<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/NCT04436510</url>
  </required_header>
  <id_info>
    <org_study_id>2019P003518B</org_study_id>
    <nct_id>NCT04436510</nct_id>
  </id_info>
  <brief_title>HEALEY ALS Platform Trial - Regimen B Verdiperstat</brief_title>
  <official_title>HEALEY ALS Platform Trial - Regimen B Verdiperstat</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Merit E. Cudkowicz, MD</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Biohaven Pharmaceuticals, Inc.</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>Massachusetts General Hospital</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>
      The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial&#xD;
      evaluating the safety and efficacy of investigational products for the treatment of ALS.&#xD;
&#xD;
      Regimen B will evaluate the safety and efficacy of a single study drug, verdiperstat, in&#xD;
      participants with ALS.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial&#xD;
      evaluating the safety and efficacy of investigational products for the treatment of ALS. This&#xD;
      trial is designed as a perpetual platform trial. This means that there is a single Master&#xD;
      Protocol dictating the conduct of the trial. The HEALEY ALS Platform Trial Master Protocol is&#xD;
      registered as NCT04297683.&#xD;
&#xD;
      Once a participant enrolls into the Master Protocol and meets all eligibility criteria, the&#xD;
      participant will be eligible to be randomized into any currently enrolling regimen. All&#xD;
      participants will have an equal chance of being randomized to any currently enrolling&#xD;
      regimen.&#xD;
&#xD;
      If a participant is randomized to Regimen B - Verdiperstat, the participant will complete a&#xD;
      screening visit to assess additional Regimen B eligibility criteria. Once Regimen B&#xD;
      eligibility criteria are confirmed, participants will complete a baseline assessment and be&#xD;
      randomized in a 3:1 ratio to either active Verdiperstat or matching placebo.&#xD;
&#xD;
      Regimen B will enroll by invitation as participants may not choose to enroll in Regimen B.&#xD;
      Participants must first enroll into the Master Protocol and be eligible to participate in the&#xD;
      Master Protocol before being able to be randomly assigned to Regimen B.&#xD;
&#xD;
      For a list of enrolling sites, please see the HEALEY ALS Platform Trial Master Protocol under&#xD;
      NCT04297683.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">July 28, 2020</start_date>
  <completion_date type="Actual">December 6, 2022</completion_date>
  <primary_completion_date type="Actual">April 13, 2022</primary_completion_date>
  <phase>Phase 2/Phase 3</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>
  </study_design_info>
  <primary_outcome>
    <measure>Disease Progression as Assessed by the ALSFRS-R-Slope</measure>
    <time_frame>Baseline to 24 Weeks</time_frame>
    <description>Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score using a Bayesian repeated measures model that accounts for loss to follow-up due to mortality. Each of 12 questions assessing distinct functional ability is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Mortality Even Rate</measure>
    <time_frame>Baseline to 24 Weeks</time_frame>
    <description>Mortality is defined as death or death equivalent. A participant is determined to meet the criteria of death equivalent if permanent assisted ventilation (PAV) is used for more than 22 hours per day for more than seven days in a row. The rate of mortality was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Respiratory Function</measure>
    <time_frame>Baseline to 24 Weeks</time_frame>
    <description>Change in respiratory function over time as measured by Slow Vital Capacity (SVC).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Muscle Strength</measure>
    <time_frame>Baseline to 24 Weeks</time_frame>
    <description>Change in muscle strength over time as measured isometrically using hand-held dynamometry (HHD).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of Participants That Experienced Death or Death Equivalent</measure>
    <time_frame>24 Weeks</time_frame>
    <description>The number of participants who died or met the criterion for a death equivalent from the date of their baseline visit to the end of the Week 24 visit window (generally 175 days after baseline). The death equivalent criterion is use of permanent assisted ventilation (PAV) for more than 22 hours per day for more than 7 days in a row.</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">167</enrollment>
  <condition>Amyotrophic Lateral Sclerosis</condition>
  <arm_group>
    <arm_group_label>Verdiperstat</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Verdiperstat is administered twice daily p.o. for 24 weeks.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Matching Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Matching placebo is administered twice daily p.o. for 24 weeks.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Matching Placebo</intervention_name>
    <description>Drug: Matching Placebo&#xD;
Administration: Oral&#xD;
Dose: two tablets twice daily</description>
    <arm_group_label>Matching Placebo</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Verdiperstat</intervention_name>
    <description>Drug: Verdiperstat&#xD;
Administration: Oral&#xD;
Dose: 600mg twice daily</description>
    <arm_group_label>Verdiperstat</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  No additional inclusion criteria beyond the inclusion criteria specified in the Master&#xD;
             Protocol (NCT NCT04297683).&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  The following exclusion criteria are in addition to the exclusion criteria specified&#xD;
             in the Master Protocol (NCT NCT04297683).&#xD;
&#xD;
               1. Participants who are taking strong inhibitors of CYP1A2 (i.e., ciprofloxacin,&#xD;
                  enoxacin, fluvoxamine, zafirlukast) for chronic/long-term use defined as more&#xD;
                  than two weeks.&#xD;
&#xD;
               2. Participants who are taking strong inhibitors of CYP3A4 (i.e., conivaptan,&#xD;
                  itraconazole, ketoconazole, posaconazole, troleandomycin, voriconazole,&#xD;
                  clarithromycin, diltiazem, idelalisib, nefazodone, and certain antiviral agents&#xD;
                  [cobicistat, danoprevir, ritonavir, elvitegravir, indinavir, lopinavir,&#xD;
                  paritaprevir, ombitasavir, dasabuvir, saquinavir, tipranavir, nelfinavir]) for&#xD;
                  chronic/long-term use defined as more than two weeks. Note: Topical antifungal&#xD;
                  use is not exclusionary. Participants should not consume large quantities of&#xD;
                  grapefruit juice (more than 8oz per day) on a regular basis.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Merit Cudkowicz, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Massachusetts General Hospital</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Healey Center for ALS at Mass General</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02114</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>May 2023</verification_date>
  <study_first_submitted>June 1, 2020</study_first_submitted>
  <study_first_submitted_qc>June 17, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">June 18, 2020</study_first_posted>
  <results_first_submitted>April 6, 2023</results_first_submitted>
  <results_first_submitted_qc>May 15, 2023</results_first_submitted_qc>
  <results_first_posted type="Actual">June 12, 2023</results_first_posted>
  <last_update_submitted>May 15, 2023</last_update_submitted>
  <last_update_submitted_qc>May 15, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">June 12, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor-Investigator</responsible_party_type>
    <investigator_affiliation>Massachusetts General Hospital</investigator_affiliation>
    <investigator_full_name>Merit E. Cudkowicz, MD</investigator_full_name>
    <investigator_title>Chief, Neurology Department</investigator_title>
  </responsible_party>
  <keyword>ALS</keyword>
  <keyword>Placebo-Controlled</keyword>
  <keyword>Double-Blind</keyword>
  <keyword>Regimen Specific Appendix</keyword>
  <keyword>Lou Gehrig's Disease</keyword>
  <keyword>Verdiperstat</keyword>
  <keyword>Biohaven Pharmaceuticals</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Motor Neuron Disease</mesh_term>
    <mesh_term>Amyotrophic Lateral Sclerosis</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>December 2, 2021</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/10/NCT04436510/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 22, 2022</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/10/NCT04436510/SAP_001.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>Verdiperstat</title>
          <description>Verdiperstat is administered twice daily p.o. for 24 weeks.&#xD;
Verdiperstat: Drug: Verdiperstat&#xD;
Administration: Oral&#xD;
Dose: 600mg twice daily</description>
        </group>
        <group group_id="P2">
          <title>Matching Placebo</title>
          <description>Matching placebo is administered twice daily p.o. for 24 weeks.&#xD;
Matching Placebo: Drug: Matching Placebo&#xD;
Administration: Oral&#xD;
Dose: two tablets twice daily</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="126"/>
                <participants group_id="P2" count="41"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="95"/>
                <participants group_id="P2" count="35"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="31"/>
                <participants group_id="P2" count="6"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <population>Baseline Analysis Population includes only placebo participants from the Regimen B Efficacy Regimen Only (ERO) sample; shared placebo participants from other regimens are not included in the Baseline Analysis Population.</population>
      <group_list>
        <group group_id="B1">
          <title>Verdiperstat</title>
          <description>Verdiperstat is administered twice daily p.o. for 24 weeks.&#xD;
Verdiperstat: Drug: Verdiperstat&#xD;
Administration: Oral&#xD;
Dose: 600mg twice daily</description>
        </group>
        <group group_id="B2">
          <title>Matching Placebo</title>
          <description>Matching placebo is administered twice daily p.o. for 24 weeks.&#xD;
Matching Placebo: Drug: Matching Placebo&#xD;
Administration: Oral&#xD;
Dose: two tablets twice daily</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="126"/>
            <count group_id="B2" value="41"/>
            <count group_id="B3" value="167"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>years</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="126"/>
                    <count group_id="B2" value="41"/>
                    <count group_id="B3" value="167"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="58.1" spread="11.54"/>
                    <measurement group_id="B2" value="57.3" spread="11.07"/>
                    <measurement group_id="B3" value="57.9" spread="11.40"/>
                  </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>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="126"/>
                    <count group_id="B2" value="41"/>
                    <count group_id="B3" value="167"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="47"/>
                    <measurement group_id="B2" value="12"/>
                    <measurement group_id="B3" value="59"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="79"/>
                    <measurement group_id="B2" value="29"/>
                    <measurement group_id="B3" value="108"/>
                  </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>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="126"/>
                    <count group_id="B2" value="41"/>
                    <count group_id="B3" value="167"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <title>Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="8"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="8"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="116"/>
                    <measurement group_id="B2" value="41"/>
                    <measurement group_id="B3" value="157"/>
                  </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="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>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="126"/>
                    <count group_id="B2" value="41"/>
                    <count group_id="B3" value="167"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <title>American Indian or Alaska Native</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="5"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="6"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Native Hawaiian or Other Pacific Islander</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="4"/>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="6"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="116"/>
                    <measurement group_id="B2" value="37"/>
                    <measurement group_id="B3" value="153"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>El Escorial Diagnosis</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="126"/>
                    <count group_id="B2" value="41"/>
                    <count group_id="B3" value="167"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <title>Clinically Definite ALS</title>
                  <measurement_list>
                    <measurement group_id="B1" value="45"/>
                    <measurement group_id="B2" value="18"/>
                    <measurement group_id="B3" value="63"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Clinically Probable ALS</title>
                  <measurement_list>
                    <measurement group_id="B1" value="53"/>
                    <measurement group_id="B2" value="11"/>
                    <measurement group_id="B3" value="64"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Clinically Probable ALS - Laboratory Supported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="20"/>
                    <measurement group_id="B2" value="8"/>
                    <measurement group_id="B3" value="28"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Clinically Possible ALS</title>
                  <measurement_list>
                    <measurement group_id="B1" value="8"/>
                    <measurement group_id="B2" value="4"/>
                    <measurement group_id="B3" value="12"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Time Since Symptom onset at Baseline</title>
          <units>months</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="126"/>
                    <count group_id="B2" value="41"/>
                    <count group_id="B3" value="167"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="21.2" spread="8.93"/>
                    <measurement group_id="B2" value="22.5" spread="7.91"/>
                    <measurement group_id="B3" value="21.5" spread="8.69"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Delay in ALS Symptom Onset and Diagnosis</title>
          <units>months</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="126"/>
                    <count group_id="B2" value="41"/>
                    <count group_id="B3" value="167"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="10.5" spread="5.96"/>
                    <measurement group_id="B2" value="10.5" spread="6.15"/>
                    <measurement group_id="B3" value="10.5" spread="5.99"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>ALS Onset Location</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="126"/>
                    <count group_id="B2" value="41"/>
                    <count group_id="B3" value="167"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <title>Axial</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="3"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Bulbar</title>
                  <measurement_list>
                    <measurement group_id="B1" value="20"/>
                    <measurement group_id="B2" value="7"/>
                    <measurement group_id="B3" value="27"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Generalized</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Limb</title>
                  <measurement_list>
                    <measurement group_id="B1" value="103"/>
                    <measurement group_id="B2" value="32"/>
                    <measurement group_id="B3" value="135"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Respiratory</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Baseline Edaravone Use</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="126"/>
                    <count group_id="B2" value="41"/>
                    <count group_id="B3" value="167"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <title>Yes</title>
                  <measurement_list>
                    <measurement group_id="B1" value="28"/>
                    <measurement group_id="B2" value="11"/>
                    <measurement group_id="B3" value="39"/>
                  </measurement_list>
                </category>
                <category>
                  <title>No</title>
                  <measurement_list>
                    <measurement group_id="B1" value="98"/>
                    <measurement group_id="B2" value="30"/>
                    <measurement group_id="B3" value="128"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Baseline Riluzole Use</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="126"/>
                    <count group_id="B2" value="41"/>
                    <count group_id="B3" value="167"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <title>Yes</title>
                  <measurement_list>
                    <measurement group_id="B1" value="95"/>
                    <measurement group_id="B2" value="31"/>
                    <measurement group_id="B3" value="126"/>
                  </measurement_list>
                </category>
                <category>
                  <title>No</title>
                  <measurement_list>
                    <measurement group_id="B1" value="31"/>
                    <measurement group_id="B2" value="10"/>
                    <measurement group_id="B3" value="41"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>ALSFRS-R Total Score</title>
          <description>The ALS Functional Rating Scare-Revised (ALSFRS-R) measures 12 aspects of physical function. Each of the 12 questions assessing distinct functional ability is scored from4(normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.</description>
          <units>scores on a scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="126"/>
                    <count group_id="B2" value="41"/>
                    <count group_id="B3" value="167"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="34.3" spread="6.45"/>
                    <measurement group_id="B2" value="34.3" spread="6.88"/>
                    <measurement group_id="B3" value="34.3" spread="6.54"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Change in ALSFRS-R prior to Baseline</title>
          <description>The ALS Functional Rating Scare-Revised (ALSFRS-R) measures 12 aspects of physical function. Each of the 12 questions assessing distinct functional ability is scored from4(normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0.&#xD;
Baseline decline in ALSFRS-R was calculated as 48minus the ALSFRS-R total score at Baseline. The difference between 48 and ALSFRS-R total score at Baseline is divided by the number of months between onset of weakness due to ALS and the date of Baseline.&#xD;
A higher number indicates greater decline.</description>
          <units>points per month</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="126"/>
                    <count group_id="B2" value="41"/>
                    <count group_id="B3" value="167"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="0.75" spread="0.499"/>
                    <measurement group_id="B2" value="0.64" spread="0.303"/>
                    <measurement group_id="B3" value="0.72" spread="0.461"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>SVC</title>
          <population>Number analyzed in row differs from overall number due to missing data.</population>
          <units>Percent Predicted</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="121"/>
                    <count group_id="B2" value="39"/>
                    <count group_id="B3" value="160"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="77.2" spread="20.37"/>
                    <measurement group_id="B2" value="74.5" spread="17.50"/>
                    <measurement group_id="B3" value="76.6" spread="19.69"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Kings Stage</title>
          <description>The King's ALS Clinical Staging System is a 4-level ordinal scale with the first three levels indicating the number (1, 2, or 3) of distinct central nervous system regions (bulbar, upper limb, and lower limb) with neuromuscular dysfunction. Level 4a indicates nutritional failure and level 4b indicates respiratory failure. Higher scores indicate a worse disease state.</description>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="126"/>
                    <count group_id="B2" value="41"/>
                    <count group_id="B3" value="167"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <title>1 Region with Neuromuscular Dysfunction</title>
                  <measurement_list>
                    <measurement group_id="B1" value="13"/>
                    <measurement group_id="B2" value="9"/>
                    <measurement group_id="B3" value="22"/>
                  </measurement_list>
                </category>
                <category>
                  <title>2 Regions with Neuromuscular Dysfunction</title>
                  <measurement_list>
                    <measurement group_id="B1" value="39"/>
                    <measurement group_id="B2" value="10"/>
                    <measurement group_id="B3" value="49"/>
                  </measurement_list>
                </category>
                <category>
                  <title>3 Regions with Neuromuscular Dysfunction</title>
                  <measurement_list>
                    <measurement group_id="B1" value="36"/>
                    <measurement group_id="B2" value="14"/>
                    <measurement group_id="B3" value="50"/>
                  </measurement_list>
                </category>
                <category>
                  <title>4a/b Nutritional/Respiratory Failure</title>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                    <measurement group_id="B2" value="0"/>
                    <measurement group_id="B3" value="2"/>
                  </measurement_list>
                </category>
                <category>
                  <title>4b Respiratory Failure</title>
                  <measurement_list>
                    <measurement group_id="B1" value="36"/>
                    <measurement group_id="B2" value="8"/>
                    <measurement group_id="B3" value="44"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Weight</title>
          <units>kg</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="126"/>
                    <count group_id="B2" value="41"/>
                    <count group_id="B3" value="167"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="78.7" spread="17.44"/>
                    <measurement group_id="B2" value="82.4" spread="18.66"/>
                    <measurement group_id="B3" value="79.6" spread="17.76"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Body Mass Index</title>
          <units>kg/m^2</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="126"/>
                    <count group_id="B2" value="41"/>
                    <count group_id="B3" value="167"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="26.4" spread="5.13"/>
                    <measurement group_id="B2" value="27.1" spread="4.89"/>
                    <measurement group_id="B3" value="26.6" spread="5.07"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Serum Creatinine Concentration</title>
          <units>mg/dL</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="126"/>
                    <count group_id="B2" value="41"/>
                    <count group_id="B3" value="167"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="0.7" spread="0.17"/>
                    <measurement group_id="B2" value="0.7" spread="0.17"/>
                    <measurement group_id="B3" value="0.7" spread="0.17"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Neurofilament Light (NfL) Protein in Serum</title>
          <population>Number analyzed in row differs from overall number due to missing data.</population>
          <units>ng/L</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="B1" value="124"/>
                    <count group_id="B2" value="40"/>
                    <count group_id="B3" value="164"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="92.3" spread="75.27"/>
                    <measurement group_id="B2" value="65.8" spread="25.60"/>
                    <measurement group_id="B3" value="85.9" spread="67.55"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Disease Progression as Assessed by the ALSFRS-R-Slope</title>
        <description>Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score using a Bayesian repeated measures model that accounts for loss to follow-up due to mortality. Each of 12 questions assessing distinct functional ability is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.</description>
        <time_frame>Baseline to 24 Weeks</time_frame>
        <population>Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A Zilucoplan (NCT04436497) and Regimen C CNM-Au8 (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.</population>
        <group_list>
          <group group_id="O1">
            <title>Verdiperstat</title>
            <description>Verdiperstat is administered twice daily p.o. for 24 weeks.&#xD;
Verdiperstat: Drug: Verdiperstat&#xD;
Administration: Oral&#xD;
Dose: 600mg twice daily</description>
          </group>
          <group group_id="O2">
            <title>Matching Placebo</title>
            <description>Matching placebo is administered twice daily p.o. for 24 weeks.&#xD;
Matching Placebo: Drug: Matching Placebo&#xD;
Administration: Oral&#xD;
Dose: two tablets twice daily</description>
          </group>
        </group_list>
        <measure>
          <title>Disease Progression as Assessed by the ALSFRS-R-Slope</title>
          <description>Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score using a Bayesian repeated measures model that accounts for loss to follow-up due to mortality. Each of 12 questions assessing distinct functional ability is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.</description>
          <population>Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A Zilucoplan (NCT04436497) and Regimen C CNM-Au8 (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.</population>
          <units>ALSFRS-R total score points per month</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="126"/>
                <count group_id="O2" value="122"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-1.02" spread="0.091"/>
                    <measurement group_id="O2" value="-1.05" spread="0.086"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <param_type>Disease Rate Ratio</param_type>
            <param_value>0.98</param_value>
            <dispersion_type>Standard Deviation</dispersion_type>
            <dispersion_value>0.118</dispersion_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>0.769</ci_lower_limit>
            <ci_upper_limit>1.237</ci_upper_limit>
            <estimate_desc>DDR &lt;1 imply slowing of disease progression by verdiperstat relative to placebo. Note: reported &quot;Confidence Interval&quot; is actually a Bayesian credible interval.</estimate_desc>
            <other_analysis_desc>The model includes covariates for baseline use of edaravone, baseline use of riluzole, months since onset of symptoms, and pre-baseline slope of ALSFRS-R, and random effects for regimen and participant-specific slopes.</other_analysis_desc>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Mortality Even Rate</title>
        <description>Mortality is defined as death or death equivalent. A participant is determined to meet the criteria of death equivalent if permanent assisted ventilation (PAV) is used for more than 22 hours per day for more than seven days in a row. The rate of mortality was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times.</description>
        <time_frame>Baseline to 24 Weeks</time_frame>
        <population>Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A Zilucoplan (NCT04436497) and Regimen C CNM-Au8 (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.</population>
        <group_list>
          <group group_id="O1">
            <title>Verdiperstat</title>
            <description>Verdiperstat is administered twice daily p.o. for 24 weeks.&#xD;
Verdiperstat: Drug: Verdiperstat&#xD;
Administration: Oral&#xD;
Dose: 600mg twice daily</description>
          </group>
          <group group_id="O2">
            <title>Matching Placebo</title>
            <description>Matching placebo is administered twice daily p.o. for 24 weeks.&#xD;
Matching Placebo: Drug: Matching Placebo&#xD;
Administration: Oral&#xD;
Dose: two tablets twice daily</description>
          </group>
        </group_list>
        <measure>
          <title>Mortality Even Rate</title>
          <description>Mortality is defined as death or death equivalent. A participant is determined to meet the criteria of death equivalent if permanent assisted ventilation (PAV) is used for more than 22 hours per day for more than seven days in a row. The rate of mortality was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times.</description>
          <population>Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A Zilucoplan (NCT04436497) and Regimen C CNM-Au8 (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.</population>
          <units>events per month</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="126"/>
                <count group_id="O2" value="122"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.011" spread="0.003"/>
                    <measurement group_id="O2" value="0.012" spread="0.003"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Respiratory Function</title>
        <description>Change in respiratory function over time as measured by Slow Vital Capacity (SVC).</description>
        <time_frame>Baseline to 24 Weeks</time_frame>
        <population>Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A Zilucoplan (NCT04436497) and Regimen C CNM-Au8 (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.</population>
        <group_list>
          <group group_id="O1">
            <title>Verdiperstat</title>
            <description>Verdiperstat is administered twice daily p.o. for 24 weeks.&#xD;
Verdiperstat: Drug: Verdiperstat&#xD;
Administration: Oral&#xD;
Dose: 600mg twice daily</description>
          </group>
          <group group_id="O2">
            <title>Matching Placebo</title>
            <description>Matching placebo is administered twice daily p.o. for 24 weeks.&#xD;
Matching Placebo: Drug: Matching Placebo&#xD;
Administration: Oral&#xD;
Dose: two tablets twice daily</description>
          </group>
        </group_list>
        <measure>
          <title>Respiratory Function</title>
          <description>Change in respiratory function over time as measured by Slow Vital Capacity (SVC).</description>
          <population>Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A Zilucoplan (NCT04436497) and Regimen C CNM-Au8 (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.</population>
          <units>percent change</units>
          <param>Least Squares Mean</param>
          <dispersion>Standard Error</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="126"/>
                <count group_id="O2" value="122"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-7.76" spread="1.519"/>
                    <measurement group_id="O2" value="-8.05" spread="1.418"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.8875</p_value>
            <method>Mixed Models Analysis</method>
            <method_desc>Covariates include baseline use of edaravone and riluzole, months since symptom onset, and pre-baseline ALSFRS-R slope.</method_desc>
            <param_type>Mean Difference (Net)</param_type>
            <param_value>0.29</param_value>
            <dispersion_type>Standard Error of the Mean</dispersion_type>
            <dispersion_value>2.054</dispersion_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>-3.74</ci_lower_limit>
            <ci_upper_limit>4.32</ci_upper_limit>
            <estimate_desc>Verdiperstat 24-week change from baseline relative to placebo 24-week change from baseline.</estimate_desc>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Muscle Strength</title>
        <description>Change in muscle strength over time as measured isometrically using hand-held dynamometry (HHD).</description>
        <time_frame>Baseline to 24 Weeks</time_frame>
        <population>Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A Zilucoplan (NCT04436497) and Regimen C CNM-Au8 (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.</population>
        <group_list>
          <group group_id="O1">
            <title>Verdiperstat</title>
            <description>Verdiperstat is administered twice daily p.o. for 24 weeks.&#xD;
Verdiperstat: Drug: Verdiperstat&#xD;
Administration: Oral&#xD;
Dose: 600mg twice daily</description>
          </group>
          <group group_id="O2">
            <title>Matching Placebo</title>
            <description>Matching placebo is administered twice daily p.o. for 24 weeks.&#xD;
Matching Placebo: Drug: Matching Placebo&#xD;
Administration: Oral&#xD;
Dose: two tablets twice daily</description>
          </group>
        </group_list>
        <measure>
          <title>Muscle Strength</title>
          <description>Change in muscle strength over time as measured isometrically using hand-held dynamometry (HHD).</description>
          <population>Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A Zilucoplan (NCT04436497) and Regimen C CNM-Au8 (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.</population>
          <units>percent change</units>
          <param>Least Squares Mean</param>
          <dispersion>Standard Error</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="126"/>
                <count group_id="O2" value="122"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-23.24" spread="2.863"/>
                    <measurement group_id="O2" value="-26.81" spread="2.682"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.3538</p_value>
            <method>Mixed Models Analysis</method>
            <method_desc>Covariates include baseline use of edaravone and riluzole, months since symptom onset, and pre-baseline ALSFRS-R slope.</method_desc>
            <param_type>Mean Difference (Net)</param_type>
            <param_value>3.57</param_value>
            <dispersion_type>Standard Error of the Mean</dispersion_type>
            <dispersion_value>3.848</dispersion_value>
            <ci_percent>95</ci_percent>
            <ci_n_sides>2-Sided</ci_n_sides>
            <ci_lower_limit>-3.99</ci_lower_limit>
            <ci_upper_limit>11.13</ci_upper_limit>
            <estimate_desc>Verdiperstat 24-week change from baseline relative to placebo 24-week change from baseline.</estimate_desc>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Number of Participants That Experienced Death or Death Equivalent</title>
        <description>The number of participants who died or met the criterion for a death equivalent from the date of their baseline visit to the end of the Week 24 visit window (generally 175 days after baseline). The death equivalent criterion is use of permanent assisted ventilation (PAV) for more than 22 hours per day for more than 7 days in a row.</description>
        <time_frame>24 Weeks</time_frame>
        <population>Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A Zilucoplan (NCT04436497) and Regimen C CNM-Au8 (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.</population>
        <group_list>
          <group group_id="O1">
            <title>Verdiperstat</title>
            <description>Verdiperstat is administered twice daily p.o. for 24 weeks.&#xD;
Verdiperstat: Drug: Verdiperstat&#xD;
Administration: Oral&#xD;
Dose: 600mg twice daily</description>
          </group>
          <group group_id="O2">
            <title>Matching Placebo</title>
            <description>Matching placebo is administered twice daily p.o. for 24 weeks.&#xD;
Matching Placebo: Drug: Matching Placebo&#xD;
Administration: Oral&#xD;
Dose: two tablets twice daily</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants That Experienced Death or Death Equivalent</title>
          <description>The number of participants who died or met the criterion for a death equivalent from the date of their baseline visit to the end of the Week 24 visit window (generally 175 days after baseline). The death equivalent criterion is use of permanent assisted ventilation (PAV) for more than 22 hours per day for more than 7 days in a row.</description>
          <population>Outcome measure data was analyzed using the Full Analysis Set, which included shared placebo from other regimens, as pre-specified in the Regimen Statistical Analysis Plan. Regimen A Zilucoplan (NCT04436497) and Regimen C CNM-Au8 (NCT04414345) contributed placebo participants into the shared placebo cohort used for analysis.</population>
          <units>Participants</units>
          <param>Count of Participants</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="126"/>
                <count group_id="O2" value="122"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="7"/>
                    <measurement group_id="O2" value="5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <non_inferiority_type>Superiority</non_inferiority_type>
            <p_value>0.318</p_value>
            <method>Log Rank</method>
          </analysis>
        </analysis_list>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Up to 35 weeks after participant signed Master Protocol consent.</time_frame>
      <group_list>
        <group group_id="E1">
          <title>Verdiperstat</title>
          <description>Verdiperstat is administered twice daily p.o. for 24 weeks.&#xD;
Verdiperstat: Drug: Verdiperstat&#xD;
Administration: Oral&#xD;
Dose: 600mg twice daily</description>
        </group>
        <group group_id="E2">
          <title>Matching Placebo</title>
          <description>Matching placebo is administered twice daily p.o. for 24 weeks.&#xD;
Matching Placebo: Drug: Matching Placebo&#xD;
Administration: Oral&#xD;
Dose: two tablets twice daily</description>
        </group>
      </group_list>
      <serious_events>
        <default_vocab>MedDRA 23.0</default_vocab>
        <default_assessment>Non-systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="6" subjects_at_risk="126"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="20" subjects_at_risk="126"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>Febrile neutropenia</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Abdominal pain</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Ileus</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Sudden death</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Bacteraemia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Bacterial sepsis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>COVID-19</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>COVID-19 pneumonia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Hip fracture</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Hepatic enzyme abnormal</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Dehydration</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Failure to thrive</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Neoplasms benign, malignant and unspecified (incl cysts and polyps)</title>
            <event_list>
              <event>
                <sub_title>B-cell lymphoma</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Amyotrophic lateral sclerosis</sub_title>
                <counts group_id="E1" events="2" subjects_affected="2" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Aphasia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Cerebral infarction</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Syncope</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="126"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Pulmonary embolism</sub_title>
                <counts group_id="E1" events="3" subjects_affected="3" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Respiratory failure</sub_title>
                <counts group_id="E1" events="3" subjects_affected="2" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Acute respiratory failure</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Vascular disorders</title>
            <event_list>
              <event>
                <sub_title>Deep vein thrombosis</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="126"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <default_vocab>MedDRA 23.0</default_vocab>
        <default_assessment>Non-systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="114" subjects_at_risk="126"/>
                <counts group_id="E2" subjects_affected="37" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Nausea</sub_title>
                <counts group_id="E1" events="40" subjects_affected="35" subjects_at_risk="126"/>
                <counts group_id="E2" events="3" subjects_affected="3" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Constipation</sub_title>
                <counts group_id="E1" events="26" subjects_affected="24" subjects_at_risk="126"/>
                <counts group_id="E2" events="8" subjects_affected="8" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Dysphagia</sub_title>
                <counts group_id="E1" events="13" subjects_affected="12" subjects_at_risk="126"/>
                <counts group_id="E2" events="6" subjects_affected="4" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Salivary hypersecretion</sub_title>
                <counts group_id="E1" events="10" subjects_affected="10" subjects_at_risk="126"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Diarrhoea</sub_title>
                <counts group_id="E1" events="12" subjects_affected="10" subjects_at_risk="126"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Fatigue</sub_title>
                <counts group_id="E1" events="21" subjects_affected="20" subjects_at_risk="126"/>
                <counts group_id="E2" events="14" subjects_affected="11" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Oedema peripheral</sub_title>
                <counts group_id="E1" events="8" subjects_affected="7" subjects_at_risk="126"/>
                <counts group_id="E2" events="4" subjects_affected="4" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Urinary tract infection</sub_title>
                <counts group_id="E1" events="11" subjects_affected="10" subjects_at_risk="126"/>
                <counts group_id="E2" events="4" subjects_affected="4" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Fall</sub_title>
                <counts group_id="E1" events="50" subjects_affected="24" subjects_at_risk="126"/>
                <counts group_id="E2" events="16" subjects_affected="9" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Blood thyroid stimulating hormone increased</sub_title>
                <counts group_id="E1" events="12" subjects_affected="11" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Decreased appetite</sub_title>
                <counts group_id="E1" events="13" subjects_affected="13" subjects_at_risk="126"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Back pain</sub_title>
                <counts group_id="E1" events="9" subjects_affected="7" subjects_at_risk="126"/>
                <counts group_id="E2" events="8" subjects_affected="2" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Arthralgia</sub_title>
                <counts group_id="E1" events="11" subjects_affected="7" subjects_at_risk="126"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Pain in extremity</sub_title>
                <counts group_id="E1" events="4" subjects_affected="4" subjects_at_risk="126"/>
                <counts group_id="E2" events="4" subjects_affected="3" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" events="31" subjects_affected="27" subjects_at_risk="126"/>
                <counts group_id="E2" events="11" subjects_affected="9" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Muscular weakness</sub_title>
                <counts group_id="E1" events="35" subjects_affected="22" subjects_at_risk="126"/>
                <counts group_id="E2" events="18" subjects_affected="12" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Neuromyopathy</sub_title>
                <counts group_id="E1" events="22" subjects_affected="16" subjects_at_risk="126"/>
                <counts group_id="E2" events="9" subjects_affected="8" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Tension headache</sub_title>
                <counts group_id="E1" events="17" subjects_affected="13" subjects_at_risk="126"/>
                <counts group_id="E2" events="7" subjects_affected="3" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Dizziness</sub_title>
                <counts group_id="E1" events="15" subjects_affected="13" subjects_at_risk="126"/>
                <counts group_id="E2" events="3" subjects_affected="3" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Dysarthria</sub_title>
                <counts group_id="E1" events="4" subjects_affected="4" subjects_at_risk="126"/>
                <counts group_id="E2" events="4" subjects_affected="3" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>Insomnia</sub_title>
                <counts group_id="E1" events="28" subjects_affected="25" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Anxiety</sub_title>
                <counts group_id="E1" events="9" subjects_affected="9" subjects_at_risk="126"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Renal and urinary disorders</title>
            <event_list>
              <event>
                <sub_title>Urine odour abnormal</sub_title>
                <counts group_id="E1" events="8" subjects_affected="8" subjects_at_risk="126"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Dyspnoea</sub_title>
                <counts group_id="E1" events="11" subjects_affected="10" subjects_at_risk="126"/>
                <counts group_id="E2" events="2" subjects_affected="2" subjects_at_risk="41"/>
              </event>
              <event>
                <sub_title>Cough</sub_title>
                <counts group_id="E1" events="7" subjects_affected="7" subjects_at_risk="126"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="41"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>All Principal Investigators ARE 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>
    <point_of_contact>
      <name_or_title>Healey Center for ALS Project Management</name_or_title>
      <organization>Healey Center for ALS at Massachusetts General Hospital</organization>
      <phone>833-425-8257 (HALT ALS)</phone>
      <email>healeyalsplatform@mgh.harvard.edu</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/NCT04436510
org study id: 2019P003518B
nct id: NCT04436510
lead sponsor: 
collaborator: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial
      evaluating the safety and efficacy of investigational products for the treatment of ALS.

      Regimen B will evaluate the safety and efficacy of a single study drug, verdiperstat, in
      participants with ALS.
The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial
      evaluating the safety and efficacy of investigational products for the treatment of ALS. This
      trial is designed as a perpetual platform trial. This means that there is a single Master
      Protocol dictating the conduct of the trial. The HEALEY ALS Platform Trial Master Protocol is
      registered as NCT04297683.

      Once a participant enrolls into the Master Protocol and meets all eligibility criteria, the
      participant will be eligible to be randomized into any currently enrolling regimen. All
      participants will have an equal chance of being randomized to any currently enrolling
      regimen.

      If a participant is randomized to Regimen B - Verdiperstat, the participant will complete a
      screening visit to assess additional Regimen B eligibility criteria. Once Regimen B
      eligibility criteria are confirmed, participants will complete a baseline assessment and be
      randomized in a 3:1 ratio to either active Verdiperstat or matching placebo.

      Regimen B will enroll by invitation as participants may not choose to enroll in Regimen B.
      Participants must first enroll into the Master Protocol and be eligible to participate in the
      Master Protocol before being able to be randomly assigned to Regimen B.

      For a list of enrolling sites, please see the HEALEY ALS Platform Trial Master Protocol under
      NCT04297683.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
measure: Disease Progression as Assessed by the ALSFRS-R-Slope
time frame: Baseline to 24 Weeks
description: Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score using a Bayesian repeated measures model that accounts for loss to follow-up due to mortality. Each of 12 questions assessing distinct functional ability is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
measure: Mortality Even Rate
time frame: Baseline to 24 Weeks
description: Mortality is defined as death or death equivalent. A participant is determined to meet the criteria of death equivalent if permanent assisted ventilation (PAV) is used for more than 22 hours per day for more than seven days in a row. The rate of mortality was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times.
measure: Respiratory Function
time frame: Baseline to 24 Weeks
description: Change in respiratory function over time as measured by Slow Vital Capacity (SVC).
measure: Muscle Strength
time frame: Baseline to 24 Weeks
description: Change in muscle strength over time as measured isometrically using hand-held dynamometry (HHD).
measure: Number of Participants That Experienced Death or Death Equivalent
time frame: 24 Weeks
description: The number of participants who died or met the criterion for a death equivalent from the date of their baseline visit to the end of the Week 24 visit window (generally 175 days after baseline). The death equivalent criterion is use of permanent assisted ventilation (PAV) for more than 22 hours per day for more than 7 days in a row.
arm group label: Verdiperstat
arm group type: Experimental
description: Verdiperstat is administered twice daily p.o. for 24 weeks.
arm group label: Matching Placebo
arm group type: Placebo Comparator
description: Matching placebo is administered twice daily p.o. for 24 weeks.
intervention type: Drug
intervention name: Matching Placebo
description: Drug: Matching Placebo
Administration: Oral
Dose: two tablets twice daily
arm group label: Matching Placebo
intervention type: Drug
intervention name: Verdiperstat
description: Drug: Verdiperstat
Administration: Oral
Dose: 600mg twice daily
arm group label: Verdiperstat
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Merit Cudkowicz, MD
role: Principal Investigator
affiliation: Massachusetts General Hospital
facility: 
country: United States
responsible party type: Sponsor-Investigator
investigator affiliation: Massachusetts General Hospital
investigator full name: Merit E. Cudkowicz, MD
investigator title: Chief, Neurology Department
mesh term: Motor Neuron Disease
mesh term: Amyotrophic Lateral Sclerosis
sharing ipd: No
provided document: 
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 