 29
description: Participants who required initiation of mechanical ventilation after study entry are analyzed.
measure: Count Of Participants Considered Severe Acute Respiratory Syndrome Coronavirus (SARS-CoV-2) Free By Day 15
time frame: Day 15
description: This outcome measure evaluates the antiviral effects of axatilimab in hospitalized adults by Day 15 or hospital discharge, whichever was sooner, with recently diagnosed SARS-CoV-2 infection.
measure: Pharmacokinetics: Serum Concentration Of Axatilimab And Anti-drug Antibodies
time frame: Day 29
description: The serum concentration of axatilimab and presence of anti-drug antibodies is evaluated.
arm group label: Axatilimab (SNDX-6352)
arm group type: Active Comparator
description: Axatilimab on Days 1 and 15, IV + SOC
arm group label: Placebo
arm group type: Placebo Comparator
description: Matching placebo on Days 1 and 15, IV + SOC
intervention type: Drug
intervention name: SNDX-6352
description: SNDX-6352
arm group label: Axatilimab (SNDX-6352)
other name: Axatilimab
intervention type: Drug
intervention name: Placebo
description: Placebo comparator
arm group label: Placebo
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
facility: 
facility: 
country: United States
responsible party type: Sponsor
mesh term: Coronavirus Infections
mesh term: Cytokine Release Syndrome
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
limitations and caveats: This study was terminated by the Sponsor given enrollment challenges, partly attributable to the constantly changing COVID-19 treatment landscape. Due to study termination and only 1 participant being enrolled there are concerns regarding participant confidentiality, therefore no data are being reported.
point of contact: 