<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/NCT03836716</url>
  </required_header>
  <id_info>
    <org_study_id>ORARIALS-02</org_study_id>
    <nct_id>NCT03836716</nct_id>
  </id_info>
  <brief_title>Arimoclomol in Amyotropic Lateral Sclerosis - Open Label Extension Trial</brief_title>
  <official_title>Open Label, Non-randomized Extension Trial to Assess Long Term Safety and Efficacy of Arimoclomol in Subjects With Amyotropic Lateral Sclerosis Who Have Completed the ORARIALS-01 Trial</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>ZevraDenmark</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>ZevraDenmark</source>
  <oversight_info>
    <has_dmc>No</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>
      A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of&#xD;
      Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS) who have completed the&#xD;
      ORARIALS-01 trial.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The planned duration of the open-label trial was 152 weeks, but the trial was terminated&#xD;
      early as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy&#xD;
      endpoints. Therefore, the actual mean duration of open-label treatment was approximately 28&#xD;
      weeks (range approximately 2 to 71 weeks).&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    As a consequence of the results of ORARIALS-01 which did not meet any of its efficacy&#xD;
    endpoints. The planned duration was 152 weeks. After termination, the actual mean duration of&#xD;
    treatment was approx. 28 weeks (range approx. 2 to 71 weeks).&#xD;
  </why_stopped>
  <start_date type="Actual">September 19, 2019</start_date>
  <completion_date type="Actual">July 2, 2021</completion_date>
  <primary_completion_date type="Actual">July 2, 2021</primary_completion_date>
  <phase>Phase 3</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <intervention_model_description>Non-randomized open label</intervention_model_description>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Number of Participants With Treatment-emergent Adverse Events (TEAEs) Over the Open-label Treatment Period</measure>
    <time_frame>From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks</time_frame>
    <description>Adverse event (AE) data were collected throughout the trial until early termination. The average duration of exposure was 198.7 days (approximately 28 weeks; standard deviation 99.57 days; minimum 16 days, maximum 494 days). 58 participants (48.3%) were exposed less than 6 months; 55 participants (45.8%) were exposed 6 to less than 12 months; 7 participants (5.8%) were exposed 12 to less than 18 months. No participant was treated for 76 weeks.&#xD;
Participants with on-treatment TEAEs are reported. An on-treatment TEAE is any TEAE in the on-treatment period defined as the time from first dose of IMP until 14 days since the last preceding administration of IMP (either before a temporary IMP interruption with duration &gt;14 days or the last dose at the end of trial). A participant may have several on-treatment periods separated by interruption intervals.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematology (1)</measure>
    <time_frame>Week 76</time_frame>
    <description>Standard hematology parameters. White blood cell differential count for basophils, eosinophils, leukocytes, lymphocytes, monocytes, and neutrophils, and platelet count.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematology (2)</measure>
    <time_frame>Week 76</time_frame>
    <description>Standard hematology parameters. Percentage of leukocytes were determined for basophils, eosinophils, lymphocytes, monocytes, and neutrophils</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Erythrocytes</measure>
    <time_frame>Week 76</time_frame>
    <description>Standard hematology parameter.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematocrit</measure>
    <time_frame>Week 76</time_frame>
    <description>Standard hematology parameter.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hemoglobin</measure>
    <time_frame>Week 76</time_frame>
    <description>Standard hematology parameter.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (1)</measure>
    <time_frame>Week 76</time_frame>
    <description>Standard clinical chemistry parameters. Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Creatine Kinase, Gamma Glutamyl Transferase, and Lactate Dehydrogenase.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (2)</measure>
    <time_frame>Week 76</time_frame>
    <description>Standard clinical chemistry parameters. Calcium, Calcium Corrected, Cholesterol, Glucose, HDL Cholesterol, LDL Cholesterol, Potassium, Sodium, Triglycerides, and Urea Nitrogen.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (3)</measure>
    <time_frame>Week 76</time_frame>
    <description>Standard clinical chemistry parameters. Bilirubin, Creatinine, Direct Bilirubin, and Indirect Bilirubin.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Albumin and Protein</measure>
    <time_frame>Week 76</time_frame>
    <description>Standard clinical chemistry parameters.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Cystatin C</measure>
    <time_frame>Week 76</time_frame>
    <description>Standard clinical chemistry parameter.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean and Change From Baseline in Vital Signs - Blood Pressure</measure>
    <time_frame>Week 76</time_frame>
    <description>Standard vital signs. Systolic and diastolic blood pressure.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean and Change From Baseline in Vital Signs - Pulse Rate</measure>
    <time_frame>Week 76</time_frame>
    <description>Standard vital signs measurement.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean and Change From Baseline in Vital Signs - Respiratory Rate</measure>
    <time_frame>Week 76</time_frame>
    <description>Standard vital signs measurement.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Mean and Change From Baseline in Vital Signs - Temperature</measure>
    <time_frame>Week 76</time_frame>
    <description>Standard vital signs measurement.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of Participants With Potentially Clinically Significant Abnormalities in Clinical Safety Laboratory Tests and Vital Signs Over the Open-label Treatment Period</measure>
    <time_frame>From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks</time_frame>
    <description>Clinical safety laboratory data and vital signs were collected throughout the trial until early termination. The average duration of exposure was 198.7 days (approximately 28 weeks; standard deviation 99.57 days; minimum 16 days, maximum 494 days). 58 participants (48.3%) were exposed less than 6 months; 55 participants (45.8%) were exposed 6 to less than 12 months; 7 participants (5.8%) were exposed 12 to less than 18 months. No patient was treated for 76 weeks.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Columbia-Suicide Severity Rating Scale (C-SSRS) Over the Open-label Treatment Period</measure>
    <time_frame>From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks</time_frame>
    <description>The C-SSRS is a detailed questionnaire assessing both suicidal behavior and suicidal ideation through a series of simple, plain-language questions administered as an interview by a qualified investigator or delegate.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Change in ALS Functional Rating Scale - Revised (ALSFRS-R) From Baseline to Week 76</measure>
    <time_frame>Week 76</time_frame>
    <description>The ALSFRS-R is an ordinal rating scale used to determine subjects' subjective assessment of their capability and independence with 12 functional activities ('speech', 'salivation', 'swallowing', handwriting', 'cutting food and handling utensils', 'dressing and hygiene', 'turning in bed and adjusting bed clothes', 'walking', 'dyspnoea', 'orthopnoea' and 'respiratory insufficiency'). Each activity is rated on a 5-point scale (from 0 [no ability] to 4 [normal]), giving a maximal ALSFRS-R score of 48. A lower score corresponds to a lower capability and independence.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change in Percentage (%) Predicted Slow Vital Capacity (SVC) From Baseline to Week 76 (for Subjects Who Did Not Meet the Survival Endpoint in the ORARIALS-01 Trial)</measure>
    <time_frame>76 weeks</time_frame>
    <description>Slow vital capacity (SVC) measures the volume that can be exhaled from a full inhalation after exhaling to a maximum as slowly as possible. Predicted SVC was derived per European Community of Coal and Steel (ECCS) reference equations:&#xD;
If male: Predicted SVC = 0.061 x height (cm) - 0.028 x age (years) - 4.65&#xD;
If female: Predicted SVC = 0.0466 x height (cm) - 0.024 x age (years) - 3.28</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">120</enrollment>
  <condition>Amyotrophic Lateral Sclerosis</condition>
  <arm_group>
    <arm_group_label>Arimoclomol</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Arimoclomol</intervention_name>
    <description>2 capsules (2 x 124 mg arimoclomol base; equivalent to 2 x 200 mg arimoclomol citrate) taken 3 times daily</description>
    <arm_group_label>Arimoclomol</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Subject is able to comprehend and is willing to provide written informed consent and&#xD;
             is capable and willing to comply with trial procedures, or in the circumstance that&#xD;
             the subject is incompetent, informed consent/assent is provided in accordance with&#xD;
             local regulation and/or procedures&#xD;
&#xD;
          -  Subject has completed the ORARIALS-01 trial (i.e., met one of the surrogate survival&#xD;
             endpoints of tracheostomy or PAV or has completed the 76 weeks randomized treatment&#xD;
             period)&#xD;
&#xD;
          -  Subject completed ORARIALS-01 while on treatment, where on treatment is defined as&#xD;
             having taken the last dose of IMP within 2 weeks of the End of Trial visit (whether at&#xD;
             week 76 or prior)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Known or suspected allergy or intolerance to the IMP (Arimoclomol or constituents)&#xD;
&#xD;
          -  Exposure to any other investigational treatment, advanced therapy medicinal product or&#xD;
             use of any other prohibited concomitant medications&#xD;
&#xD;
          -  Women who are lactating or pregnant, or men or women unwilling to use a highly&#xD;
             effective method of birth control if not surgically sterile (defined as bilateral&#xD;
             tubal ligation, bilateral oophorectomy, or hysterectomy for women; vasectomy for men)&#xD;
             for female participants until 4 weeks after last dose and for male participants until&#xD;
             3 months after last dose. Pre-menopausal women must have a negative pregnancy test&#xD;
             prior to dosing with trial medication.&#xD;
&#xD;
          -  Any of the following medically significant conditions:&#xD;
&#xD;
               1. Clinically significant renal or hepatic disease OR clinical laboratory assessment&#xD;
                  (results ≥ 3 times the upper limit of normal [ULN] for aspartate aminotransferase&#xD;
                  and/or alanine aminotransferase, bilirubin ≥ 2 times the ULN, or creatinine ≥ 1.5&#xD;
                  times the ULN).&#xD;
&#xD;
               2. Any new condition or worsening of existing condition which, in the opinion of the&#xD;
                  investigator, would put the subject at undue risk.&#xD;
&#xD;
          -  Any serious adverse event or moderate/severe adverse event from the ORARIALS-01 trial&#xD;
             which is ongoing at the time of transitioning to ORARIALS-02 and assessed as probably&#xD;
             related to IMP&#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>Michael Benatar, MD PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>University of Miami</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center</name>
      <address>
        <city>Phoenix</city>
        <state>Arizona</state>
        <zip>85013</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UC Irvine Health ALS and Neuromuscular Center</name>
      <address>
        <city>Orange</city>
        <state>California</state>
        <zip>92868</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Miami</name>
      <address>
        <city>Miami</city>
        <state>Florida</state>
        <zip>33136</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital for Special Surgery</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10021</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Providence Brain &amp; Spine Institute</name>
      <address>
        <city>Portland</city>
        <state>Oregon</state>
        <zip>97213</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Pensylvania, Perelman Center for Advanced Medicine - Penn Neuroscience Center</name>
      <address>
        <city>Philadelphia</city>
        <state>Pennsylvania</state>
        <zip>19107</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Virginia Health System</name>
      <address>
        <city>Charlottesville</city>
        <state>Virginia</state>
        <zip>22908</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Catholic University Leuven</name>
      <address>
        <city>Leuven</city>
        <country>Belgium</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>London Health Sciences Centre</name>
      <address>
        <city>London</city>
        <state>Ontario</state>
        <zip>N6A 5A5</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Centre Hospitalier Regional Universitaire (CHRU) Montpellier - Hopital Gui De Chauliac</name>
      <address>
        <city>Montpellier</city>
        <zip>34295</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Groupe Hospitalier Pitie-Salpetriere - Centre d'Investigation Clinique Neurosciences 1422</name>
      <address>
        <city>Paris</city>
        <zip>75013</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Charite - Universitaetsmedizin Berlin - Campus Virchow-Klinikum (CVK) - Ambulanz fuer ALS und andere Motoneuronenerkrankungen</name>
      <address>
        <city>Berlin</city>
        <zip>13353</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Medizinische Hochschule Hannover (MHH) - Klinik fuer Neurologie</name>
      <address>
        <city>Hannover</city>
        <zip>30625</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Universitaetsklinikum Ulm - Klinik fuer Neurologie</name>
      <address>
        <city>Ulm</city>
        <zip>89081</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Instituti Clinica Scientifici Maugeri</name>
      <address>
        <city>Milano</city>
        <zip>20138</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Azienda Ospedaliero Universitaria (AUO) di Torino - Citta'della Salute e della Scienza di Torino</name>
      <address>
        <city>Torino</city>
        <zip>10126</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Medical Center Utrecht</name>
      <address>
        <city>Utrecht</city>
        <zip>3584CX</zip>
        <country>Netherlands</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Centrum Medyczne NeuroProtect</name>
      <address>
        <city>Warsaw</city>
        <zip>01-684</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Citi Clinic</name>
      <address>
        <city>Warsaw</city>
        <zip>02-473</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Universitario Vall d'Hebron ALS Unit. Consultas Externas; Office: 9-10-11</name>
      <address>
        <city>Barcelona</city>
        <zip>08035</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Carlos III - Hospital Universitario La Paz, ALS Unit</name>
      <address>
        <city>Madrid</city>
        <zip>28046</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Umeå University Hospital</name>
      <address>
        <city>Umeå</city>
        <zip>90737</zip>
        <country>Sweden</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Leonard Wolfson Experimental Neurology Centre</name>
      <address>
        <city>London</city>
        <zip>WC1N 3BG</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Belgium</country>
    <country>Canada</country>
    <country>France</country>
    <country>Germany</country>
    <country>Italy</country>
    <country>Netherlands</country>
    <country>Poland</country>
    <country>Spain</country>
    <country>Sweden</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <removed_countries>
    <country>Switzerland</country>
  </removed_countries>
  <verification_date>August 2023</verification_date>
  <study_first_submitted>February 7, 2019</study_first_submitted>
  <study_first_submitted_qc>February 8, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">February 11, 2019</study_first_posted>
  <results_first_submitted>May 15, 2023</results_first_submitted>
  <results_first_submitted_qc>August 22, 2023</results_first_submitted_qc>
  <results_first_posted type="Actual">August 24, 2023</results_first_posted>
  <last_update_submitted>August 22, 2023</last_update_submitted>
  <last_update_submitted_qc>August 22, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">August 24, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Arimoclomol</keyword>
  <keyword>ALS</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>
    <mesh_term>Sclerosis</mesh_term>
  </condition_browse>
  <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>June 8, 2020</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/16/NCT03836716/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>August 30, 2021</document_date>
      <document_url>https://ClinicalTrials.gov/ProvidedDocs/16/NCT03836716/SAP_001.pdf</document_url>
    </provided_document>
  </provided_document_section>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>Arimoclomol (Open-label)</title>
          <description>248 mg arimoclomol base 3 times daily (planned duration 152 weeks; actual duration after early termination of the trial approx. 28 weeks [range 2-71 weeks]).</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="120"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="120"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Adverse Event</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Death</title>
              <participants_list>
                <participants group_id="P1" count="22"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Withdrawal by Subject</title>
              <participants_list>
                <participants group_id="P1" count="8"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Study terminated by sponsor</title>
              <participants_list>
                <participants group_id="P1" count="87"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Arimoclomol (Open-label)</title>
          <description>248 mg arimoclomol base 3 times daily</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="120"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>years</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="57.6" spread="10.30"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex: Female, Male</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Female</title>
                  <measurement_list>
                    <measurement group_id="B1" value="36"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="84"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Ethnicity (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="3"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Not Hispanic or Latino</title>
                  <measurement_list>
                    <measurement group_id="B1" value="79"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="38"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race (NIH/OMB)</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>American Indian or Alaska Native</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Asian</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Native Hawaiian or Other Pacific Islander</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Black or African American</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>White</title>
                  <measurement_list>
                    <measurement group_id="B1" value="80"/>
                  </measurement_list>
                </category>
                <category>
                  <title>More than one race</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Unknown or Not Reported</title>
                  <measurement_list>
                    <measurement group_id="B1" value="39"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>Canada</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Netherlands</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="10"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Sweden</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Belgium</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>United States</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="28"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Poland</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="15"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Italy</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="14"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>United Kingdom</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>France</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="17"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Germany</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="7"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Spain</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="15"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>ALS Functional Rating Scale - Revised (ALSFRS-R)</title>
          <description>The ALSFRS-R is an ordinal rating scale used to determine subjects' subjective assessment of their capability and independence with 12 functional activities ('speech', 'salivation', 'swallowing', handwriting', 'cutting food and handling utensils', 'dressing and hygiene', 'turning in bed and adjusting bed clothes', 'walking', 'dyspnoea', 'orthopnoea' and 'respiratory insufficiency'). Each activity is rated on a 5-point scale (from 0 [no ability] to 4 [normal]), giving a maximal ALSFRS-R score of 48. A lower score corresponds to a lower capability and independence.</description>
          <units>units on a scale</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="26.0" spread="10.38"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Number of Participants With Treatment-emergent Adverse Events (TEAEs) Over the Open-label Treatment Period</title>
        <description>Adverse event (AE) data were collected throughout the trial until early termination. The average duration of exposure was 198.7 days (approximately 28 weeks; standard deviation 99.57 days; minimum 16 days, maximum 494 days). 58 participants (48.3%) were exposed less than 6 months; 55 participants (45.8%) were exposed 6 to less than 12 months; 7 participants (5.8%) were exposed 12 to less than 18 months. No participant was treated for 76 weeks.&#xD;
Participants with on-treatment TEAEs are reported. An on-treatment TEAE is any TEAE in the on-treatment period defined as the time from first dose of IMP until 14 days since the last preceding administration of IMP (either before a temporary IMP interruption with duration &gt;14 days or the last dose at the end of trial). A participant may have several on-treatment periods separated by interruption intervals.</description>
        <time_frame>From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks</time_frame>
        <population>Safety analysis set: All enrolled patients who received at least one dose of arimoclomol.</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With Treatment-emergent Adverse Events (TEAEs) Over the Open-label Treatment Period</title>
          <description>Adverse event (AE) data were collected throughout the trial until early termination. The average duration of exposure was 198.7 days (approximately 28 weeks; standard deviation 99.57 days; minimum 16 days, maximum 494 days). 58 participants (48.3%) were exposed less than 6 months; 55 participants (45.8%) were exposed 6 to less than 12 months; 7 participants (5.8%) were exposed 12 to less than 18 months. No participant was treated for 76 weeks.&#xD;
Participants with on-treatment TEAEs are reported. An on-treatment TEAE is any TEAE in the on-treatment period defined as the time from first dose of IMP until 14 days since the last preceding administration of IMP (either before a temporary IMP interruption with duration &gt;14 days or the last dose at the end of trial). A participant may have several on-treatment periods separated by interruption intervals.</description>
          <population>Safety analysis set: All enrolled patients who received at least one dose of arimoclomol.</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="120"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Treatment-emergent adverse events (TEAEs)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="93"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Mild TEAEs</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="34"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Moderate TEAEs</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="44"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Severe TEAEs</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="15"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Treatment-related TEAEs</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="31"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Probably related TEAEs</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="9"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Possibly related TEAEs</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="22"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Not related TEAEs</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="62"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Serious TEAEs (SAEs)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="21"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Treatment-related serious TEAEs</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>TEAEs leading to IMP withdrawal</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="9"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>TEAEs leading to IMP interruption</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="12"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematology (1)</title>
        <description>Standard hematology parameters. White blood cell differential count for basophils, eosinophils, leukocytes, lymphocytes, monocytes, and neutrophils, and platelet count.</description>
        <time_frame>Week 76</time_frame>
        <population>Data only available for 2 participants at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematology (1)</title>
          <description>Standard hematology parameters. White blood cell differential count for basophils, eosinophils, leukocytes, lymphocytes, monocytes, and neutrophils, and platelet count.</description>
          <population>Data only available for 2 participants at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
          <units>10^9 cells/L</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="2"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Basophils, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.030" spread="0.0000"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Basophils, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.015" spread="0.0071"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Eosinophils, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.150" spread="0.0141"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Eosinophils, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.070" spread="0.0424"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Leukocytes, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="9.120" spread="3.1537"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Leukocytes, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.565" spread="2.4395"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Lymphocytes, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.445" spread="0.4738"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Lymphocytes, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.365" spread="0.3041"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Monocytes, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.460" spread="0.1414"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Monocytes, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.055" spread="0.0212"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Neutrophils, Segmented, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="7.030" spread="3.4648"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Neutrophils, Segmented, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.070" spread="2.8001"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Platelets, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="334.0" spread="114.55"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Platelets, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="62.5" spread="103.94"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematology (2)</title>
        <description>Standard hematology parameters. Percentage of leukocytes were determined for basophils, eosinophils, lymphocytes, monocytes, and neutrophils</description>
        <time_frame>Week 76</time_frame>
        <population>Data only available for 2 participants at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematology (2)</title>
          <description>Standard hematology parameters. Percentage of leukocytes were determined for basophils, eosinophils, lymphocytes, monocytes, and neutrophils</description>
          <population>Data only available for 2 participants at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
          <units>percentage of leukocytes</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="2"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Basophils/Leukocytes, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.40" spread="0.141"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Basophils/Leukocytes, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.05" spread="0.212"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Eosinophils/Leukocytes, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.70" spread="0.424"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Eosinophils/Leukocytes, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.50" spread="0.849"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Lymphocytes/Leukocytes, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="17.85" spread="11.384"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Lymphocytes/Leukocytes, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.95" spread="8.132"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Monocytes/Leukocytes, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5.05" spread="0.212"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Monocytes/Leukocytes, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.20" spread="1.556"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Neutrophils/Leukocytes, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="74.95" spread="12.092"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Neutrophils/Leukocytes, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.70" spread="10.607"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Erythrocytes</title>
        <description>Standard hematology parameter.</description>
        <time_frame>Week 76</time_frame>
        <population>Data only available for 2 participants at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Erythrocytes</title>
          <description>Standard hematology parameter.</description>
          <population>Data only available for 2 participants at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
          <units>10^12 cells/L</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="2"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Erythrocytes, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="3.70" spread="0.000"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Erythrocytes, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.40" spread="0.707"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematocrit</title>
        <description>Standard hematology parameter.</description>
        <time_frame>Week 76</time_frame>
        <population>Data only available for 2 participants at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematocrit</title>
          <description>Standard hematology parameter.</description>
          <population>Data only available for 2 participants at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
          <units>L of cells / L of blood</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="2"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Hematocrit, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.345" spread="0.0071"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Hematocrit, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.025" spread="0.0636"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hemoglobin</title>
        <description>Standard hematology parameter.</description>
        <time_frame>Week 76</time_frame>
        <population>Data only available for 2 participants at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hemoglobin</title>
          <description>Standard hematology parameter.</description>
          <population>Data only available for 2 participants at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
          <units>g/L</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="2"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Hemoglobin, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="115.5" spread="3.54"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Hemoglobin, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-14.0" spread="21.21"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (1)</title>
        <description>Standard clinical chemistry parameters. Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Creatine Kinase, Gamma Glutamyl Transferase, and Lactate Dehydrogenase.</description>
        <time_frame>Week 76</time_frame>
        <population>Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (1)</title>
          <description>Standard clinical chemistry parameters. Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Creatine Kinase, Gamma Glutamyl Transferase, and Lactate Dehydrogenase.</description>
          <population>Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints</population>
          <units>U/L</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Alanine Aminotransferase, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="14.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Alanine Aminotransferase, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-31.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Alkaline Phosphatase, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="97.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Alkaline Phosphatase, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="16.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Aspartate Aminotransferase, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="11.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Aspartate Aminotransferase, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-15.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Creatine Kinase, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="39.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Creatine Kinase, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-118.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Gamma Glutamyl Transferase, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="13.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Gamma Glutamyl Transferase, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-15.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Lactate Dehydrogenase, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="151.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Lactate Dehydrogenase, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-42.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (2)</title>
        <description>Standard clinical chemistry parameters. Calcium, Calcium Corrected, Cholesterol, Glucose, HDL Cholesterol, LDL Cholesterol, Potassium, Sodium, Triglycerides, and Urea Nitrogen.</description>
        <time_frame>Week 76</time_frame>
        <population>Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (2)</title>
          <description>Standard clinical chemistry parameters. Calcium, Calcium Corrected, Cholesterol, Glucose, HDL Cholesterol, LDL Cholesterol, Potassium, Sodium, Triglycerides, and Urea Nitrogen.</description>
          <population>Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
          <units>mmol/L</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Calcium, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2.220" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Calcium, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.350" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Calcium Corrected, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2.270" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Calcium Corrected, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.080" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Cholesterol, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="3.050" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Cholesterol, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-1.060" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Glucose, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="8.50" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Glucose, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.60" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>HDL Cholesterol, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.830" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>HDL Cholesterol, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.770" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>LDL Cholesterol, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.760" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>LDL Cholesterol, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.540" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Potassium, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="3.90" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Potassium, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.50" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Sodium, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="138.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Sodium, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-6.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Triglycerides, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.100" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Triglycerides, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.320" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Urea Nitrogen, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4.60" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Urea Nitrogen, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-0.40" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (3)</title>
        <description>Standard clinical chemistry parameters. Bilirubin, Creatinine, Direct Bilirubin, and Indirect Bilirubin.</description>
        <time_frame>Week 76</time_frame>
        <population>Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (3)</title>
          <description>Standard clinical chemistry parameters. Bilirubin, Creatinine, Direct Bilirubin, and Indirect Bilirubin.</description>
          <population>Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
          <units>micromol/L</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Bilirubin, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Bilirubin, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-7.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Creatinine, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="18.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Creatinine, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-35.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Direct Bilirubin, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Direct Bilirubin, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-1.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Indirect Bilirubin, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="3.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Indirect Bilirubin, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-6.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Albumin and Protein</title>
        <description>Standard clinical chemistry parameters.</description>
        <time_frame>Week 76</time_frame>
        <population>Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Albumin and Protein</title>
          <description>Standard clinical chemistry parameters.</description>
          <population>Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
          <units>g/L</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Albumin, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="38.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Albumin, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-13.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Protein, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="64.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Protein, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-16.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Cystatin C</title>
        <description>Standard clinical chemistry parameter.</description>
        <time_frame>Week 76</time_frame>
        <population>Data only available for 2 participants at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Mean and Change From Baseline in Clinical Safety Laboratory Tests - Cystatin C</title>
          <description>Standard clinical chemistry parameter.</description>
          <population>Data only available for 2 participants at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
          <units>mg/L</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="2"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Cystatin C, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1.310" spread="0.7495"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Cystatin C, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.350" spread="0.6364"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Mean and Change From Baseline in Vital Signs - Blood Pressure</title>
        <description>Standard vital signs. Systolic and diastolic blood pressure.</description>
        <time_frame>Week 76</time_frame>
        <population>Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Mean and Change From Baseline in Vital Signs - Blood Pressure</title>
          <description>Standard vital signs. Systolic and diastolic blood pressure.</description>
          <population>Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
          <units>mmHg</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Systolic Blood Pressure, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="112.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Systolic Blood Pressure, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-10.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Diastolic Blood Pressure, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="76.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Diastolic Blood Pressure, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-1.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Mean and Change From Baseline in Vital Signs - Pulse Rate</title>
        <description>Standard vital signs measurement.</description>
        <time_frame>Week 76</time_frame>
        <population>Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Mean and Change From Baseline in Vital Signs - Pulse Rate</title>
          <description>Standard vital signs measurement.</description>
          <population>Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
          <units>beats/min</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Pulse Rate, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="86.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Pulse Rate, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-3.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Mean and Change From Baseline in Vital Signs - Respiratory Rate</title>
        <description>Standard vital signs measurement.</description>
        <time_frame>Week 76</time_frame>
        <population>Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Mean and Change From Baseline in Vital Signs - Respiratory Rate</title>
          <description>Standard vital signs measurement.</description>
          <population>Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
          <units>breaths/min</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Respiratory Rate, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="12.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Respiratory Rate, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-4.0" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Mean and Change From Baseline in Vital Signs - Temperature</title>
        <description>Standard vital signs measurement.</description>
        <time_frame>Week 76</time_frame>
        <population>Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Mean and Change From Baseline in Vital Signs - Temperature</title>
          <description>Standard vital signs measurement.</description>
          <population>Data only available for 1 participant at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
          <units>Degrees Celsius</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Temperature, Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="36.40" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Temperature, change from baseline to Week 76</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.80" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Number of Participants With Potentially Clinically Significant Abnormalities in Clinical Safety Laboratory Tests and Vital Signs Over the Open-label Treatment Period</title>
        <description>Clinical safety laboratory data and vital signs were collected throughout the trial until early termination. The average duration of exposure was 198.7 days (approximately 28 weeks; standard deviation 99.57 days; minimum 16 days, maximum 494 days). 58 participants (48.3%) were exposed less than 6 months; 55 participants (45.8%) were exposed 6 to less than 12 months; 7 participants (5.8%) were exposed 12 to less than 18 months. No patient was treated for 76 weeks.</description>
        <time_frame>From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks</time_frame>
        <population>Safety analysis set: All enrolled patients that received at least one dose of arimoclomol.</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With Potentially Clinically Significant Abnormalities in Clinical Safety Laboratory Tests and Vital Signs Over the Open-label Treatment Period</title>
          <description>Clinical safety laboratory data and vital signs were collected throughout the trial until early termination. The average duration of exposure was 198.7 days (approximately 28 weeks; standard deviation 99.57 days; minimum 16 days, maximum 494 days). 58 participants (48.3%) were exposed less than 6 months; 55 participants (45.8%) were exposed 6 to less than 12 months; 7 participants (5.8%) were exposed 12 to less than 18 months. No patient was treated for 76 weeks.</description>
          <population>Safety analysis set: All enrolled patients that received at least one dose of arimoclomol.</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="120"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Hemoglobin (g/L) &lt;= 95 (Females) &lt;= 115 (Males)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="10"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Hemoglobin (g/L) &gt;= 165 (Females), &gt;= 185 (Males)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Erythrocytes (10^12/L) &lt;= 3.5 (Females), &lt;= 3.8 (Males)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="16"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Erythrocytes (10^12/L) &gt;= 6.0 (Females), &gt;= 7.0 (Males)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Hematocrit &lt;= 0.32 (Females), &lt;= 0.37 (Males)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="21"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Hematocrit &gt;= 0.5 (Females), &gt;= 0.55 (Males)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Leukocytes (10^9/L) &lt;= 2.8</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Leukocytes (10^9/L) &gt;= 16</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Neutrophils/Leukocytes (%) &lt;= 20</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Neutrophils/Leukocytes (%) &gt;= 85</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Eosinophils/Leukocytes (%) &gt;= 10</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Basophils/Leukocytes (%) &gt;= 10</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Lymphocytes/Leukocytes (%) &lt;= 10</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Lymphocytes/Leukocytes (%) &gt;= 75</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Monocytes/Leukocytes (%) &gt;= 15</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Platelets (10^9/L) &lt;= 75</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Platelets (10^9/L) &gt;= 600</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Aspartate Aminotransferase (U/L) &gt;= 3 x Upper Limit of Normal (ULN)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Alanine Aminotransferase (U/L) &gt;= 3 x ULN</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="7"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Bilirubin (umol/L) &gt;= 34</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Direct Bilirubin (umol/L) &gt;= 12</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Indirect Bilirubin (umol/L) &gt;= 22</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Alkaline Phosphatase (U/L) &gt;= 3 x ULN</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Gamma Glutamyl Transferase (U/L) &gt;= 200</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Creatinine (umol/L) &gt;= 1.5 x ULN</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Urea Nitrogen (mmol/L) &gt;= 11</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="7"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Sodium (mmol/L) &lt;= 125</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Sodium (mmol/L) &gt;= 155</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Potassium (mmol/L) &lt;= 3.0</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Potassium (mmol/L) &gt;= 6.0</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Calcium (mmol/L) &lt;= 1.8</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Calcium (mmol/L) &gt;= 3.0</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Glucose (mmol/L) &lt;= 3.9 (Fasting=No or blank)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="8"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Glucose (mmol/L) &gt;= 11.1 (Fasting=No or blank)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="7"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Glucose (mmol/L) &lt;= 3.5 (Fasting=Yes)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Glucose (mmol/L) &gt;= 7.0 (Fasting=Yes)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="7"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Protein (g/L) &lt;= 45</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Protein (g/L) &gt;= 95</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Albumin (g/L) &lt;= 27</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Cholesterol (mmol/L) &gt;= 7.8 (Fasting=No or blank)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="15"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Cholesterol (mmol/L) &gt;= 6.2 (Fasting=Yes)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="15"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Triglycerides (mmol/L) &gt;= 5.65 (Fasting=No or blank)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Triglycerides (mmol/L) &gt;= 4.2 (Fasting=Yes)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>LDL Cholesterol (mmol/L) &gt;= 5.3 (Fasting=No or blank)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="13"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>LDL Cholesterol (mmol/L) &gt;= 4.9 (Fasting=Yes)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="7"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>HDL Cholesterol (mmol/L) &lt;= 0.8 (Fasting=No or blank)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="11"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>HDL Cholesterol (mmol/L) &lt;= 0.9 (Fasting=Yes)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Creatine Kinase (U/L) &gt;= 400 (Females) &gt;= 750 (Males)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="20"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Lactate Dehydrogenase (U/L) &gt;= 750</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Pulse Rate &lt;50 and decrease from baseline of &gt;= 15 beats/min</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Pulse Rate &gt;120 and increase from baseline of &gt;= 15 beats/min</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Diastolic Blood Pressure &lt;=50 and decrease from baseline of &gt;= 15 mmHg</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Diastolic Blood Pressure &gt;105 and increase from baseline of &gt;= 15 mmHg</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Systolic Blood Pressure &lt;=90 and decrease from baseline of &gt;= 20 mmHg</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Systolic Blood Pressure &gt;180 and increase from baseline of &gt;= 20 mmHg</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Columbia-Suicide Severity Rating Scale (C-SSRS) Over the Open-label Treatment Period</title>
        <description>The C-SSRS is a detailed questionnaire assessing both suicidal behavior and suicidal ideation through a series of simple, plain-language questions administered as an interview by a qualified investigator or delegate.</description>
        <time_frame>From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks</time_frame>
        <population>Safety analysis set: All enrolled patients who received at least one dose of arimoclomol.</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Columbia-Suicide Severity Rating Scale (C-SSRS) Over the Open-label Treatment Period</title>
          <description>The C-SSRS is a detailed questionnaire assessing both suicidal behavior and suicidal ideation through a series of simple, plain-language questions administered as an interview by a qualified investigator or delegate.</description>
          <population>Safety analysis set: All enrolled patients who received at least one dose of arimoclomol.</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="120"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Suicidal ideation (yes on any 1 of the following 5 items)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="16"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Wish to be Dead</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="15"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Non-specific active suicidal thoughts</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="10"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Active Suicidal ideation with any methods (not Plan) without intent to Act</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Active suicidal ideation with some intent, without Specific Plan</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Active Suicidal ideation with specific Plan and Intent</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Suicidal Behavior (yes on any 1 of the following 5 items)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Preparatory acts or behavior</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Aborted Attempt</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Interrupted Attempt</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Actual Attempt (non-fatal)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Completed Suicide</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Suicidal ideation or Behavior (yes to any of the above 10 items)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="16"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Self-injurious Behavior without suicidal intent</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change in ALS Functional Rating Scale - Revised (ALSFRS-R) From Baseline to Week 76</title>
        <description>The ALSFRS-R is an ordinal rating scale used to determine subjects' subjective assessment of their capability and independence with 12 functional activities ('speech', 'salivation', 'swallowing', handwriting', 'cutting food and handling utensils', 'dressing and hygiene', 'turning in bed and adjusting bed clothes', 'walking', 'dyspnoea', 'orthopnoea' and 'respiratory insufficiency'). Each activity is rated on a 5-point scale (from 0 [no ability] to 4 [normal]), giving a maximal ALSFRS-R score of 48. A lower score corresponds to a lower capability and independence.</description>
        <time_frame>Week 76</time_frame>
        <population>Data were not collected at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Change in ALS Functional Rating Scale - Revised (ALSFRS-R) From Baseline to Week 76</title>
          <description>The ALSFRS-R is an ordinal rating scale used to determine subjects' subjective assessment of their capability and independence with 12 functional activities ('speech', 'salivation', 'swallowing', handwriting', 'cutting food and handling utensils', 'dressing and hygiene', 'turning in bed and adjusting bed clothes', 'walking', 'dyspnoea', 'orthopnoea' and 'respiratory insufficiency'). Each activity is rated on a 5-point scale (from 0 [no ability] to 4 [normal]), giving a maximal ALSFRS-R score of 48. A lower score corresponds to a lower capability and independence.</description>
          <population>Data were not collected at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change in Percentage (%) Predicted Slow Vital Capacity (SVC) From Baseline to Week 76 (for Subjects Who Did Not Meet the Survival Endpoint in the ORARIALS-01 Trial)</title>
        <description>Slow vital capacity (SVC) measures the volume that can be exhaled from a full inhalation after exhaling to a maximum as slowly as possible. Predicted SVC was derived per European Community of Coal and Steel (ECCS) reference equations:&#xD;
If male: Predicted SVC = 0.061 x height (cm) - 0.028 x age (years) - 4.65&#xD;
If female: Predicted SVC = 0.0466 x height (cm) - 0.024 x age (years) - 3.28</description>
        <time_frame>76 weeks</time_frame>
        <population>Data only available for 1 patient at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Arimoclomol (Open-label)</title>
            <description>248 mg arimoclomol base 3 times daily</description>
          </group>
        </group_list>
        <measure>
          <title>Change in Percentage (%) Predicted Slow Vital Capacity (SVC) From Baseline to Week 76 (for Subjects Who Did Not Meet the Survival Endpoint in the ORARIALS-01 Trial)</title>
          <description>Slow vital capacity (SVC) measures the volume that can be exhaled from a full inhalation after exhaling to a maximum as slowly as possible. Predicted SVC was derived per European Community of Coal and Steel (ECCS) reference equations:&#xD;
If male: Predicted SVC = 0.061 x height (cm) - 0.028 x age (years) - 4.65&#xD;
If female: Predicted SVC = 0.0466 x height (cm) - 0.024 x age (years) - 3.28</description>
          <population>Data only available for 1 patient at Week 76 since the trial was terminated early by the sponsor as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy endpoints.</population>
          <units>percentage of predicted SVC</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="1"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-21.6" spread="NA">Standard deviation not applicable as N=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Adverse event (AE) data were collected throughout the trial until early termination. The average duration of exposure was 198.7 days (approximately 28 weeks; standard deviation 99.57 days; minimum 16 days, maximum 494 days). 58 participants (48.3%) were exposed less than 6 months; 55 participants (45.8%) were exposed 6 to less than 12 months; 7 participants (5.8%) were exposed 12 to less than 18 months.</time_frame>
      <desc>A treatment-emergent AE (TEAE) is defined as an AE with onset, or a pre-existing AE that worsened in severity, on or after the first dose of IMP. An on-treatment TEAE is any TEAE in the on-treatment period defined as the time from first dose of IMP until 14 days since the last preceding administration of IMP (either before a temporary IMP interruption with duration &gt;14 days or the last dose at the end of trial). A patient may have several on-treatment periods separated by interruption intervals.</desc>
      <group_list>
        <group group_id="E1">
          <title>Arimoclomol (Open-label)</title>
          <description>248 mg arimoclomol base 3 times daily</description>
        </group>
      </group_list>
      <serious_events>
        <default_vocab>MedDRA (21.1)</default_vocab>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="23" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="21" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Cardiac disorders</title>
            <event_list>
              <event>
                <sub_title>Cardiac arrest</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Cardiac failure</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Coronary artery disease</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Ileus</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Pneumonia</sub_title>
                <counts group_id="E1" subjects_affected="7" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Clostridium difficile infection</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Corona virus infection</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Respiratory tract infection</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Rib fracture</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>False positive investigation result</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Renal and urinary disorders</title>
            <event_list>
              <event>
                <sub_title>Acute kidney injury</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Urinary incontinence</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Pneumonia aspiration</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Acute respiratory failure</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Pulmonary embolism</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Chronic obstructive pulmonary disease</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Dyspnoea</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Pneumothorax</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>2</frequency_threshold>
        <default_vocab>MedDRA (21.1)</default_vocab>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="91" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>Anaemia</sub_title>
                <counts group_id="E1" subjects_affected="6" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Leukocytosis</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Cardiac disorders</title>
            <event_list>
              <event>
                <sub_title>Tachycardia</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Constipation</sub_title>
                <counts group_id="E1" subjects_affected="8" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Flatulence</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Gastrooesophageal reflux disease</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Diarrhoea</sub_title>
                <counts group_id="E1" subjects_affected="4" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Nausea</sub_title>
                <counts group_id="E1" subjects_affected="5" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Urinary tract infection</sub_title>
                <counts group_id="E1" subjects_affected="7" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Corona virus infection</sub_title>
                <counts group_id="E1" subjects_affected="6" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Fungal skin infection</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Fall</sub_title>
                <counts group_id="E1" subjects_affected="8" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Head injury</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Cystatin C increased</sub_title>
                <counts group_id="E1" subjects_affected="7" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Alanine aminotransferase increased</sub_title>
                <counts group_id="E1" subjects_affected="4" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Gamma-glutamyltransferase increased</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Type 2 diabetes mellitus</sub_title>
                <counts group_id="E1" subjects_affected="4" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Decreased appetite</sub_title>
                <counts group_id="E1" subjects_affected="4" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Hypokalaemia</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Muscle spasms</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Pain in extremity</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>Anxiety</sub_title>
                <counts group_id="E1" subjects_affected="4" subjects_at_risk="120"/>
              </event>
              <event>
                <sub_title>Depression</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Vascular disorders</title>
            <event_list>
              <event>
                <sub_title>Hypertension</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="120"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.</restrictive_agreement>
    </certain_agreements>
    <limitations_and_caveats>Early termination led to data for a limited number of patients. No conclusions can be drawn from the laboratory results due to data for only a few patients.</limitations_and_caveats>
    <point_of_contact>
      <name_or_title>Medical Affairs</name_or_title>
      <organization>Zevra Denmark A/S</organization>
      <phone>+1-888-289-5607</phone>
      <email>medicalaffairs@zevra.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/NCT03836716
org study id: ORARIALS-02
nct id: NCT03836716
lead sponsor: 
has dmc: No
is fda regulated drug: Yes
is fda regulated device: No
A multicenter, non-randomized, open label trial, to assess long term safety and efficacy of
      Arimoclomol in subjects with Amyotrophic Lateral Sclerosis (ALS) who have completed the
      ORARIALS-01 trial.
The planned duration of the open-label trial was 152 weeks, but the trial was terminated
      early as a consequence of the results of ORARIALS-01 which did not meet any of its efficacy
      endpoints. Therefore, the actual mean duration of open-label treatment was approximately 28
      weeks (range approximately 2 to 71 weeks).
allocation: N/A
intervention model: Single Group Assignment
intervention model description: Non-randomized open label
primary purpose: Treatment
masking: None (Open Label)
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs) Over the Open-label Treatment Period
time frame: From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks
description: Adverse event (AE) data were collected throughout the trial until early termination. The average duration of exposure was 198.7 days (approximately 28 weeks; standard deviation 99.57 days; minimum 16 days, maximum 494 days). 58 participants (48.3%) were exposed less than 6 months; 55 participants (45.8%) were exposed 6 to less than 12 months; 7 participants (5.8%) were exposed 12 to less than 18 months. No participant was treated for 76 weeks.
Participants with on-treatment TEAEs are reported. An on-treatment TEAE is any TEAE in the on-treatment period defined as the time from first dose of IMP until 14 days since the last preceding administration of IMP (either before a temporary IMP interruption with duration >14 days or the last dose at the end of trial). A participant may have several on-treatment periods separated by interruption intervals.
measure: Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematology (1)
time frame: Week 76
description: Standard hematology parameters. White blood cell differential count for basophils, eosinophils, leukocytes, lymphocytes, monocytes, and neutrophils, and platelet count.
measure: Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematology (2)
time frame: Week 76
description: Standard hematology parameters. Percentage of leukocytes were determined for basophils, eosinophils, lymphocytes, monocytes, and neutrophils
measure: Mean and Change From Baseline in Clinical Safety Laboratory Tests - Erythrocytes
time frame: Week 76
description: Standard hematology parameter.
measure: Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hematocrit
time frame: Week 76
description: Standard hematology parameter.
measure: Mean and Change From Baseline in Clinical Safety Laboratory Tests - Hemoglobin
time frame: Week 76
description: Standard hematology parameter.
measure: Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (1)
time frame: Week 76
description: Standard clinical chemistry parameters. Alanine Aminotransferase, Alkaline Phosphatase, Aspartate Aminotransferase, Creatine Kinase, Gamma Glutamyl Transferase, and Lactate Dehydrogenase.
measure: Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (2)
time frame: Week 76
description: Standard clinical chemistry parameters. Calcium, Calcium Corrected, Cholesterol, Glucose, HDL Cholesterol, LDL Cholesterol, Potassium, Sodium, Triglycerides, and Urea Nitrogen.
measure: Mean and Change From Baseline in Clinical Safety Laboratory Tests - Clinical Chemistry (3)
time frame: Week 76
description: Standard clinical chemistry parameters. Bilirubin, Creatinine, Direct Bilirubin, and Indirect Bilirubin.
measure: Mean and Change From Baseline in Clinical Safety Laboratory Tests - Albumin and Protein
time frame: Week 76
description: Standard clinical chemistry parameters.
measure: Mean and Change From Baseline in Clinical Safety Laboratory Tests - Cystatin C
time frame: Week 76
description: Standard clinical chemistry parameter.
measure: Mean and Change From Baseline in Vital Signs - Blood Pressure
time frame: Week 76
description: Standard vital signs. Systolic and diastolic blood pressure.
measure: Mean and Change From Baseline in Vital Signs - Pulse Rate
time frame: Week 76
description: Standard vital signs measurement.
measure: Mean and Change From Baseline in Vital Signs - Respiratory Rate
time frame: Week 76
description: Standard vital signs measurement.
measure: Mean and Change From Baseline in Vital Signs - Temperature
time frame: Week 76
description: Standard vital signs measurement.
measure: Number of Participants With Potentially Clinically Significant Abnormalities in Clinical Safety Laboratory Tests and Vital Signs Over the Open-label Treatment Period
time frame: From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks
description: Clinical safety laboratory data and vital signs were collected throughout the trial until early termination. The average duration of exposure was 198.7 days (approximately 28 weeks; standard deviation 99.57 days; minimum 16 days, maximum 494 days). 58 participants (48.3%) were exposed less than 6 months; 55 participants (45.8%) were exposed 6 to less than 12 months; 7 participants (5.8%) were exposed 12 to less than 18 months. No patient was treated for 76 weeks.
measure: Columbia-Suicide Severity Rating Scale (C-SSRS) Over the Open-label Treatment Period
time frame: From Day 1 in ORARIALS-02 to Early Termination, an average of approximately 28 weeks
description: The C-SSRS is a detailed questionnaire assessing both suicidal behavior and suicidal ideation through a series of simple, plain-language questions administered as an interview by a qualified investigator or delegate.
measure: Change in ALS Functional Rating Scale - Revised (ALSFRS-R) From Baseline to Week 76
time frame: Week 76
description: The ALSFRS-R is an ordinal rating scale used to determine subjects' subjective assessment of their capability and independence with 12 functional activities ('speech', 'salivation', 'swallowing', handwriting', 'cutting food and handling utensils', 'dressing and hygiene', 'turning in bed and adjusting bed clothes', 'walking', 'dyspnoea', 'orthopnoea' and 'respiratory insufficiency'). Each activity is rated on a 5-point scale (from 0 [no ability] to 4 [normal]), giving a maximal ALSFRS-R score of 48. A lower score corresponds to a lower capability and independence.
measure: Change in Percentage (%) Predicted Slow Vital Capacity (SVC) From Baseline to Week 76 (for Subjects Who Did Not Meet the Survival Endpoint in the ORARIALS-01 Trial)
time frame: 76 weeks
description: Slow vital capacity (SVC) measures the volume that can be exhaled from a full inhalation after exhaling to a maximum as slowly as possible. Predicted SVC was derived per European Community of Coal and Steel (ECCS) reference equations:
If male: Predicted SVC = 0.061 x height (cm) - 0.028 x age (years) - 4.65
If female: Predicted SVC = 0.0466 x height (cm) - 0.024 x age (years) - 3.28
arm group label: Arimoclomol
arm group type: Experimental
description: 248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily
intervention type: Drug
intervention name: Arimoclomol
description: 2 capsules (2 x 124 mg arimoclomol base; equivalent to 2 x 200 mg arimoclomol citrate) taken 3 times daily
arm group label: Arimoclomol
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Michael Benatar, MD PhD
role: Principal Investigator
affiliation: University of Miami
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country: Belgium
country: Canada
country: France
country: Germany
country: Italy
country: Netherlands
country: Poland
country: Spain
country: Sweden
country: United Kingdom
country: United States
country: Switzerland
responsible party type: Sponsor
mesh term: Motor Neuron Disease
mesh term: Amyotrophic Lateral Sclerosis
mesh term: Sclerosis
provided document: 
provided document: 
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
limitations and caveats: Early termination led to data for a limited number of patients. No conclusions can be drawn from the laboratory results due to data for only a few patients.
point of contact: 