<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/NCT02239224</url>
  </required_header>
  <id_info>
    <org_study_id>ATYR1940-C-002</org_study_id>
    <secondary_id>2014-001753-17</secondary_id>
    <nct_id>NCT02239224</nct_id>
  </id_info>
  <brief_title>Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Participants With Muscular Dystrophy</brief_title>
  <official_title>A Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics (PK), and Biological Activity of ATYR1940 in Adult Patients With Molecularly Defined Genetic Muscular Dystrophies</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>aTyr Pharma, Inc.</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>aTyr Pharma, Inc.</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the&#xD;
      treatment of adult participants with molecularly defined genetic muscular dystrophies&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Study ATYR1940-C-002 is a multi-national, multi-center, double-blind, randomized,&#xD;
      placebo-controlled, ascending dose study designed to evaluate the safety, tolerability, PK,&#xD;
      immunogenicity, and pharmacodynamic effects of ATYR1940 in participants with FSHD. Up to 44&#xD;
      participants are planned to be enrolled at multiple study centers in the United States and&#xD;
      Europe; the actual number of participants enrolled will depend on the number of cohorts&#xD;
      initiated.&#xD;
&#xD;
      Participants will be screened for study eligibility during the Screening period within 3&#xD;
      weeks before Baseline (that is, Day 1, the first day of Study Drug administration). Eligible&#xD;
      participants, based on Screening assessments, will be randomly assigned to treatment with&#xD;
      ATYR1940 or placebo. Participants who are randomized will be considered enrolled in the&#xD;
      study.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">September 4, 2014</start_date>
  <completion_date type="Actual">December 14, 2015</completion_date>
  <primary_completion_date type="Actual">December 14, 2015</primary_completion_date>
  <phase>Phase 1/Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Double (Participant, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Number of Participants With Treatment Emergent Adverse Events (TEAEs)</measure>
    <time_frame>Up to End of Study (up to Week 25)</time_frame>
    <description>TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. An AE was defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with study drug. Worsening of a pre-existing medical condition, (that is, diabetes, migraine headaches, gout) should have been considered an AE if there was either an increase in severity, frequency, or duration of the condition or an association with significantly worse outcomes. Classification of AEs was to be done by the Investigator according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of Participants With Positive Anti-Drug Antibodies (ADA)</measure>
    <time_frame>Baseline up to Week 14</time_frame>
    <description>Titers through Week 14 are summarized. For Cohorts 1 and 2, samples that screened positive but did not confirm on confirmatory assay were not titered.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of Participants With a Positive or Equivocal Jo-1 Antibody (Ab) Test Result</measure>
    <time_frame>Baseline up to Week 14</time_frame>
    <description>Criterion for a positive Jo-1 Ab test result: &gt;10.0 units/milliliter (U/mL), a cut-point associated with anti-synthetase syndrome. Criterion for an equivocal Jo-1 Ab test result: 7.0 to 10.0 U/mL. Participants were required to have a negative Jo-1 Ab test result (defined as &lt;7 U/mL) for inclusion in the study as well as to continue dosing with study drug during the study.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of Participants With a Clinically Significant Laboratory Abnormality</measure>
    <time_frame>Baseline up to Week 14</time_frame>
    <description>Laboratory parameters included hematology (hematocrit, hemoglobin, platelet count, red blood cell count, white blood cell count, neutrophils, lymphocytes, monocytes, eosinophils, basophils); serum chemistries (blood urea nitrogen, creatinine, total bilirubin, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase (GGT), alkaline phosphatase, sodium, total protein, bicarbonate, potassium, calcium, chloride, magnesium, inorganic phosphate, creatine phosphokinase [will be fractionated if elevated], lactic dehydrogenase, erythrocyte sedimentation rate, C-reactive protein, troponin, myoglobin, insulin-like growth factor 1, cholesterol [non-fasting]); Urinalysis (Color, pH, specific gravity, protein, glucose, ketones, blood). Clinically significant laboratory abnormalities were based upon Investigator's discretion. A summary of all Serious AEs and Other AEs (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of Participants With a Physical Examination Abnormality</measure>
    <time_frame>Up to End of Study (up to Week 25)</time_frame>
    <description>Body systems that were evaluated during the physical examination included general appearance, head, eyes, ears, nose, throat (HEENT), cardiovascular system, respiratory system, chest (breasts), gastrointestinal system (abdomen), lymphatic system, musculoskeletal system, skin, psychiatric, and neurologic. Neurologic examination included assessment of mental status, memory, cranial nerves, motor function, and reflexes, and sensory testing. The body systems with at least 1 physical abnormality is presented. One participant could be represented in more than 1 body system category. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of Participants With a Vital Sign-Related Event Resulting in a TEAE</measure>
    <time_frame>Up to End of Study (Up to Week 25)</time_frame>
    <description>The vital sign parameters that were evaluated included heart rate, systolic and diastolic blood pressure, and respiration rate as well as temperature. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of Participants With a Pulmonary Function Event Resulting in a TEAE</measure>
    <time_frame>Up to End of Study (Up to Week 25)</time_frame>
    <description>Pulmonary function testing included measurements of forced vital capacity (FVC) and forced expiratory volume in 1 second (FEV1). A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Percent Change From Baseline in Manual Muscle Testing (MMT) Score at Week 6 and Week 14</measure>
    <time_frame>Baseline, Week 6 and Week 14</time_frame>
    <description>MMT was graded on a scale from 0 (no movement) to 10 (normal movement). Each side of the body and the position in which each muscle was tested were recorded for each participant. The total MMT score were calculated by averaging a converted MMT scores across all tested muscle groups. Decreased motor function was indicated by decreased individual muscle or composite MMT score.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline in Individualized Neuromuscular Quality of Life (INQoL) - Overall QoL at Week 6 and Week 14</measure>
    <time_frame>Baseline, Week 6 and Week 14</time_frame>
    <description>The INQoL is a validated muscle disease-specific measure of quality of life. The self-administered questionnaire consisted of 45 questions with 4 domains measuring the impact of common muscle disease symptoms (weakness, locking [or myotonia], pain, and fatigue); 5 domains measuring the influence of the muscle disease on particular areas of life (activities, independence, relationships, emotions, and body image); and the last domain focused on the positive and negative effects of treatment and was divided into 2 scores. All responses were given in a 7-point Likert scale, with improved QoL indicated by decreased scores. Overall QoL score was calculated from preselected 5 domains (activities, independence, relationships, emotions, and body image) by adding the scores from each domain. In summary, INQoL yielded 11 scores and 1 QoL score. The Overall scoring used a scale of 0-100, with improved QoL indicated by decreased scores.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Maximum Observed Plasma Concentration (Cmax) of ATYR1940</measure>
    <time_frame>Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only)</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Time to Reach Maximum Observed Plasma Concentration (Tmax) of ATYR1940</measure>
    <time_frame>Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only)</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Area Under the Plasma Concentration Time Curve From Time 0 to Time t (AUC 0-t) of ATYR1940</measure>
    <time_frame>Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only)</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Average of Half-life (T1/2) of ATYR1940</measure>
    <time_frame>Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only)</time_frame>
  </secondary_outcome>
  <number_of_arms>4</number_of_arms>
  <enrollment type="Actual">20</enrollment>
  <condition>Facioscapulohumeral Muscular Dystrophy (FSHD)</condition>
  <arm_group>
    <arm_group_label>Cohort 1: ATYR1940 0.3 mg/kg</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants will receive ATYR1940 0.3 milligrams/kilograms (mg/kg) intravenous (IV) infusion once weekly for 4 weeks.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Cohort 2: ATYR1940 1.0 mg/kg</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants will receive ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Cohort 3: ATYR1940 3.0 mg/kg</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants will receive ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>Participants will receive placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3.</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Placebo</intervention_name>
    <description>Concentrate for solution for infusion</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>ATYR1940</intervention_name>
    <description>Concentrate for solution for infusion</description>
    <arm_group_label>Cohort 1: ATYR1940 0.3 mg/kg</arm_group_label>
    <arm_group_label>Cohort 2: ATYR1940 1.0 mg/kg</arm_group_label>
    <arm_group_label>Cohort 3: ATYR1940 3.0 mg/kg</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Participant is a male or female aged 18 to 65 years, inclusive.&#xD;
&#xD;
          -  Participant has an established, genetically-confirmed, diagnosis of FSHD with clinical&#xD;
             findings meeting existing criteria.&#xD;
&#xD;
          -  Participant has provided written informed consent after the nature of the study has&#xD;
             been explained and prior to the performance of any research-related procedures.&#xD;
&#xD;
          -  Participant is, in the Investigator's opinion, willing and able to comply with all&#xD;
             study procedures.&#xD;
&#xD;
          -  Cohorts ≥2 only: Participant has imaging findings meeting defined criteria for muscle&#xD;
             inflammation in at least 1 skeletal muscle.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Participant is currently receiving treatment with an immunomodulatory agent or has a&#xD;
             history of such treatment, including targeted biological therapies (for example,&#xD;
             etanercept, omalizumab) within the 3 months before Baseline; corticosteroids within 4&#xD;
             weeks before Baseline; or high-dose non-steroidal anti-inflammatory agents (NSAIDs)&#xD;
             within 2 weeks before Baseline.&#xD;
&#xD;
          -  Participant is currently receiving curcumin or albuterol or requires such treatment&#xD;
             during study participation.&#xD;
&#xD;
          -  Participant has evidence of an alternative diagnosis other than FSHD, based on prior&#xD;
             muscle biopsy or genetic test findings.&#xD;
&#xD;
          -  Participant has a presumptive diagnosis of FSHD, based on clinical assessment, but&#xD;
             does not yet have genetic confirmation of the diagnosis.&#xD;
&#xD;
          -  Participant has a severe retinopathy.&#xD;
&#xD;
          -  Participant has a history of obstructive or restrictive lung disease (including&#xD;
             interstitial lung disease, pulmonary fibrosis, or asthma), or evidence for&#xD;
             interstitial lung disease on Screening chest radiograph.&#xD;
&#xD;
          -  Participant has a history of anti-synthetase syndrome, prior Jo-1 antibody&#xD;
             (Ab)-positivity, or has a positive or equivocally positive Jo-1 Ab test result during&#xD;
             Screening.&#xD;
&#xD;
          -  Participant has acute or clinically relevant Epstein-Barr virus or cytomegalovirus&#xD;
             infection or re-activation.&#xD;
&#xD;
          -  Participant has a chronic infection such as hepatitis B virus, hepatitis C virus, or&#xD;
             human immunodeficiency virus or a history of tuberculosis.&#xD;
&#xD;
          -  Participant has received a vaccination within 8 weeks before Baseline or vaccination&#xD;
             is planned during study participation.&#xD;
&#xD;
          -  Participant has symptomatic cardiomyopathy or severe cardiac arrhythmia that may, in&#xD;
             the Investigator's opinion, limit the participant's ability to complete the study&#xD;
             protocol.&#xD;
&#xD;
          -  Participant has anemia (as defined for participant's age and gender by local&#xD;
             laboratory range).&#xD;
&#xD;
          -  Participant has gamma-glutamyl transferase (GGT) or serum creatinine levels &gt;2 × the&#xD;
             upper limit of normal (ULN).&#xD;
&#xD;
          -  Participant has abnormal baseline findings, medical condition(s), or laboratory&#xD;
             findings that, in the Investigator's opinion, might jeopardize participant's safety or&#xD;
             decrease the chance of obtaining satisfactory data needed to achieve the objectives of&#xD;
             the study.&#xD;
&#xD;
          -  Participant has evidence of clinically significant cardiovascular, pulmonary, hepatic,&#xD;
             renal, hematological, metabolic, dermatological, or gastrointestinal disease, or has a&#xD;
             condition that requires immediate surgical intervention or other treatment or may not&#xD;
             allow safe participation.&#xD;
&#xD;
          -  Participant has used any investigational product or device (other than a mobility&#xD;
             assistance device) within 30 days before Baseline.&#xD;
&#xD;
          -  Participant has received a product intended to enhance muscle growth within 30 days&#xD;
             before Baseline.&#xD;
&#xD;
          -  Participant underwent muscle biopsy within 30 days before Baseline.&#xD;
&#xD;
          -  Participant initiated treatment with a statin or had a significant adjustment to their&#xD;
             statin regimen within 3 months before Baseline. (Stable, chronic statin use is&#xD;
             permissible.)&#xD;
&#xD;
          -  Participant has received a product that putatively enhances muscle growth (for&#xD;
             example, insulin-like growth factor, growth hormone) or activity (for example,&#xD;
             Coenzyme A) on a chronic basis within 4 weeks before Baseline.&#xD;
&#xD;
          -  Participant is unwilling to abstain from strenuous physical activity for 24 hours&#xD;
             prior to each study center visit.&#xD;
&#xD;
          -  Participant previously received ATYR1940.&#xD;
&#xD;
          -  If female and of childbearing potential (premenopausal and not surgically sterile),&#xD;
             participant has a positive pregnancy test at Screening or is unwilling to use&#xD;
             contraception from the time of Screening through 1 month after the last Study Drug&#xD;
             dose. Acceptable methods of birth control include abstinence, barrier methods,&#xD;
             hormones, or intra-uterine device.&#xD;
&#xD;
          -  If male, participant is unwilling to use a condom plus spermicide during sexual&#xD;
             intercourse from the time of Screening through 1 month after the last Study Drug dose.&#xD;
&#xD;
          -  Cohorts ≥2 only: Participant has a known contraindication for magnetic resonance&#xD;
             imaging (MRI) assessments (for example metal prosthesis or pacemaker) as per local&#xD;
             site MRI protocol&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>65 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Sanjay Shukla, MD</last_name>
    <role>Study Director</role>
    <affiliation>aTyr Pharma</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>aTyr Pharma Investigative Site</name>
      <address>
        <city>Rochester</city>
        <state>New York</state>
        <zip>14642</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>aTyr Pharma Investigative Site</name>
      <address>
        <city>Columbus</city>
        <state>Ohio</state>
        <zip>43210</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>aTyr Pharma Investigative Site</name>
      <address>
        <city>Marseille</city>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>aTyr Pharma Investigative Site</name>
      <address>
        <city>Rome</city>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>aTyr Pharma Investigative Site</name>
      <address>
        <city>Nijmegen</city>
        <country>Netherlands</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>France</country>
    <country>Italy</country>
    <country>Netherlands</country>
    <country>United States</country>
  </location_countries>
  <verification_date>July 2021</verification_date>
  <study_first_submitted>September 7, 2014</study_first_submitted>
  <study_first_submitted_qc>September 11, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 12, 2014</study_first_posted>
  <results_first_submitted>July 19, 2021</results_first_submitted>
  <results_first_submitted_qc>July 19, 2021</results_first_submitted_qc>
  <results_first_posted type="Actual">August 11, 2021</results_first_posted>
  <disposition_first_submitted>January 25, 2017</disposition_first_submitted>
  <disposition_first_submitted_qc>February 27, 2017</disposition_first_submitted_qc>
  <disposition_first_posted type="Actual">March 28, 2017</disposition_first_posted>
  <last_update_submitted>July 19, 2021</last_update_submitted>
  <last_update_submitted_qc>July 19, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">August 11, 2021</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>FSHD</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Muscular Dystrophies</mesh_term>
    <mesh_term>Muscular Dystrophy, Facioscapulohumeral</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
  </patient_data>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>Cohort 1: ATYR1940 0.3 mg/kg</title>
          <description>Participants received ATYR1940 0.3 milligrams/kilograms (mg/kg) intravenous (IV) infusion once weekly for 4 weeks.</description>
        </group>
        <group group_id="P2">
          <title>Cohort 2: ATYR1940 1.0 mg/kg</title>
          <description>Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.</description>
        </group>
        <group group_id="P3">
          <title>Cohort 3: ATYR1940 3.0 mg/kg</title>
          <description>Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.</description>
        </group>
        <group group_id="P4">
          <title>Placebo</title>
          <description>Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3.</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
                <participants group_id="P2" count="6"/>
                <participants group_id="P3" count="6"/>
                <participants group_id="P4" count="5"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>Received at Least One Dose of Study Drug</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
                <participants group_id="P2" count="6"/>
                <participants group_id="P3" count="6"/>
                <participants group_id="P4" count="5"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
                <participants group_id="P2" count="6"/>
                <participants group_id="P3" count="3"/>
                <participants group_id="P4" count="4"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="0"/>
                <participants group_id="P3" count="3"/>
                <participants group_id="P4" count="1"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Administrative</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="0"/>
                <participants group_id="P3" count="3"/>
                <participants group_id="P4" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <population>Intent to treat (ITT) analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.</population>
      <group_list>
        <group group_id="B1">
          <title>Cohort 1: ATYR1940 0.3 mg/kg</title>
          <description>Participants received ATYR1940 0.3 mg/kg IV infusion once weekly for 4 weeks.</description>
        </group>
        <group group_id="B2">
          <title>Cohort 2: ATYR1940 1.0 mg/kg</title>
          <description>Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.</description>
        </group>
        <group group_id="B3">
          <title>Cohort 3: ATYR1940 3.0 mg/kg</title>
          <description>Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.</description>
        </group>
        <group group_id="B4">
          <title>Placebo</title>
          <description>Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3.</description>
        </group>
        <group group_id="B5">
          <title>Total</title>
          <description>Total of all reporting groups</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="3"/>
            <count group_id="B2" value="6"/>
            <count group_id="B3" value="6"/>
            <count group_id="B4" value="5"/>
            <count group_id="B5" value="20"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>years</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="44.3" lower_limit="25" upper_limit="55"/>
                    <measurement group_id="B2" value="44.5" lower_limit="33" upper_limit="52"/>
                    <measurement group_id="B3" value="45.3" lower_limit="25" upper_limit="72"/>
                    <measurement group_id="B4" value="54.0" lower_limit="39" upper_limit="66"/>
                    <measurement group_id="B5" value="49.35" lower_limit="25" upper_limit="72"/>
                  </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="1"/>
                    <measurement group_id="B2" value="4"/>
                    <measurement group_id="B3" value="2"/>
                    <measurement group_id="B4" value="1"/>
                    <measurement group_id="B5" value="8"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="2"/>
                    <measurement group_id="B2" value="2"/>
                    <measurement group_id="B3" value="4"/>
                    <measurement group_id="B4" value="4"/>
                    <measurement group_id="B5" value="12"/>
                  </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)</title>
        <description>TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. An AE was defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with study drug. Worsening of a pre-existing medical condition, (that is, diabetes, migraine headaches, gout) should have been considered an AE if there was either an increase in severity, frequency, or duration of the condition or an association with significantly worse outcomes. Classification of AEs was to be done by the Investigator according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.</description>
        <time_frame>Up to End of Study (up to Week 25)</time_frame>
        <population>ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1: ATYR1940 0.3 mg/kg</title>
            <description>Participants received ATYR1940 0.3 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2: ATYR1940 1.0 mg/kg</title>
            <description>Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3: ATYR1940 3.0 mg/kg</title>
            <description>Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.</description>
          </group>
          <group group_id="O4">
            <title>Placebo</title>
            <description>Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3.</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With Treatment Emergent Adverse Events (TEAEs)</title>
          <description>TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. An AE was defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with study drug. Worsening of a pre-existing medical condition, (that is, diabetes, migraine headaches, gout) should have been considered an AE if there was either an increase in severity, frequency, or duration of the condition or an association with significantly worse outcomes. Classification of AEs was to be done by the Investigator according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.</description>
          <population>ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.</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="3"/>
                <count group_id="O2" value="6"/>
                <count group_id="O3" value="6"/>
                <count group_id="O4" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="3"/>
                    <measurement group_id="O2" value="6"/>
                    <measurement group_id="O3" value="6"/>
                    <measurement group_id="O4" value="5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Number of Participants With Positive Anti-Drug Antibodies (ADA)</title>
        <description>Titers through Week 14 are summarized. For Cohorts 1 and 2, samples that screened positive but did not confirm on confirmatory assay were not titered.</description>
        <time_frame>Baseline up to Week 14</time_frame>
        <population>ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1: ATYR1940 0.3 mg/kg</title>
            <description>Participants received ATYR1940 0.3 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2: ATYR1940 1.0 mg/kg</title>
            <description>Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3: ATYR1940 3.0 mg/kg</title>
            <description>Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.</description>
          </group>
          <group group_id="O4">
            <title>Placebo</title>
            <description>Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3.</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With Positive Anti-Drug Antibodies (ADA)</title>
          <description>Titers through Week 14 are summarized. For Cohorts 1 and 2, samples that screened positive but did not confirm on confirmatory assay were not titered.</description>
          <population>ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.</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="3"/>
                <count group_id="O2" value="6"/>
                <count group_id="O3" value="6"/>
                <count group_id="O4" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                    <measurement group_id="O2" value="1"/>
                    <measurement group_id="O3" value="3"/>
                    <measurement group_id="O4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Number of Participants With a Positive or Equivocal Jo-1 Antibody (Ab) Test Result</title>
        <description>Criterion for a positive Jo-1 Ab test result: &gt;10.0 units/milliliter (U/mL), a cut-point associated with anti-synthetase syndrome. Criterion for an equivocal Jo-1 Ab test result: 7.0 to 10.0 U/mL. Participants were required to have a negative Jo-1 Ab test result (defined as &lt;7 U/mL) for inclusion in the study as well as to continue dosing with study drug during the study.</description>
        <time_frame>Baseline up to Week 14</time_frame>
        <population>ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1: ATYR1940 0.3 mg/kg</title>
            <description>Participants received ATYR1940 0.3 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2: ATYR1940 1.0 mg/kg</title>
            <description>Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3: ATYR1940 3.0 mg/kg</title>
            <description>Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.</description>
          </group>
          <group group_id="O4">
            <title>Placebo</title>
            <description>Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3.</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With a Positive or Equivocal Jo-1 Antibody (Ab) Test Result</title>
          <description>Criterion for a positive Jo-1 Ab test result: &gt;10.0 units/milliliter (U/mL), a cut-point associated with anti-synthetase syndrome. Criterion for an equivocal Jo-1 Ab test result: 7.0 to 10.0 U/mL. Participants were required to have a negative Jo-1 Ab test result (defined as &lt;7 U/mL) for inclusion in the study as well as to continue dosing with study drug during the study.</description>
          <population>ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.</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="3"/>
                <count group_id="O2" value="6"/>
                <count group_id="O3" value="6"/>
                <count group_id="O4" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="0"/>
                    <measurement group_id="O3" value="0"/>
                    <measurement group_id="O4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Number of Participants With a Clinically Significant Laboratory Abnormality</title>
        <description>Laboratory parameters included hematology (hematocrit, hemoglobin, platelet count, red blood cell count, white blood cell count, neutrophils, lymphocytes, monocytes, eosinophils, basophils); serum chemistries (blood urea nitrogen, creatinine, total bilirubin, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase (GGT), alkaline phosphatase, sodium, total protein, bicarbonate, potassium, calcium, chloride, magnesium, inorganic phosphate, creatine phosphokinase [will be fractionated if elevated], lactic dehydrogenase, erythrocyte sedimentation rate, C-reactive protein, troponin, myoglobin, insulin-like growth factor 1, cholesterol [non-fasting]); Urinalysis (Color, pH, specific gravity, protein, glucose, ketones, blood). Clinically significant laboratory abnormalities were based upon Investigator's discretion. A summary of all Serious AEs and Other AEs (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.</description>
        <time_frame>Baseline up to Week 14</time_frame>
        <population>ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1: ATYR1940 0.3 mg/kg</title>
            <description>Participants received ATYR1940 0.3 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2: ATYR1940 1.0 mg/kg</title>
            <description>Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3: ATYR1940 3.0 mg/kg</title>
            <description>Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.</description>
          </group>
          <group group_id="O4">
            <title>Placebo</title>
            <description>Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3.</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With a Clinically Significant Laboratory Abnormality</title>
          <description>Laboratory parameters included hematology (hematocrit, hemoglobin, platelet count, red blood cell count, white blood cell count, neutrophils, lymphocytes, monocytes, eosinophils, basophils); serum chemistries (blood urea nitrogen, creatinine, total bilirubin, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase (GGT), alkaline phosphatase, sodium, total protein, bicarbonate, potassium, calcium, chloride, magnesium, inorganic phosphate, creatine phosphokinase [will be fractionated if elevated], lactic dehydrogenase, erythrocyte sedimentation rate, C-reactive protein, troponin, myoglobin, insulin-like growth factor 1, cholesterol [non-fasting]); Urinalysis (Color, pH, specific gravity, protein, glucose, ketones, blood). Clinically significant laboratory abnormalities were based upon Investigator's discretion. A summary of all Serious AEs and Other AEs (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.</description>
          <population>ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.</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="3"/>
                <count group_id="O2" value="6"/>
                <count group_id="O3" value="6"/>
                <count group_id="O4" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="2"/>
                    <measurement group_id="O3" value="0"/>
                    <measurement group_id="O4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Number of Participants With a Physical Examination Abnormality</title>
        <description>Body systems that were evaluated during the physical examination included general appearance, head, eyes, ears, nose, throat (HEENT), cardiovascular system, respiratory system, chest (breasts), gastrointestinal system (abdomen), lymphatic system, musculoskeletal system, skin, psychiatric, and neurologic. Neurologic examination included assessment of mental status, memory, cranial nerves, motor function, and reflexes, and sensory testing. The body systems with at least 1 physical abnormality is presented. One participant could be represented in more than 1 body system category. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.</description>
        <time_frame>Up to End of Study (up to Week 25)</time_frame>
        <population>ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. Here, 'Overall Number of Participants Analyzed' (N) signifies number of participants evaluable for this outcome measure.</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1: ATYR1940 0.3 mg/kg</title>
            <description>Participants received ATYR1940 0.3 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2: ATYR1940 1.0 mg/kg</title>
            <description>Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3: ATYR1940 3.0 mg/kg</title>
            <description>Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.</description>
          </group>
          <group group_id="O4">
            <title>Placebo</title>
            <description>Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3.</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With a Physical Examination Abnormality</title>
          <description>Body systems that were evaluated during the physical examination included general appearance, head, eyes, ears, nose, throat (HEENT), cardiovascular system, respiratory system, chest (breasts), gastrointestinal system (abdomen), lymphatic system, musculoskeletal system, skin, psychiatric, and neurologic. Neurologic examination included assessment of mental status, memory, cranial nerves, motor function, and reflexes, and sensory testing. The body systems with at least 1 physical abnormality is presented. One participant could be represented in more than 1 body system category. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.</description>
          <population>ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. Here, 'Overall Number of Participants Analyzed' (N) signifies number of participants evaluable for this outcome measure.</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="3"/>
                <count group_id="O2" value="4"/>
                <count group_id="O3" value="3"/>
                <count group_id="O4" value="4"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>General appearance</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="1"/>
                    <measurement group_id="O3" value="0"/>
                    <measurement group_id="O4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>HEENT</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="1"/>
                    <measurement group_id="O3" value="0"/>
                    <measurement group_id="O4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Lymphatic system</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="0"/>
                    <measurement group_id="O3" value="0"/>
                    <measurement group_id="O4" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Musculoskeletal system</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="1"/>
                    <measurement group_id="O3" value="0"/>
                    <measurement group_id="O4" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Respiratory system</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="1"/>
                    <measurement group_id="O3" value="0"/>
                    <measurement group_id="O4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Skin</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="1"/>
                    <measurement group_id="O3" value="1"/>
                    <measurement group_id="O4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Number of Participants With a Vital Sign-Related Event Resulting in a TEAE</title>
        <description>The vital sign parameters that were evaluated included heart rate, systolic and diastolic blood pressure, and respiration rate as well as temperature. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.</description>
        <time_frame>Up to End of Study (Up to Week 25)</time_frame>
        <population>ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1: ATYR1940 0.3 mg/kg</title>
            <description>Participants received ATYR1940 0.3 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2: ATYR1940 1.0 mg/kg</title>
            <description>Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3: ATYR1940 3.0 mg/kg</title>
            <description>Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.</description>
          </group>
          <group group_id="O4">
            <title>Placebo</title>
            <description>Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3.</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With a Vital Sign-Related Event Resulting in a TEAE</title>
          <description>The vital sign parameters that were evaluated included heart rate, systolic and diastolic blood pressure, and respiration rate as well as temperature. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.</description>
          <population>ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.</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="3"/>
                <count group_id="O2" value="6"/>
                <count group_id="O3" value="6"/>
                <count group_id="O4" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="0"/>
                    <measurement group_id="O3" value="1"/>
                    <measurement group_id="O4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Primary</type>
        <title>Number of Participants With a Pulmonary Function Event Resulting in a TEAE</title>
        <description>Pulmonary function testing included measurements of forced vital capacity (FVC) and forced expiratory volume in 1 second (FEV1). A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.</description>
        <time_frame>Up to End of Study (Up to Week 25)</time_frame>
        <population>ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1: ATYR1940 0.3 mg/kg</title>
            <description>Participants received ATYR1940 0.3 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2: ATYR1940 1.0 mg/kg</title>
            <description>Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3: ATYR1940 3.0 mg/kg</title>
            <description>Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.</description>
          </group>
          <group group_id="O4">
            <title>Placebo</title>
            <description>Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3.</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With a Pulmonary Function Event Resulting in a TEAE</title>
          <description>Pulmonary function testing included measurements of forced vital capacity (FVC) and forced expiratory volume in 1 second (FEV1). A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.</description>
          <population>ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.</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="3"/>
                <count group_id="O2" value="6"/>
                <count group_id="O3" value="6"/>
                <count group_id="O4" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="1"/>
                    <measurement group_id="O3" value="0"/>
                    <measurement group_id="O4" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Percent Change From Baseline in Manual Muscle Testing (MMT) Score at Week 6 and Week 14</title>
        <description>MMT was graded on a scale from 0 (no movement) to 10 (normal movement). Each side of the body and the position in which each muscle was tested were recorded for each participant. The total MMT score were calculated by averaging a converted MMT scores across all tested muscle groups. Decreased motor function was indicated by decreased individual muscle or composite MMT score.</description>
        <time_frame>Baseline, Week 6 and Week 14</time_frame>
        <population>ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1: ATYR1940 0.3 mg/kg</title>
            <description>Participants received ATYR1940 0.3 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2: ATYR1940 1.0 mg/kg</title>
            <description>Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3: ATYR1940 3.0 mg/kg</title>
            <description>Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.</description>
          </group>
          <group group_id="O4">
            <title>Placebo</title>
            <description>Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3.</description>
          </group>
        </group_list>
        <measure>
          <title>Percent Change From Baseline in Manual Muscle Testing (MMT) Score at Week 6 and Week 14</title>
          <description>MMT was graded on a scale from 0 (no movement) to 10 (normal movement). Each side of the body and the position in which each muscle was tested were recorded for each participant. The total MMT score were calculated by averaging a converted MMT scores across all tested muscle groups. Decreased motor function was indicated by decreased individual muscle or composite MMT score.</description>
          <population>ITT analysis set included all participants who were randomized and received at least 1 dose (full or partial) of study drug. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints.</population>
          <units>percent change</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="3"/>
                <count group_id="O2" value="6"/>
                <count group_id="O3" value="6"/>
                <count group_id="O4" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Change at Week 6</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="3"/>
                    <count group_id="O2" value="6"/>
                    <count group_id="O3" value="6"/>
                    <count group_id="O4" value="5"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2.83" lower_limit="1.0" upper_limit="4.4"/>
                    <measurement group_id="O2" value="3.07" lower_limit="0.7" upper_limit="5.6"/>
                    <measurement group_id="O3" value="-0.85" lower_limit="-5.1" upper_limit="5.5"/>
                    <measurement group_id="O4" value="1.34" lower_limit="-3.3" upper_limit="7.8"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change at Week 14</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                    <count group_id="O2" value="0"/>
                    <count group_id="O3" value="6"/>
                    <count group_id="O4" value="2"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O3" value="0.70" lower_limit="-5.9" upper_limit="9.2"/>
                    <measurement group_id="O4" value="-1.40" lower_limit="-1.5" upper_limit="-1.3"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline in Individualized Neuromuscular Quality of Life (INQoL) - Overall QoL at Week 6 and Week 14</title>
        <description>The INQoL is a validated muscle disease-specific measure of quality of life. The self-administered questionnaire consisted of 45 questions with 4 domains measuring the impact of common muscle disease symptoms (weakness, locking [or myotonia], pain, and fatigue); 5 domains measuring the influence of the muscle disease on particular areas of life (activities, independence, relationships, emotions, and body image); and the last domain focused on the positive and negative effects of treatment and was divided into 2 scores. All responses were given in a 7-point Likert scale, with improved QoL indicated by decreased scores. Overall QoL score was calculated from preselected 5 domains (activities, independence, relationships, emotions, and body image) by adding the scores from each domain. In summary, INQoL yielded 11 scores and 1 QoL score. The Overall scoring used a scale of 0-100, with improved QoL indicated by decreased scores.</description>
        <time_frame>Baseline, Week 6 and Week 14</time_frame>
        <population>Per-Protocol (PP) analysis set included all participants in the ITT analysis set with no major protocol deviations and ≥1 post-baseline pharmacodynamic (PD) or clinical parameter assessed. Change from baseline included only those participants with both a baseline value and a value for the summarized time period. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1: ATYR1940 0.3 mg/kg</title>
            <description>Participants received ATYR1940 0.3 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2: ATYR1940 1.0 mg/kg</title>
            <description>Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3: ATYR1940 3.0 mg/kg</title>
            <description>Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.</description>
          </group>
          <group group_id="O4">
            <title>Placebo</title>
            <description>Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3.</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline in Individualized Neuromuscular Quality of Life (INQoL) - Overall QoL at Week 6 and Week 14</title>
          <description>The INQoL is a validated muscle disease-specific measure of quality of life. The self-administered questionnaire consisted of 45 questions with 4 domains measuring the impact of common muscle disease symptoms (weakness, locking [or myotonia], pain, and fatigue); 5 domains measuring the influence of the muscle disease on particular areas of life (activities, independence, relationships, emotions, and body image); and the last domain focused on the positive and negative effects of treatment and was divided into 2 scores. All responses were given in a 7-point Likert scale, with improved QoL indicated by decreased scores. Overall QoL score was calculated from preselected 5 domains (activities, independence, relationships, emotions, and body image) by adding the scores from each domain. In summary, INQoL yielded 11 scores and 1 QoL score. The Overall scoring used a scale of 0-100, with improved QoL indicated by decreased scores.</description>
          <population>Per-Protocol (PP) analysis set included all participants in the ITT analysis set with no major protocol deviations and ≥1 post-baseline pharmacodynamic (PD) or clinical parameter assessed. Change from baseline included only those participants with both a baseline value and a value for the summarized time period. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints.</population>
          <units>score on a scale</units>
          <param>Mean</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="3"/>
                <count group_id="O2" value="6"/>
                <count group_id="O3" value="6"/>
                <count group_id="O4" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Baseline</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="3"/>
                    <count group_id="O2" value="6"/>
                    <count group_id="O3" value="6"/>
                    <count group_id="O4" value="5"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="54.37" lower_limit="36.8" upper_limit="68.4"/>
                    <measurement group_id="O2" value="72.80" lower_limit="63.2" upper_limit="84.2"/>
                    <measurement group_id="O3" value="69.30" lower_limit="52.6" upper_limit="78.9"/>
                    <measurement group_id="O4" value="53.68" lower_limit="36.8" upper_limit="73.7"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change at Week 6</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="3"/>
                    <count group_id="O2" value="6"/>
                    <count group_id="O3" value="6"/>
                    <count group_id="O4" value="5"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2.77" lower_limit="-5.0" upper_limit="6.7"/>
                    <measurement group_id="O2" value="-2.98" lower_limit="-16.1" upper_limit="8.4"/>
                    <measurement group_id="O3" value="-3.78" lower_limit="-16.6" upper_limit="16.7"/>
                    <measurement group_id="O4" value="4.12" lower_limit="-8.3" upper_limit="22.2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Change at Week 14</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                    <count group_id="O2" value="0"/>
                    <count group_id="O3" value="6"/>
                    <count group_id="O4" value="2"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O3" value="-9.90" lower_limit="-19.4" upper_limit="5.0"/>
                    <measurement group_id="O4" value="15.55" lower_limit="3.9" upper_limit="27.2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Maximum Observed Plasma Concentration (Cmax) of ATYR1940</title>
        <time_frame>Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only)</time_frame>
        <population>Pharmacokinetic (PK) analysis set included all participants who had baseline samples and sufficient samples collected post-baseline to permit analysis of PK parameters. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1: ATYR1940 0.3 mg/kg</title>
            <description>Participants received ATYR1940 0.3 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2: ATYR1940 1.0 mg/kg</title>
            <description>Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3: ATYR1940 3.0 mg/kg</title>
            <description>Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Maximum Observed Plasma Concentration (Cmax) of ATYR1940</title>
          <population>Pharmacokinetic (PK) analysis set included all participants who had baseline samples and sufficient samples collected post-baseline to permit analysis of PK parameters. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints.</population>
          <units>nanogram/milliliter</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="3"/>
                <count group_id="O2" value="6"/>
                <count group_id="O3" value="6"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Week 2</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="3"/>
                    <count group_id="O2" value="6"/>
                    <count group_id="O3" value="6"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1360" spread="267"/>
                    <measurement group_id="O2" value="6800" spread="752"/>
                    <measurement group_id="O3" value="21900" spread="6960"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 5</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="3"/>
                    <count group_id="O2" value="5"/>
                    <count group_id="O3" value="6"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1980" spread="249"/>
                    <measurement group_id="O2" value="7490" spread="1040"/>
                    <measurement group_id="O3" value="23800" spread="8780"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 13</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                    <count group_id="O2" value="0"/>
                    <count group_id="O3" value="5"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O3" value="26200" spread="14200"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Time to Reach Maximum Observed Plasma Concentration (Tmax) of ATYR1940</title>
        <time_frame>Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only)</time_frame>
        <population>PK analysis set included all participants who had baseline samples and sufficient samples collected post-baseline to permit analysis of PK parameters. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1: ATYR1940 0.3 mg/kg</title>
            <description>Participants received ATYR1940 0.3 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2: ATYR1940 1.0 mg/kg</title>
            <description>Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3: ATYR1940 3.0 mg/kg</title>
            <description>Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Time to Reach Maximum Observed Plasma Concentration (Tmax) of ATYR1940</title>
          <population>PK analysis set included all participants who had baseline samples and sufficient samples collected post-baseline to permit analysis of PK parameters. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints.</population>
          <units>hours</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="3"/>
                <count group_id="O2" value="6"/>
                <count group_id="O3" value="6"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Week 2</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="3"/>
                    <count group_id="O2" value="6"/>
                    <count group_id="O3" value="6"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.417" spread="0.144"/>
                    <measurement group_id="O2" value="0.500" spread="0"/>
                    <measurement group_id="O3" value="0.500" spread="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 5</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="3"/>
                    <count group_id="O2" value="5"/>
                    <count group_id="O3" value="6"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.500" spread="0"/>
                    <measurement group_id="O2" value="0.500" spread="0"/>
                    <measurement group_id="O3" value="0.500" spread="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 13</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                    <count group_id="O2" value="0"/>
                    <count group_id="O3" value="5"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O3" value="0.500" spread="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Area Under the Plasma Concentration Time Curve From Time 0 to Time t (AUC 0-t) of ATYR1940</title>
        <time_frame>Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only)</time_frame>
        <population>PK analysis set included all participants who had baseline samples and sufficient samples collected post-baseline to permit analysis of PK parameters. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1: ATYR1940 0.3 mg/kg</title>
            <description>Participants received ATYR1940 0.3 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2: ATYR1940 1.0 mg/kg</title>
            <description>Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3: ATYR1940 3.0 mg/kg</title>
            <description>Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Area Under the Plasma Concentration Time Curve From Time 0 to Time t (AUC 0-t) of ATYR1940</title>
          <population>PK analysis set included all participants who had baseline samples and sufficient samples collected post-baseline to permit analysis of PK parameters. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints.</population>
          <units>nanogram*hour/milliliter</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="3"/>
                <count group_id="O2" value="6"/>
                <count group_id="O3" value="6"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Week 2</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="3"/>
                    <count group_id="O2" value="6"/>
                    <count group_id="O3" value="6"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5800" spread="1480"/>
                    <measurement group_id="O2" value="24200" spread="1490"/>
                    <measurement group_id="O3" value="82200" spread="25500"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 5</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="3"/>
                    <count group_id="O2" value="5"/>
                    <count group_id="O3" value="6"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="8550" spread="1350"/>
                    <measurement group_id="O2" value="26500" spread="2650"/>
                    <measurement group_id="O3" value="83700" spread="25800"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 13</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                    <count group_id="O2" value="0"/>
                    <count group_id="O3" value="5"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O3" value="84400" spread="36200"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Average of Half-life (T1/2) of ATYR1940</title>
        <time_frame>Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only)</time_frame>
        <population>PK analysis set included all participants who had baseline samples and sufficient samples collected post-baseline to permit analysis of PK parameters. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints.</population>
        <group_list>
          <group group_id="O1">
            <title>Cohort 1: ATYR1940 0.3 mg/kg</title>
            <description>Participants received ATYR1940 0.3 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O2">
            <title>Cohort 2: ATYR1940 1.0 mg/kg</title>
            <description>Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.</description>
          </group>
          <group group_id="O3">
            <title>Cohort 3: ATYR1940 3.0 mg/kg</title>
            <description>Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.</description>
          </group>
        </group_list>
        <measure>
          <title>Average of Half-life (T1/2) of ATYR1940</title>
          <population>PK analysis set included all participants who had baseline samples and sufficient samples collected post-baseline to permit analysis of PK parameters. Here, 'Number Analyzed' signifies participants evaluable at specified timepoints.</population>
          <units>hours</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="3"/>
                <count group_id="O2" value="6"/>
                <count group_id="O3" value="6"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Week 2</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="3"/>
                    <count group_id="O2" value="6"/>
                    <count group_id="O3" value="6"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="3.91" spread="0.193"/>
                    <measurement group_id="O2" value="3.68" spread="0.354"/>
                    <measurement group_id="O3" value="4.33" spread="0.772"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 5</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="3"/>
                    <count group_id="O2" value="5"/>
                    <count group_id="O3" value="6"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4.16" spread="1.04"/>
                    <measurement group_id="O2" value="3.91" spread="1.29"/>
                    <measurement group_id="O3" value="4.32" spread="0.823"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Week 13</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="0"/>
                    <count group_id="O2" value="0"/>
                    <count group_id="O3" value="5"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O3" value="5.09" spread="2.64"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Up to End of Study (up to Week 25)</time_frame>
      <desc>ITT Analysis Set included all participants who were randomized and received at least 1 dose (full or partial) of study drug.</desc>
      <group_list>
        <group group_id="E1">
          <title>Cohort 1: ATYR1940 0.3 mg/kg</title>
          <description>Participants received ATYR1940 0.3 mg/kg IV infusion once weekly for 4 weeks.</description>
        </group>
        <group group_id="E2">
          <title>Cohort 2: ATYR1940 1.0 mg/kg</title>
          <description>Participants received ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.</description>
        </group>
        <group group_id="E3">
          <title>Cohort 3: ATYR1940 3.0 mg/kg</title>
          <description>Participants received ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.</description>
        </group>
        <group group_id="E4">
          <title>Placebo</title>
          <description>Participants received placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3.</description>
        </group>
      </group_list>
      <serious_events>
        <default_vocab>MedDRA 18.0</default_vocab>
        <default_assessment>Non-systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Infusion-related Reaction</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>0</frequency_threshold>
        <default_vocab>MedDRA 18.0</default_vocab>
        <default_assessment>Non-systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="6" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="6" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="5" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Eye disorders</title>
            <event_list>
              <event>
                <sub_title>Eyelid Irritation</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Scintillating Scotoma</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Abdominal Pain Upper</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Diarrhoea</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Haematochezia</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Nausea</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Asthenia</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Chills</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Fatigue</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Infusion Site Erythema</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Malaise</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Non-Cardiac Chest Pain</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Pain</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Immune system disorders</title>
            <event_list>
              <event>
                <sub_title>Seasonal Allergy</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Bronchitis</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Cystitis</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Folliculitis</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Influenza</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Nasopharyngitis</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Pharyngitis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Urinary Tract Infection</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Contusion</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Skin Abrasion</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Thermal Burn</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Blood Pressure Increased</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Electrocardiogram PR Shortened</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Electrocardiogram QT Prolonged</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Electrocardiogram T Wave Inversion</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Neutrophil Count Decreased</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Protein Urine Present</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Pulmonary Function Test Abnormal</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>White Blood Cell Count Decreased</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Arthralgia</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Back Pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="3" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Muscular Weakness</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Musculoskeletal Pain</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Myalgia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Pain in Extremity</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Tendonitis</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="2" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Presyncope</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Somnolence</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Syncope</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>Anxiety</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Insomnia</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Reproductive system and breast disorders</title>
            <event_list>
              <event>
                <sub_title>Polymenorrhoea</sub_title>
                <description>This Adverse event is gender specific.</description>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="4"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="2"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="1"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Respiratory, thoracic and mediastinal disorders</title>
            <event_list>
              <event>
                <sub_title>Cough</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="2" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Dyspnoea</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Nasal Congestion</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Hyperhidrosis</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Skin Lesion</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Vascular disorders</title>
            <event_list>
              <event>
                <sub_title>Flushing</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Hot Flush</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Hypotension</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="3"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="6"/>
                <counts group_id="E3" subjects_affected="1" subjects_at_risk="6"/>
                <counts group_id="E4" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed. </restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Gennyne Walker</name_or_title>
      <organization>aTyR Pharma</organization>
      <phone>858 731 8389</phone>
      <email>clinicaltrials@atyrpharma.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/NCT02239224
org study id: ATYR1940-C-002
secondary id: 2014-001753-17
nct id: NCT02239224
lead sponsor: 
has dmc: Yes
The purpose of this study is to assess the safety and tolerability profile of ATYR1940 in the
      treatment of adult participants with molecularly defined genetic muscular dystrophies
Study ATYR1940-C-002 is a multi-national, multi-center, double-blind, randomized,
      placebo-controlled, ascending dose study designed to evaluate the safety, tolerability, PK,
      immunogenicity, and pharmacodynamic effects of ATYR1940 in participants with FSHD. Up to 44
      participants are planned to be enrolled at multiple study centers in the United States and
      Europe; the actual number of participants enrolled will depend on the number of cohorts
      initiated.

      Participants will be screened for study eligibility during the Screening period within 3
      weeks before Baseline (that is, Day 1, the first day of Study Drug administration). Eligible
      participants, based on Screening assessments, will be randomly assigned to treatment with
      ATYR1940 or placebo. Participants who are randomized will be considered enrolled in the
      study.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Double (Participant, Investigator)
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs)
time frame: Up to End of Study (up to Week 25)
description: TEAEs were defined as adverse events (AEs) with an onset following administration of the first dose of study drug. An AE was defined as any untoward medical occurrence in a participant or clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with study drug. Worsening of a pre-existing medical condition, (that is, diabetes, migraine headaches, gout) should have been considered an AE if there was either an increase in severity, frequency, or duration of the condition or an association with significantly worse outcomes. Classification of AEs was to be done by the Investigator according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 4.03. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
measure: Number of Participants With Positive Anti-Drug Antibodies (ADA)
time frame: Baseline up to Week 14
description: Titers through Week 14 are summarized. For Cohorts 1 and 2, samples that screened positive but did not confirm on confirmatory assay were not titered.
measure: Number of Participants With a Positive or Equivocal Jo-1 Antibody (Ab) Test Result
time frame: Baseline up to Week 14
description: Criterion for a positive Jo-1 Ab test result: >10.0 units/milliliter (U/mL), a cut-point associated with anti-synthetase syndrome. Criterion for an equivocal Jo-1 Ab test result: 7.0 to 10.0 U/mL. Participants were required to have a negative Jo-1 Ab test result (defined as <7 U/mL) for inclusion in the study as well as to continue dosing with study drug during the study.
measure: Number of Participants With a Clinically Significant Laboratory Abnormality
time frame: Baseline up to Week 14
description: Laboratory parameters included hematology (hematocrit, hemoglobin, platelet count, red blood cell count, white blood cell count, neutrophils, lymphocytes, monocytes, eosinophils, basophils); serum chemistries (blood urea nitrogen, creatinine, total bilirubin, aspartate aminotransferase, alanine aminotransferase, gamma-glutamyl transferase (GGT), alkaline phosphatase, sodium, total protein, bicarbonate, potassium, calcium, chloride, magnesium, inorganic phosphate, creatine phosphokinase [will be fractionated if elevated], lactic dehydrogenase, erythrocyte sedimentation rate, C-reactive protein, troponin, myoglobin, insulin-like growth factor 1, cholesterol [non-fasting]); Urinalysis (Color, pH, specific gravity, protein, glucose, ketones, blood). Clinically significant laboratory abnormalities were based upon Investigator's discretion. A summary of all Serious AEs and Other AEs (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
measure: Number of Participants With a Physical Examination Abnormality
time frame: Up to End of Study (up to Week 25)
description: Body systems that were evaluated during the physical examination included general appearance, head, eyes, ears, nose, throat (HEENT), cardiovascular system, respiratory system, chest (breasts), gastrointestinal system (abdomen), lymphatic system, musculoskeletal system, skin, psychiatric, and neurologic. Neurologic examination included assessment of mental status, memory, cranial nerves, motor function, and reflexes, and sensory testing. The body systems with at least 1 physical abnormality is presented. One participant could be represented in more than 1 body system category. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
measure: Number of Participants With a Vital Sign-Related Event Resulting in a TEAE
time frame: Up to End of Study (Up to Week 25)
description: The vital sign parameters that were evaluated included heart rate, systolic and diastolic blood pressure, and respiration rate as well as temperature. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
measure: Number of Participants With a Pulmonary Function Event Resulting in a TEAE
time frame: Up to End of Study (Up to Week 25)
description: Pulmonary function testing included measurements of forced vital capacity (FVC) and forced expiratory volume in 1 second (FEV1). A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.
measure: Percent Change From Baseline in Manual Muscle Testing (MMT) Score at Week 6 and Week 14
time frame: Baseline, Week 6 and Week 14
description: MMT was graded on a scale from 0 (no movement) to 10 (normal movement). Each side of the body and the position in which each muscle was tested were recorded for each participant. The total MMT score were calculated by averaging a converted MMT scores across all tested muscle groups. Decreased motor function was indicated by decreased individual muscle or composite MMT score.
measure: Change From Baseline in Individualized Neuromuscular Quality of Life (INQoL) - Overall QoL at Week 6 and Week 14
time frame: Baseline, Week 6 and Week 14
description: The INQoL is a validated muscle disease-specific measure of quality of life. The self-administered questionnaire consisted of 45 questions with 4 domains measuring the impact of common muscle disease symptoms (weakness, locking [or myotonia], pain, and fatigue); 5 domains measuring the influence of the muscle disease on particular areas of life (activities, independence, relationships, emotions, and body image); and the last domain focused on the positive and negative effects of treatment and was divided into 2 scores. All responses were given in a 7-point Likert scale, with improved QoL indicated by decreased scores. Overall QoL score was calculated from preselected 5 domains (activities, independence, relationships, emotions, and body image) by adding the scores from each domain. In summary, INQoL yielded 11 scores and 1 QoL score. The Overall scoring used a scale of 0-100, with improved QoL indicated by decreased scores.
measure: Maximum Observed Plasma Concentration (Cmax) of ATYR1940
time frame: Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only)
measure: Time to Reach Maximum Observed Plasma Concentration (Tmax) of ATYR1940
time frame: Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only)
measure: Area Under the Plasma Concentration Time Curve From Time 0 to Time t (AUC 0-t) of ATYR1940
time frame: Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only)
measure: Average of Half-life (T1/2) of ATYR1940
time frame: Cohort 1: Predose, 0.25, 0.5, 0.75, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 hours after the start of infusion at Weeks 2 and 5; Cohorts 2 and 3: Predose, 0.5, 1.0, 2.0, 4.0, and 6.0 hours after the start of infusion at Weeks 2, 5, and 13 (Cohort 3 only)
arm group label: Cohort 1: ATYR1940 0.3 mg/kg
arm group type: Experimental
description: Participants will receive ATYR1940 0.3 milligrams/kilograms (mg/kg) intravenous (IV) infusion once weekly for 4 weeks.
arm group label: Cohort 2: ATYR1940 1.0 mg/kg
arm group type: Experimental
description: Participants will receive ATYR1940 1.0 mg/kg IV infusion once weekly for 4 weeks.
arm group label: Cohort 3: ATYR1940 3.0 mg/kg
arm group type: Experimental
description: Participants will receive ATYR1940 3.0 mg/kg IV infusion once weekly for 12 weeks.
arm group label: Placebo
arm group type: Placebo Comparator
description: Participants will receive placebo matched to ATYR1940 IV infusion once weekly for 4 weeks in Cohorts 1 and 2 and for 12 weeks in Cohort 3.
intervention type: Biological
intervention name: Placebo
description: Concentrate for solution for infusion
arm group label: Placebo
intervention type: Biological
intervention name: ATYR1940
description: Concentrate for solution for infusion
arm group label: Cohort 1: ATYR1940 0.3 mg/kg
arm group label: Cohort 2: ATYR1940 1.0 mg/kg
arm group label: Cohort 3: ATYR1940 3.0 mg/kg
criteria: 
gender: All
minimum age: 18 Years
maximum age: 65 Years
healthy volunteers: No
last name: Sanjay Shukla, MD
role: Study Director
affiliation: aTyr Pharma
facility: 
facility: 
facility: 
facility: 
facility: 
country: France
country: Italy
country: Netherlands
country: United States
responsible party type: Sponsor
mesh term: Muscular Dystrophies
mesh term: Muscular Dystrophy, Facioscapulohumeral
sharing ipd: No
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 