<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/NCT02250443</url>
  </required_header>
  <id_info>
    <org_study_id>CBYM338X2205E1</org_study_id>
    <nct_id>NCT02250443</nct_id>
  </id_info>
  <brief_title>Study of Long-term Safety, Efficacy Tolerability of BYM338 in Patients With Sporadic Inclusion Body Myositis</brief_title>
  <acronym>BYM338</acronym>
  <official_title>An Open-label, Long-term Study to Evaluate the Safety and Tolerability of BYM338 in Patients With Sporadic Inclusion Body Myositis</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Novartis Pharmaceuticals</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Novartis</source>
  <brief_summary>
    <textblock>
      This study is an open-label, long-term study for those patients who participated in the prior&#xD;
      proof-of-concept protocol, in which the preliminary efficacy for BYM338 in patients with sIBM&#xD;
      was demonstrated after a single 30 mg/kg i.v. dose of BYM338. This study is designed to&#xD;
      confirm the efficacy, safety and tolerability of BYM338 in sIBM with long-term dosing.&#xD;
      However due to lack of efficacy in patients with sIBM, the study was terminated early.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This is a non-confirmatory, multicenter, open-label, non-randomized trial which will extend&#xD;
      active treatment to those patients that participated in the preceding proof-of-concept study&#xD;
      (CBYM338X2205) in order to collect long-term safety and tolerability data. Up to 14 patients&#xD;
      with sIBM will be invited to enroll into the study to receive BYM338 open-label for a period&#xD;
      of approximately 3 years or until BYM338 is commercially available, whichever comes first.&#xD;
      The study consists of a maximum 28-day screening period, a 5-day baseline period, and a&#xD;
      treatment period consisting of the site visits at 4-week intervals for treatment&#xD;
      administration, safety and pharmacokinetic follow-up. All patients will be administered a&#xD;
      medium level i.v. BYM338 dose regardless of their treatment allocation in the prior study.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">March 11, 2014</start_date>
  <completion_date type="Actual">August 23, 2016</completion_date>
  <primary_completion_date type="Actual">August 23, 2016</primary_completion_date>
  <phase>Phase 2/Phase 3</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Number of Participants With Adverse Events as a Measure of Safety and Tolerability</measure>
    <time_frame>Up to 29 month</time_frame>
    <description>Any Adverse Event was defined as occurrence of any symptom regardless of intensity grade, Serious Adverse Event (SAEs) assessed as medical occurrences that result in death, are life threatening, require hospitalization or prolongation of hospitalization or result in persistent or significant disability/incapacity</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Changes From Baseline in Lean Body Mass (LBM) by Dual-Energy X-ray Absorptiometery (DXA)</measure>
    <time_frame>Baseline, Day 1, 57, 113, 169, 365, 533, and day 729</time_frame>
    <description>To assess the effect of multiple doses of BYM338 on lean body mass as measured by DXA in terms of change from baseline.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Pharmacokinetics (PK) Parameter of Cmin From Multiple i.v. Dosing</measure>
    <time_frame>Day 29, 85, 169, 253, 337, 421, 505, 589, 673, 757, 1177</time_frame>
    <description>To obtain pharmacokinetic data from multiple i.v. dosing of BYM338 in this patient population. Pre-dose, 30 mins &amp; 4 hours post-dose on Day 1. Pre-dose only on each subsequent administration</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Changes From Baseline in Physical Function Reported by Patients</measure>
    <time_frame>Baseline, Week 104</time_frame>
    <description>Self-reported physical function was assessed by a newly developed patient reported outcome named sporadic inclusion body myositis (sIBM) functional assessment (sIFA). The sIFA consists of 11 items scored on an 11 point numerical rating scale from 0 (no difficulty) to 10 (unable to do) across 3 domains: upper body functioning, lower body functioning and general functioning. Participants completed the assessment where the recall period was the past week prior to completing the patient reported outcome (PRO). The total score on the sIFA scale ranges from 0 (minimum) to 110 (maximum). Higher values represent a worse outcome. A positive change from baseline indicates deterioration. Due to the no-signal this analysis was cancelled.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Changes From Baseline in Muscle Strength.</measure>
    <time_frame>Baseline, Day 1, 113, 169, 365, 533, 729</time_frame>
    <description>Quadriceps muscle strength was measured, Quadriceps Quantitative Muscle Testing (QMT) by portable fixed dynamometry (PFD). A negative change from baseline indicates deterioration</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Changes From Baseline in Muscle Function (Hand-grip and Pinch-grip Dynamometry)</measure>
    <time_frame>Baseline,Day 1, 113, 169, 365, 533, 729</time_frame>
    <description>The effect of BYM338 on additional muscle function measures (hand-grip and pinch-grip dynamometry).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Changes From Baseline in Muscle Function 6 Minute Walking Distance</measure>
    <time_frame>Baseline,Day 1, 113, 169, 365, 533, 729</time_frame>
    <description>The effect of BYM338 on additional muscle function measures (6 minute walking distance). The 6MWD test measured the distance (in meters) that a participant walked in a 6 minute timeframe. A positive change from baseline indicates improvement.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change From Baseline of Thigh Muscle Volume (TMV) by MRI Scan</measure>
    <time_frame>Baseline, Day 1, 57, 113</time_frame>
    <description>Thigh Muscle Volume (TMV) change was evaluated by a responder analysis. Patients whose loss of muscle TMV by MRI was equal or more than 2% at Week 8 and 16 were considered responders</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Pharmacokinetics (PK) Parameter of Cmax</measure>
    <time_frame>Day 1</time_frame>
    <description>To obtain pharmacokinetic data from multiple i.v. dosing of BYM338 in this patient population. Pre-dose, 30 mins &amp; 4 hours post-dose on Day 1.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Time to Reach the Maximum Concentration After Drug Administration (Tmax)</measure>
    <time_frame>Day 1</time_frame>
    <description>The time to reach the maximum concentration after drug administration</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">10</enrollment>
  <condition>Sporadic Inclusion Body Myositis (sIBM)</condition>
  <arm_group>
    <arm_group_label>BYM338</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>BYM338 Group</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>BYM338 (Bimagrumab)</intervention_name>
    <arm_group_label>BYM338</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Written informed consent must be obtained before any assessment is performed.&#xD;
&#xD;
          -  Patients who participated in the CBYM338X2205 study. A patient is defined as&#xD;
             participating in the study if they were enrolled and received study medication.&#xD;
&#xD;
          -  Able to communicate well with the investigator, to understand and comply with the&#xD;
             requirements of the study.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Patients for whom the treating physician considers it inappropriate for continuation&#xD;
             into the study, including consideration of physical and laboratory assessment of the&#xD;
             patient at screening.&#xD;
&#xD;
          -  Patients who were non-compliant or demonstrated a serious protocol deviation in the&#xD;
             previous study.&#xD;
&#xD;
          -  Use of other investigational drugs at the time of enrollment, within 30 days or 5&#xD;
             half-lives of enrollment, or until the expected PD effect has returned to baseline,&#xD;
             whichever is longer; or longer if required by local regulations.&#xD;
&#xD;
          -  History of hypersensitivity to any of the study drugs or to drugs of similar chemical&#xD;
             classes&#xD;
&#xD;
          -  Swallowing difficulty or other reason that precludes adequate intake of energy and&#xD;
             protein, defined as at least 20 kcal/kg/day and 0.6 g protein/kg/day as determined by&#xD;
             the investigators assessment.&#xD;
&#xD;
          -  On the Columbia-Suicide Severity Rating Scale, a score of 4 or 5 on the Suicidal&#xD;
             Ideation item or any &quot;yes&quot; on the Suicidal Behavior item that is related to suicidal&#xD;
             behavior occurring during the last 2 years.&#xD;
&#xD;
          -  Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a&#xD;
             female after conception and until the termination of gestation, confirmed by a&#xD;
             positive hCG laboratory test.&#xD;
&#xD;
          -  Women of child-bearing potential, defined as all women physiologically capable of&#xD;
             becoming pregnant, must use highly effective contraception during the study and for 5&#xD;
             half-lives (14 weeks) after stopping treatment. Highly effective contraception is&#xD;
             defined as either:&#xD;
&#xD;
               1. Total abstinence: When this is in line with the preferred and usual lifestyle of&#xD;
                  the subject. [Periodic abstinence (e.g., calendar, ovulation, symptothermal,&#xD;
                  post-ovulation methods) and withdrawal are not acceptable methods of&#xD;
                  contraception].&#xD;
&#xD;
               2. Sterilization: have had surgical bilateral oophorectomy (with or without&#xD;
                  hysterectomy) or tubal ligation at least six weeks before taking study treatment.&#xD;
                  In case of oophorectomy alone, only when the reproductive status of the woman has&#xD;
                  been confirmed by follow up hormone level assessment.&#xD;
&#xD;
               3. Male partner sterilization (with the appropriate post-vasectomy documentation of&#xD;
                  the absence of sperm in the ejaculate). [For female study subjects, the&#xD;
                  vasectomized male partner should be the sole partner for that patient].&#xD;
&#xD;
               4. Use of a combination of any two of the following (a+b or a+c or b+c):&#xD;
&#xD;
               1. Use of oral, injected or implanted hormonal methods of contraception.&#xD;
&#xD;
               2. Placement of an intrauterine device (IUD) or intrauterine system (IUS).&#xD;
&#xD;
               3. Barrier methods of contraception: Condom or Occlusive cap (diaphragm or&#xD;
                  cervical/vault caps) with spermicidal foam/gel/film/cream/vaginal suppository.&#xD;
&#xD;
        In case of use of oral contraception women should have been stable on the same pill for a&#xD;
        minimum of 3 months before taking study treatment. Postmenopausal females must have had no&#xD;
        regular menstrual bleeding for at least one (1) year prior to initial dosing. Menopause&#xD;
        will be confirmed by a plasma FSH level of &gt; 40 IU/L (or as determined by the cut off used&#xD;
        by the local clinical laboratory) at screening. Female patients who report surgical&#xD;
        sterilization must have had the procedure at least six (6) months prior to initial dosing.&#xD;
        Surgical sterilization procedures should be supported with clinical documentation made&#xD;
        available to the sponsor and noted in the Relevant Medical History / Current Medical&#xD;
        Conditions section of the eCRF. All female patients must have negative pregnancy test&#xD;
        results at screening and baseline.&#xD;
&#xD;
          -  Use of oral beta agonists, oral corticosteroids, androgens or androgen inhibitors&#xD;
             (including LHRH agonists), or intravenous gamma globulin (IVIG) within the previous 6&#xD;
             months. Short-term corticosteroids for unrelated indications, defined as &lt; 20 mg/d for&#xD;
             &lt; 30 days and ending at least 60 days prior to screening, are acceptable.&#xD;
&#xD;
          -  Patients with known bleeding disorders, or who are under treatment with&#xD;
             anti-coagulants.&#xD;
&#xD;
          -  A presence or history of clinically relevant ECG abnormalities, or including but not&#xD;
             limited to Long QT syndrome, ischemic changes that cannot be shown to be stable for at&#xD;
             least 6 months by previous ECG, or 2nd degree Mobitz II or 3rd degree heart block.&#xD;
&#xD;
          -  History of malignancy of any organ system (other than localized basal cell carcinoma&#xD;
             of the skin), treated or untreated, within the past 5 years, regardless of whether&#xD;
             there is evidence of local recurrence or metastases.&#xD;
&#xD;
          -  Significant illness which has not resolved within two (2) weeks prior to initial&#xD;
             dosing.&#xD;
&#xD;
          -  A positive HIV, Hepatitis B surface antigen or Hepatitis C test result. No additional&#xD;
             exclusions may be applied by the investigator, in order to ensure that the study&#xD;
             population will be representative of all eligible patients.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>40 Years</minimum_age>
    <maximum_age>75 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Novartis Pharmaceuticals</last_name>
    <role>Study Director</role>
    <affiliation>Novartis Pharmaceuticals</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Phoenix</city>
        <state>Arizona</state>
        <zip>85013</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Novartis Investigative Site</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <link>
    <url>https://www.novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=184</url>
    <description>A Plain Language Trial Summary is available on novartisclinicaltrials.com</description>
  </link>
  <verification_date>March 2019</verification_date>
  <study_first_submitted>March 20, 2014</study_first_submitted>
  <study_first_submitted_qc>September 24, 2014</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 26, 2014</study_first_posted>
  <results_first_submitted>August 9, 2017</results_first_submitted>
  <results_first_submitted_qc>March 12, 2018</results_first_submitted_qc>
  <results_first_posted type="Actual">April 10, 2018</results_first_posted>
  <last_update_submitted>December 9, 2020</last_update_submitted>
  <last_update_submitted_qc>December 9, 2020</last_update_submitted_qc>
  <last_update_posted type="Actual">December 30, 2020</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Inclusion Body Myositis (IBM)</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Myositis</mesh_term>
    <mesh_term>Myositis, Inclusion Body</mesh_term>
  </condition_browse>

  <clinical_results>

    <participant_flow>
      <recruitment_details>Due to lack of efficacy in patients with sIBM, the study was terminated early.</recruitment_details>
      <group_list>
        <group group_id="P1">
          <title>BYM338</title>
          <description>BYM338 Group</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="10"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="10"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Administrative problems</title>
              <participants_list>
                <participants group_id="P1" count="7"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Adverse Event</title>
              <participants_list>
                <participants group_id="P1" count="3"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>BYM338</title>
          <description>BYM338 Group</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="10"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>Years</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="70.1" spread="10.39"/>
                  </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="4"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Number of Participants With Adverse Events as a Measure of Safety and Tolerability</title>
        <description>Any Adverse Event was defined as occurrence of any symptom regardless of intensity grade, Serious Adverse Event (SAEs) assessed as medical occurrences that result in death, are life threatening, require hospitalization or prolongation of hospitalization or result in persistent or significant disability/incapacity</description>
        <time_frame>Up to 29 month</time_frame>
        <population>safety analysis set - included all patients that received at least one dose of study drug. No statistical analysis provided for Number of Participants Who Experienced Adverse Events (AEs) and Serious Adverse Events (SAEs) During this extension study</population>
        <group_list>
          <group group_id="O1">
            <title>BYM338</title>
            <description>BYM338 Group</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Participants With Adverse Events as a Measure of Safety and Tolerability</title>
          <description>Any Adverse Event was defined as occurrence of any symptom regardless of intensity grade, Serious Adverse Event (SAEs) assessed as medical occurrences that result in death, are life threatening, require hospitalization or prolongation of hospitalization or result in persistent or significant disability/incapacity</description>
          <population>safety analysis set - included all patients that received at least one dose of study drug. No statistical analysis provided for Number of Participants Who Experienced Adverse Events (AEs) and Serious Adverse Events (SAEs) During this extension study</population>
          <units>Participants</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="10"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Death</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Serious adverse events (SAE)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Adverse Events (AE)</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="10"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Changes From Baseline in Lean Body Mass (LBM) by Dual-Energy X-ray Absorptiometery (DXA)</title>
        <description>To assess the effect of multiple doses of BYM338 on lean body mass as measured by DXA in terms of change from baseline.</description>
        <time_frame>Baseline, Day 1, 57, 113, 169, 365, 533, and day 729</time_frame>
        <population>Pharmacodynamics (PD) analysis set: Patients with evaluable PD parameter data</population>
        <group_list>
          <group group_id="O1">
            <title>BYM338</title>
            <description>BYM338 Group</description>
          </group>
        </group_list>
        <measure>
          <title>Changes From Baseline in Lean Body Mass (LBM) by Dual-Energy X-ray Absorptiometery (DXA)</title>
          <description>To assess the effect of multiple doses of BYM338 on lean body mass as measured by DXA in terms of change from baseline.</description>
          <population>Pharmacodynamics (PD) analysis set: Patients with evaluable PD parameter data</population>
          <units>Percentage Change in LBM</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="10"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Day 1</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0" spread="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 57</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4.292" spread="2.7480"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 113</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="5.552" spread="3.6066"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 169</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="7.463" spread="4.5687"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 365</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="6.919" spread="2.9669"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 533</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="8"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="6.885" spread="3.7894"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 729</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="1"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.727" spread="NA">NA- Not available, standard deviation is not evaluable when n=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Pharmacokinetics (PK) Parameter of Cmin From Multiple i.v. Dosing</title>
        <description>To obtain pharmacokinetic data from multiple i.v. dosing of BYM338 in this patient population. Pre-dose, 30 mins &amp; 4 hours post-dose on Day 1. Pre-dose only on each subsequent administration</description>
        <time_frame>Day 29, 85, 169, 253, 337, 421, 505, 589, 673, 757, 1177</time_frame>
        <population>Pharmacokinetics (PK) Analysis set: Patients with available PK data and no protocol deviations with relevant impact on PK data</population>
        <group_list>
          <group group_id="O1">
            <title>BYM338</title>
            <description>BYM338 Group</description>
          </group>
        </group_list>
        <measure>
          <title>Pharmacokinetics (PK) Parameter of Cmin From Multiple i.v. Dosing</title>
          <description>To obtain pharmacokinetic data from multiple i.v. dosing of BYM338 in this patient population. Pre-dose, 30 mins &amp; 4 hours post-dose on Day 1. Pre-dose only on each subsequent administration</description>
          <population>Pharmacokinetics (PK) Analysis set: Patients with available PK data and no protocol deviations with relevant impact on PK data</population>
          <units>ng/mL</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="10"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Day 29 (n=10)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="13.4" spread="5.01"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 85 (n=10)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="24.1" spread="13.1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 169 (n=10)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="26.3" spread="15.9"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 253 (n=9)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="9"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="28.8" spread="12.1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 337 (n=10)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="24.4" spread="14.6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 421 (n=9)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="9"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="24.5" spread="11.5"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 505 (n=8)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="8"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="28.3" spread="10.6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 589 (n=6)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="6"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="39.8" spread="30.2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 673 (n=2)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="2"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="20.5" spread="1.98"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 757 (n=1)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="1"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="20.3" spread="NA">NA - Not available, standard deviation is not evaluable when n=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 1177 (n=1)</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="1"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="31.4" spread="NA">NA - Not available, standard deviation is not evaluable when n=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Changes From Baseline in Physical Function Reported by Patients</title>
        <description>Self-reported physical function was assessed by a newly developed patient reported outcome named sporadic inclusion body myositis (sIBM) functional assessment (sIFA). The sIFA consists of 11 items scored on an 11 point numerical rating scale from 0 (no difficulty) to 10 (unable to do) across 3 domains: upper body functioning, lower body functioning and general functioning. Participants completed the assessment where the recall period was the past week prior to completing the patient reported outcome (PRO). The total score on the sIFA scale ranges from 0 (minimum) to 110 (maximum). Higher values represent a worse outcome. A positive change from baseline indicates deterioration. Due to the no-signal this analysis was cancelled.</description>
        <time_frame>Baseline, Week 104</time_frame>
        <population>Due to the early study termination and the small sample size in this open-label trial, this PRO analysis was cancelled.</population>
        <group_list>
          <group group_id="O1">
            <title>BYM338</title>
            <description>BYM338 Group</description>
          </group>
        </group_list>
        <measure>
          <title>Changes From Baseline in Physical Function Reported by Patients</title>
          <description>Self-reported physical function was assessed by a newly developed patient reported outcome named sporadic inclusion body myositis (sIBM) functional assessment (sIFA). The sIFA consists of 11 items scored on an 11 point numerical rating scale from 0 (no difficulty) to 10 (unable to do) across 3 domains: upper body functioning, lower body functioning and general functioning. Participants completed the assessment where the recall period was the past week prior to completing the patient reported outcome (PRO). The total score on the sIFA scale ranges from 0 (minimum) to 110 (maximum). Higher values represent a worse outcome. A positive change from baseline indicates deterioration. Due to the no-signal this analysis was cancelled.</description>
          <population>Due to the early study termination and the small sample size in this open-label trial, this PRO analysis was cancelled.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Changes From Baseline in Muscle Strength.</title>
        <description>Quadriceps muscle strength was measured, Quadriceps Quantitative Muscle Testing (QMT) by portable fixed dynamometry (PFD). A negative change from baseline indicates deterioration</description>
        <time_frame>Baseline, Day 1, 113, 169, 365, 533, 729</time_frame>
        <population>Pharmacodynamics (PD) analysis set: Patients with available PD data and no protocol deviations with relevant impact on PD data</population>
        <group_list>
          <group group_id="O1">
            <title>BYM338</title>
            <description>BYM338 Group</description>
          </group>
        </group_list>
        <measure>
          <title>Changes From Baseline in Muscle Strength.</title>
          <description>Quadriceps muscle strength was measured, Quadriceps Quantitative Muscle Testing (QMT) by portable fixed dynamometry (PFD). A negative change from baseline indicates deterioration</description>
          <population>Pharmacodynamics (PD) analysis set: Patients with available PD data and no protocol deviations with relevant impact on PD data</population>
          <units>Newtons</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="10"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Quadriceps score left side Day 1</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="9"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0" spread="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>left side Day 113</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="9"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-5.80" spread="28.160"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>left side Day 169</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="8"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-5.95" spread="26.587"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>left side Day 365</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="9"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-9.51" spread="28.149"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>left side Day 533</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="7"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-25.77" spread="21.321"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>left side Day 729</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="1"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="178.26" spread="NA">NA- Not available, standard deviation is not evaluable when n=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Quadriceps score Right side Day 1</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="9"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0" spread="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Right side Day 113</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-4.83" spread="29.904"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Right side Day 169</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="9"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-22.81" spread="18.784"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Right side Day 365</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="9"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-11.63" spread="47.141"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Right side Day 533</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="7"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-31.00" spread="29.173"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Right side Day 729</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="1"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-57.66" spread="NA">NA - Not available, standard deviation is not evaluable when n=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Changes From Baseline in Muscle Function (Hand-grip and Pinch-grip Dynamometry)</title>
        <description>The effect of BYM338 on additional muscle function measures (hand-grip and pinch-grip dynamometry).</description>
        <time_frame>Baseline,Day 1, 113, 169, 365, 533, 729</time_frame>
        <population>Pharmacodynamics (PD) analysis set: Patients with available PD data and no protocol deviations with relevant impact on PD data</population>
        <group_list>
          <group group_id="O1">
            <title>BYM338</title>
            <description>BYM338 Group</description>
          </group>
        </group_list>
        <measure>
          <title>Changes From Baseline in Muscle Function (Hand-grip and Pinch-grip Dynamometry)</title>
          <description>The effect of BYM338 on additional muscle function measures (hand-grip and pinch-grip dynamometry).</description>
          <population>Pharmacodynamics (PD) analysis set: Patients with available PD data and no protocol deviations with relevant impact on PD data</population>
          <units>Newtons</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="10"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Left hand-grip day 1</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0" spread="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Left hand-grip day 113</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="25.58" spread="37.544"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Left hand-grip day 169</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="21.36" spread="43.815"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Left hand-grip day 365</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-1.42" spread="47.973"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Left hand-grip day 533</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="7"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="2.78" spread="16.934"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Left hand-grip day 729</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="2"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="8.95" spread="24.034"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Right hand-grip day 1</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0" spread="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Right hand-grip day 113</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="99.59" spread="184.472"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Right hand-grip day 169</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="109.05" spread="214.248"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Right hand-grip day 365</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="77.46" spread="160.133"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Right hand-grip day 533</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="8"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="53.99" spread="127.776"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Right hand-grip day 729</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="2"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="163.66" spread="237.402"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Left hand pinch grip day 1</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="1"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0" spread="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Left hand pinch grip day 113</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="7.82" spread="27.292"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Left hand pinch grip day 169</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-4.74" spread="20.210"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Left hand pinch grip day 365</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-7.30" spread="35.224"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Left hand pinch grip day 533</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="8"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-11.40" spread="21.204"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Left hand pinch grip day 729</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="2"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-8.41" spread="46.672"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Right hand pinch grip day 1</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0" spread="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Right hand pinch grip day 113</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-7.55" spread="26.792"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Right hand pinch grip day 169</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-9.35" spread="30.942"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Right hand pinch grip day 365</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.59" spread="32.094"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Right hand pinch grip day 533</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="8"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-8.65" spread="21.491"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Right hand pinch grip day 729</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="2"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-35.40" spread="44.820"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Changes From Baseline in Muscle Function 6 Minute Walking Distance</title>
        <description>The effect of BYM338 on additional muscle function measures (6 minute walking distance). The 6MWD test measured the distance (in meters) that a participant walked in a 6 minute timeframe. A positive change from baseline indicates improvement.</description>
        <time_frame>Baseline,Day 1, 113, 169, 365, 533, 729</time_frame>
        <population>Pharmacodynamics (PD) analysis set: Patients with available PD data and no protocol deviations with relevant impact on PD data</population>
        <group_list>
          <group group_id="O1">
            <title>BYM338</title>
            <description>BYM338 Group</description>
          </group>
        </group_list>
        <measure>
          <title>Changes From Baseline in Muscle Function 6 Minute Walking Distance</title>
          <description>The effect of BYM338 on additional muscle function measures (6 minute walking distance). The 6MWD test measured the distance (in meters) that a participant walked in a 6 minute timeframe. A positive change from baseline indicates improvement.</description>
          <population>Pharmacodynamics (PD) analysis set: Patients with available PD data and no protocol deviations with relevant impact on PD data</population>
          <units>Meters</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="10"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Day 1</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="10"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0" spread="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 113</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="9"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-1.56" spread="13.685"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 169</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="9"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-7.41" spread="12.463"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 365</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="7"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-8.97" spread="15.763"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 533</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="6"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-16.99" spread="22.382"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 729</title>
              <analyzed_list>
                <analyzed>
                  <units>Participants</units>
                  <scope>Class</scope>
                  <count_list>
                    <count group_id="O1" value="1"/>
                  </count_list>
                </analyzed>
              </analyzed_list>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="-17.86" spread="NA">NA - Not available, standard deviation is not evaluable when n=1</measurement>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Change From Baseline of Thigh Muscle Volume (TMV) by MRI Scan</title>
        <description>Thigh Muscle Volume (TMV) change was evaluated by a responder analysis. Patients whose loss of muscle TMV by MRI was equal or more than 2% at Week 8 and 16 were considered responders</description>
        <time_frame>Baseline, Day 1, 57, 113</time_frame>
        <population>Pharmacodynamics (PD) analysis set: Patients with available PD data and no protocol deviations with relevant impact on PD data</population>
        <group_list>
          <group group_id="O1">
            <title>BYM338</title>
            <description>BYM338 Group</description>
          </group>
        </group_list>
        <measure>
          <title>Change From Baseline of Thigh Muscle Volume (TMV) by MRI Scan</title>
          <description>Thigh Muscle Volume (TMV) change was evaluated by a responder analysis. Patients whose loss of muscle TMV by MRI was equal or more than 2% at Week 8 and 16 were considered responders</description>
          <population>Pharmacodynamics (PD) analysis set: Patients with available PD data and no protocol deviations with relevant impact on PD data</population>
          <units>Percentage Change</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="10"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Day 1</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0" spread="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 57</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4.14" spread="4.250"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Day 113</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="4.51" spread="6.300"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Pharmacokinetics (PK) Parameter of Cmax</title>
        <description>To obtain pharmacokinetic data from multiple i.v. dosing of BYM338 in this patient population. Pre-dose, 30 mins &amp; 4 hours post-dose on Day 1.</description>
        <time_frame>Day 1</time_frame>
        <population>Pharmacokinetics (PK) Analysis set: Patients with available PK data and no protocol deviations with relevant impact on PK data</population>
        <group_list>
          <group group_id="O1">
            <title>BYM338</title>
            <description>BYM338 Group</description>
          </group>
        </group_list>
        <measure>
          <title>Pharmacokinetics (PK) Parameter of Cmax</title>
          <description>To obtain pharmacokinetic data from multiple i.v. dosing of BYM338 in this patient population. Pre-dose, 30 mins &amp; 4 hours post-dose on Day 1.</description>
          <population>Pharmacokinetics (PK) Analysis set: Patients with available PK data and no protocol deviations with relevant impact on PK data</population>
          <units>ug/mL</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="278" spread="62.6"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Time to Reach the Maximum Concentration After Drug Administration (Tmax)</title>
        <description>The time to reach the maximum concentration after drug administration</description>
        <time_frame>Day 1</time_frame>
        <population>Pharmacokinetics (PK) Analysis set: Patients with available PK data and no protocol deviations with relevant impact on PK data</population>
        <group_list>
          <group group_id="O1">
            <title>BYM338</title>
            <description>BYM338 Group</description>
          </group>
        </group_list>
        <measure>
          <title>Time to Reach the Maximum Concentration After Drug Administration (Tmax)</title>
          <description>The time to reach the maximum concentration after drug administration</description>
          <population>Pharmacokinetics (PK) Analysis set: Patients with available PK data and no protocol deviations with relevant impact on PK data</population>
          <units>hr</units>
          <param>Median</param>
          <dispersion>Full Range</dispersion>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="9"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0.744" spread="NA" lower_limit="0.648" upper_limit="4.73"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>period up to 104 weeks</time_frame>
      <group_list>
        <group group_id="E1">
          <title>BYM338 10mg/kg i.v.</title>
          <description>BYM338 10mg/kg i.v.</description>
        </group>
      </group_list>
      <serious_events>
        <default_vocab>MedDRA (19.1)</default_vocab>
        <default_assessment>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="2" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>Anaemia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Cardiac disorders</title>
            <event_list>
              <event>
                <sub_title>Myocardial infarction</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Tachyarrhythmia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Gastrointestinal haemorrhage</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Haemorrhoidal haemorrhage</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Dehydration</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Iron deficiency</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Neoplasms benign, malignant and unspecified (incl cysts and polyps)</title>
            <event_list>
              <event>
                <sub_title>Lung adenocarcinoma</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Oesophageal carcinoma</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>0</frequency_threshold>
        <default_vocab>MedDRA (19.1)</default_vocab>
        <default_assessment>Systematic Assessment</default_assessment>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="10" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>Lymphadenopathy</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Cardiac disorders</title>
            <event_list>
              <event>
                <sub_title>Atrial fibrillation</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Myocardial infarction</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Ear and labyrinth disorders</title>
            <event_list>
              <event>
                <sub_title>Tinnitus</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Abdominal pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Diarrhoea</sub_title>
                <counts group_id="E1" subjects_affected="6" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Frequent bowel movements</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Haemorrhoidal haemorrhage</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Inguinal hernia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Nausea</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Vomiting</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Oedema peripheral</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Peripheral swelling</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Cellulitis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Fungal skin infection</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Influenza</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Nasopharyngitis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Upper respiratory tract infection</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Urinary tract infection</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Injury, poisoning and procedural complications</title>
            <event_list>
              <event>
                <sub_title>Avulsion fracture</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Bone contusion</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Concussion</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Contusion</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Corneal abrasion</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Fall</sub_title>
                <counts group_id="E1" subjects_affected="9" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Laceration</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Ligament sprain</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Limb injury</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Scratch</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Skin abrasion</sub_title>
                <counts group_id="E1" subjects_affected="5" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Tibia fracture</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Mammogram abnormal</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Natural killer cell count increased</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Vitamin D decreased</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Weight decreased</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Abnormal loss of weight</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Decreased appetite</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Glucose tolerance impaired</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Gout</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Iron deficiency</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </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="3" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Back pain</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Bursitis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Muscle spasms</sub_title>
                <counts group_id="E1" subjects_affected="9" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Muscular weakness</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Musculoskeletal pain</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Myalgia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Neoplasms benign, malignant and unspecified (incl cysts and polyps)</title>
            <event_list>
              <event>
                <sub_title>Basal cell carcinoma</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Squamous cell carcinoma of skin</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Nervous system disorders</title>
            <event_list>
              <event>
                <sub_title>Dementia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Dizziness</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Dysgeusia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Headache</sub_title>
                <counts group_id="E1" subjects_affected="2" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Hypogeusia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Psychiatric disorders</title>
            <event_list>
              <event>
                <sub_title>Depression</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Insomnia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Renal and urinary disorders</title>
            <event_list>
              <event>
                <sub_title>Nephrolithiasis</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Urinary incontinence</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Urinary retention</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Reproductive system and breast disorders</title>
            <event_list>
              <event>
                <sub_title>Breast hyperplasia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Cervical polyp</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </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="10"/>
              </event>
              <event>
                <sub_title>Dysphonia</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Productive cough</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Upper-airway cough syndrome</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Skin and subcutaneous tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Acne</sub_title>
                <counts group_id="E1" subjects_affected="5" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Blister</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Night sweats</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Papule</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Pruritus</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Rash</sub_title>
                <counts group_id="E1" subjects_affected="3" subjects_at_risk="10"/>
              </event>
              <event>
                <sub_title>Skin hypertrophy</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Vascular disorders</title>
            <event_list>
              <event>
                <sub_title>Thrombophlebitis superficial</sub_title>
                <counts group_id="E1" subjects_affected="1" subjects_at_risk="10"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.</restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Study Director</name_or_title>
      <organization>Novartis Pharmaceutical</organization>
      <phone>862-778-8300</phone>
    </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/NCT02250443
org study id: CBYM338X2205E1
nct id: NCT02250443
lead sponsor: 
This study is an open-label, long-term study for those patients who participated in the prior
      proof-of-concept protocol, in which the preliminary efficacy for BYM338 in patients with sIBM
      was demonstrated after a single 30 mg/kg i.v. dose of BYM338. This study is designed to
      confirm the efficacy, safety and tolerability of BYM338 in sIBM with long-term dosing.
      However due to lack of efficacy in patients with sIBM, the study was terminated early.
This is a non-confirmatory, multicenter, open-label, non-randomized trial which will extend
      active treatment to those patients that participated in the preceding proof-of-concept study
      (CBYM338X2205) in order to collect long-term safety and tolerability data. Up to 14 patients
      with sIBM will be invited to enroll into the study to receive BYM338 open-label for a period
      of approximately 3 years or until BYM338 is commercially available, whichever comes first.
      The study consists of a maximum 28-day screening period, a 5-day baseline period, and a
      treatment period consisting of the site visits at 4-week intervals for treatment
      administration, safety and pharmacokinetic follow-up. All patients will be administered a
      medium level i.v. BYM338 dose regardless of their treatment allocation in the prior study.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Number of Participants With Adverse Events as a Measure of Safety and Tolerability
time frame: Up to 29 month
description: Any Adverse Event was defined as occurrence of any symptom regardless of intensity grade, Serious Adverse Event (SAEs) assessed as medical occurrences that result in death, are life threatening, require hospitalization or prolongation of hospitalization or result in persistent or significant disability/incapacity
measure: Changes From Baseline in Lean Body Mass (LBM) by Dual-Energy X-ray Absorptiometery (DXA)
time frame: Baseline, Day 1, 57, 113, 169, 365, 533, and day 729
description: To assess the effect of multiple doses of BYM338 on lean body mass as measured by DXA in terms of change from baseline.
measure: Pharmacokinetics (PK) Parameter of Cmin From Multiple i.v. Dosing
time frame: Day 29, 85, 169, 253, 337, 421, 505, 589, 673, 757, 1177
description: To obtain pharmacokinetic data from multiple i.v. dosing of BYM338 in this patient population. Pre-dose, 30 mins & 4 hours post-dose on Day 1. Pre-dose only on each subsequent administration
measure: Changes From Baseline in Physical Function Reported by Patients
time frame: Baseline, Week 104
description: Self-reported physical function was assessed by a newly developed patient reported outcome named sporadic inclusion body myositis (sIBM) functional assessment (sIFA). The sIFA consists of 11 items scored on an 11 point numerical rating scale from 0 (no difficulty) to 10 (unable to do) across 3 domains: upper body functioning, lower body functioning and general functioning. Participants completed the assessment where the recall period was the past week prior to completing the patient reported outcome (PRO). The total score on the sIFA scale ranges from 0 (minimum) to 110 (maximum). Higher values represent a worse outcome. A positive change from baseline indicates deterioration. Due to the no-signal this analysis was cancelled.
measure: Changes From Baseline in Muscle Strength.
time frame: Baseline, Day 1, 113, 169, 365, 533, 729
description: Quadriceps muscle strength was measured, Quadriceps Quantitative Muscle Testing (QMT) by portable fixed dynamometry (PFD). A negative change from baseline indicates deterioration
measure: Changes From Baseline in Muscle Function (Hand-grip and Pinch-grip Dynamometry)
time frame: Baseline,Day 1, 113, 169, 365, 533, 729
description: The effect of BYM338 on additional muscle function measures (hand-grip and pinch-grip dynamometry).
measure: Changes From Baseline in Muscle Function 6 Minute Walking Distance
time frame: Baseline,Day 1, 113, 169, 365, 533, 729
description: The effect of BYM338 on additional muscle function measures (6 minute walking distance). The 6MWD test measured the distance (in meters) that a participant walked in a 6 minute timeframe. A positive change from baseline indicates improvement.
measure: Change From Baseline of Thigh Muscle Volume (TMV) by MRI Scan
time frame: Baseline, Day 1, 57, 113
description: Thigh Muscle Volume (TMV) change was evaluated by a responder analysis. Patients whose loss of muscle TMV by MRI was equal or more than 2% at Week 8 and 16 were considered responders
measure: Pharmacokinetics (PK) Parameter of Cmax
time frame: Day 1
description: To obtain pharmacokinetic data from multiple i.v. dosing of BYM338 in this patient population. Pre-dose, 30 mins & 4 hours post-dose on Day 1.
measure: Time to Reach the Maximum Concentration After Drug Administration (Tmax)
time frame: Day 1
description: The time to reach the maximum concentration after drug administration
arm group label: BYM338
arm group type: Experimental
description: BYM338 Group
intervention type: Drug
intervention name: BYM338 (Bimagrumab)
arm group label: BYM338
criteria: 
gender: All
minimum age: 40 Years
maximum age: 75 Years
healthy volunteers: No
last name: Novartis Pharmaceuticals
role: Study Director
affiliation: Novartis Pharmaceuticals
facility: 
facility: 
country: United States
url: https://www.novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=184
description: A Plain Language Trial Summary is available on novartisclinicaltrials.com
responsible party type: Sponsor
mesh term: Myositis
mesh term: Myositis, Inclusion Body
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 