<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/NCT01706159</url>
  </required_header>
  <id_info>
    <org_study_id>NN8717-3946</org_study_id>
    <secondary_id>2011-001568-22</secondary_id>
    <secondary_id>U1111-1120-3824</secondary_id>
    <nct_id>NCT01706159</nct_id>
  </id_info>
  <brief_title>A Placebo-controlled Trial With rFXIII Administered to Subjects With Mild to Moderate Active Ulcerative Colitis</brief_title>
  <official_title>A Multicenter, Randomised, Double-blind, Placebo-controlled, Multiple-dose Trial With rFXIII Administered to Subjects With Mild to Moderate Active Ulcerative Colitis</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Novo Nordisk A/S</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Novo Nordisk A/S</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      This trial is conducted in Europe. The aim of the trial is to investigate the effect of&#xD;
      recombinant factor XIII (rFXIII) administered to subjects with mild to moderate active&#xD;
      ulcerative colitis (UC).&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    Trial screening data did not support the medical hypothesis&#xD;
  </why_stopped>
  <start_date>October 2012</start_date>
  <completion_date type="Actual">July 2013</completion_date>
  <primary_completion_date type="Actual">July 2013</primary_completion_date>
  <phase>Phase 2</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Parallel Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>Double (Participant, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Endoscopic Remission Defined as a Modified Baron Score of 0</measure>
    <time_frame>At week 8</time_frame>
    <description>The primary endpoint was the binary variable (&quot;responder&quot; vs. &quot;non-responder&quot;) where &quot;responders&quot; were the subjects with endoscopic remission (endoscopic mucosal healing) at Week 8, defined as a modified Baron score of 0. Subjects with a modified Baron score ≥1 were designated as &quot;non-responders&quot;.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Remission (Clinical and Endoscopic)</measure>
    <time_frame>At Week 8</time_frame>
    <description>Analysis of responders defined by a clinical component of: ulcerative colitis disease activity index (UC-DAI) score of less than or equal to 1 with 0 for rectal bleeding and 0 for stool frequency and an endoscopic component of: no mucosal friability (modified Baron score less than or equal to 1).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of Adverse Events (AEs)</measure>
    <time_frame>Week 0 to 10</time_frame>
    <description>Number of adverse events reported from the first trial-related activity, after the subject was exposed to the trial drug, until the end of the post-treatment follow-up period.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Clearance (CL) of rFXIII</measure>
    <time_frame>Samples were collected before and up to 72 hours after the first dose of rFXIII.</time_frame>
    <description>The volume of plasma cleared of the drug per unit time.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Maximum Concentration (Cmax) of rFXIII</measure>
    <time_frame>Samples were collected before and up to 72 hours after the first dose of rFXIII.</time_frame>
    <description>The peak plasma concentration of the drug after dose administration.</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">20</enrollment>
  <condition>Inflammation</condition>
  <condition>Ulcerative Colitis</condition>
  <arm_group>
    <arm_group_label>rFXIII</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>catridecacog</intervention_name>
    <description>Catridecacog (recombinant factor XIII, rFXIII) will be administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week at a dose of 35 IU/kg</description>
    <arm_group_label>rFXIII</arm_group_label>
    <other_name>recombinant factor XIII</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>placebo</intervention_name>
    <description>Placebo will be administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week.</description>
    <arm_group_label>Placebo</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Diagnosis of ulcerative colitis for at least 3 months from the time of initial&#xD;
             diagnosis. The diagnosis must have been confirmed by historical endoscopy and&#xD;
             histology. The severity of disease must have been confirmed by endoscopy at screening&#xD;
&#xD;
          -  Currently receiving oral aminosalicylates at approved doses of at least 2g/day for at&#xD;
             least 6 weeks. Doses of oral aminosalicylates should be stable for at least two weeks&#xD;
             prior to dosing (Visit 2)&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Diagnosis of UC limited to the rectum (ulcerative proctitis only, defined as less than&#xD;
             15 cm from the anal verge)&#xD;
&#xD;
          -  Requiring hospitalisation for current episode of severe UC&#xD;
&#xD;
          -  Use of biologic therapies for the treatment of UC within 12 weeks prior to dosing&#xD;
             (Visit 2)&#xD;
&#xD;
          -  Treatment failures to anti-tumour necrosis factor-alfa (anti-TNF-a) agents (e.g.&#xD;
             infliximab, adalimumab)&#xD;
&#xD;
          -  Use of immunosuppressant agents (e.g. azathioprine) within 4 weeks prior to dosing&#xD;
             (Visit 2)&#xD;
&#xD;
          -  Use of corticosteroids (oral, intravenous (i.v.), intramuscular (i.m.), or rectal )&#xD;
             within 14 days prior to dosing (Visit 2)&#xD;
&#xD;
          -  Use of enemas (corticosteroid or aminosalicylate) within 14 days prior to screening&#xD;
             (Visit 1)&#xD;
&#xD;
          -  Use of cyclosporine, tacrolimus, D-penicillamine, leflunomide, methotrexate,&#xD;
             mycophenolate mofetil, or thalidomide within 4 weeks prior to dosing (Visit 2)&#xD;
&#xD;
          -  Currently receiving total parenteral nutrition&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>64 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Global Clinical Registry (GCR, 1452)</last_name>
    <role>Study Director</role>
    <affiliation>Novo Nordisk A/S</affiliation>
  </overall_official>
  <location>
    <facility>
      <address>
        <city>Rousse</city>
        <zip>7002</zip>
        <country>Bulgaria</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Zagreb</city>
        <zip>10000</zip>
        <country>Croatia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Herlev</city>
        <zip>2730</zip>
        <country>Denmark</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Bekescsaba</city>
        <zip>H5600</zip>
        <country>Hungary</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Lodz</city>
        <zip>90-153</zip>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Nizhny Novgorod</city>
        <zip>60316</zip>
        <country>Russian Federation</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <address>
        <city>Kharkiv</city>
        <zip>61000</zip>
        <country>Ukraine</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Bulgaria</country>
    <country>Croatia</country>
    <country>Denmark</country>
    <country>Hungary</country>
    <country>Poland</country>
    <country>Russian Federation</country>
    <country>Ukraine</country>
  </location_countries>
  <link>
    <url>http://novonordisk-trials.com</url>
    <description>Clinical Trials at Novo Nordisk</description>
  </link>
  <verification_date>September 2014</verification_date>
  <study_first_submitted>October 9, 2012</study_first_submitted>
  <study_first_submitted_qc>October 10, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">October 15, 2012</study_first_posted>
  <results_first_submitted>July 10, 2014</results_first_submitted>
  <results_first_submitted_qc>July 10, 2014</results_first_submitted_qc>
  <results_first_posted type="Estimate">August 5, 2014</results_first_posted>
  <disposition_first_submitted>November 7, 2013</disposition_first_submitted>
  <disposition_first_submitted_qc>November 7, 2013</disposition_first_submitted_qc>
  <disposition_first_posted type="Estimate">December 2, 2013</disposition_first_posted>
  <last_update_submitted>September 22, 2014</last_update_submitted>
  <last_update_submitted_qc>September 22, 2014</last_update_submitted_qc>
  <last_update_posted type="Estimate">October 2, 2014</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Colitis</mesh_term>
    <mesh_term>Colitis, Ulcerative</mesh_term>
    <mesh_term>Inflammation</mesh_term>
    <mesh_term>Ulcer</mesh_term>
  </condition_browse>

  <clinical_results>

    <participant_flow>
      <recruitment_details>The trial was conducted at 12 sites in 6 countries as follows: Bulgaria (4 sites), Denmark (1 site), Hungary (1 site), Poland (4 sites), Russian Federation (1 site) and Ukraine (1 site).</recruitment_details>
      <pre_assignment_details>No pre-assignments were given for this trial.</pre_assignment_details>
      <group_list>
        <group group_id="P1">
          <title>rFXIII</title>
          <description>Recombinant factor XIII (rFXIII) was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) at a dose of 35 IU/kg. The doses were administered once every second week for a total of 4 doses.</description>
        </group>
        <group group_id="P2">
          <title>Placebo</title>
          <description>Placebo formulation was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week. The doses were administered for a total of 4 doses.</description>
        </group>
      </group_list>
      <period_list>
        <period>
          <title>Overall Study</title>
          <milestone_list>
            <milestone>
              <title>STARTED</title>
              <participants_list>
                <participants group_id="P1" count="14"/>
                <participants group_id="P2" count="6"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>Exposed</title>
              <participants_list>
                <participants group_id="P1" count="13">One subject was not exposed to rFXIII.</participants>
                <participants group_id="P2" count="5">One subject was not exposed to placebo.</participants>
              </participants_list>
            </milestone>
            <milestone>
              <title>Completers (With Baron Score at Week 8)</title>
              <participants_list>
                <participants group_id="P1" count="6"/>
                <participants group_id="P2" count="3"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="6"/>
                <participants group_id="P2" count="4"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="8"/>
                <participants group_id="P2" count="2"/>
              </participants_list>
            </milestone>
          </milestone_list>
          <drop_withdraw_reason_list>
            <drop_withdraw_reason>
              <title>Withdrawal Criteria</title>
              <participants_list>
                <participants group_id="P1" count="2"/>
                <participants group_id="P2" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
            <drop_withdraw_reason>
              <title>Unclassified</title>
              <participants_list>
                <participants group_id="P1" count="6"/>
                <participants group_id="P2" count="1"/>
              </participants_list>
            </drop_withdraw_reason>
          </drop_withdraw_reason_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <population>The demographics and baseline characteristics are presented for the Full analysis set (FAS) which included all randomised and treated subjects.</population>
      <group_list>
        <group group_id="B1">
          <title>rFXIII</title>
          <description>Recombinant factor XIII (rFXIII) was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) at a dose of 35 IU/kg. The doses were administered once every second week for a total of 4 doses.</description>
        </group>
        <group group_id="B2">
          <title>Placebo</title>
          <description>Placebo formulation was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week. The doses were administered for a total of 4 doses.</description>
        </group>
        <group group_id="B3">
          <title>Total</title>
          <description>Total of all reporting groups</description>
        </group>
      </group_list>
      <analyzed_list>
        <analyzed>
          <units>Participants</units>
          <scope>Overall</scope>
          <count_list>
            <count group_id="B1" value="13"/>
            <count group_id="B2" value="5"/>
            <count group_id="B3" value="18"/>
          </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="36.9" spread="10.6"/>
                    <measurement group_id="B2" value="45.0" spread="15.4"/>
                    <measurement group_id="B3" value="39.2" spread="12.2"/>
                  </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="5"/>
                    <measurement group_id="B2" value="4"/>
                    <measurement group_id="B3" value="9"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Male</title>
                  <measurement_list>
                    <measurement group_id="B1" value="8"/>
                    <measurement group_id="B2" value="1"/>
                    <measurement group_id="B3" value="9"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race/Ethnicity, Customized</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>Whites</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="13"/>
                    <measurement group_id="B2" value="5"/>
                    <measurement group_id="B3" value="18"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Body Mass Index</title>
          <units>kg/m^2</units>
          <param>Mean</param>
          <dispersion>Standard Deviation</dispersion>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="24.5" spread="3.2"/>
                    <measurement group_id="B2" value="25.6" spread="5.8"/>
                    <measurement group_id="B3" value="24.8" spread="3.9"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Endoscopic Remission Defined as a Modified Baron Score of 0</title>
        <description>The primary endpoint was the binary variable (&quot;responder&quot; vs. &quot;non-responder&quot;) where &quot;responders&quot; were the subjects with endoscopic remission (endoscopic mucosal healing) at Week 8, defined as a modified Baron score of 0. Subjects with a modified Baron score ≥1 were designated as &quot;non-responders&quot;.</description>
        <time_frame>At week 8</time_frame>
        <population>Full analysis set (FAS) included all randomised and treated subjects.</population>
        <group_list>
          <group group_id="O1">
            <title>rFXIII</title>
            <description>Recombinant factor XIII (rFXIII) was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) at a dose of 35 IU/kg. The doses were administered once every second week for a total of 4 doses.</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>Placebo formulation was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week. The doses were administered for a total of 4 doses.</description>
          </group>
        </group_list>
        <measure>
          <title>Endoscopic Remission Defined as a Modified Baron Score of 0</title>
          <description>The primary endpoint was the binary variable (&quot;responder&quot; vs. &quot;non-responder&quot;) where &quot;responders&quot; were the subjects with endoscopic remission (endoscopic mucosal healing) at Week 8, defined as a modified Baron score of 0. Subjects with a modified Baron score ≥1 were designated as &quot;non-responders&quot;.</description>
          <population>Full analysis set (FAS) included all randomised and treated subjects.</population>
          <units>Subjects</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="13"/>
                <count group_id="O2" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                    <measurement group_id="O2" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <analysis_list>
          <analysis>
            <group_id_list>
              <group_id>O1</group_id>
              <group_id>O2</group_id>
            </group_id_list>
            <groups_desc>A sample size of 90 subjects, with a 2:1 (active:placebo) randomization ratio, would ensure 80% power to detect a difference between active treatment and placebo at Week 8 with a 2-sided significance level of 10% based on a Fisher's exact test.</groups_desc>
            <non_inferiority_type>Superiority or Other</non_inferiority_type>
            <p_value>0.3056</p_value>
            <p_value_desc>p-value was not adjusted for multiple comparisons.</p_value_desc>
            <method>Regression, Logistic</method>
            <param_type>Odds Ratio (OR)</param_type>
            <param_value>0.16</param_value>
            <ci_percent>90</ci_percent>
            <ci_lower_limit>0.01</ci_lower_limit>
            <ci_upper_limit>3.05</ci_upper_limit>
          </analysis>
        </analysis_list>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Remission (Clinical and Endoscopic)</title>
        <description>Analysis of responders defined by a clinical component of: ulcerative colitis disease activity index (UC-DAI) score of less than or equal to 1 with 0 for rectal bleeding and 0 for stool frequency and an endoscopic component of: no mucosal friability (modified Baron score less than or equal to 1).</description>
        <time_frame>At Week 8</time_frame>
        <population>Full analysis set (FAS) included all randomised and treated subjects. Two subjects in the rFXIII group had no UC-DAI score at any visit, including baseline and they were excluded from the analysis.</population>
        <group_list>
          <group group_id="O1">
            <title>rFXIII</title>
            <description>Recombinant factor XIII (rFXIII) was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) at a dose of 35 IU/kg. The doses were administered once every second week for a total of 4 doses.</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>Placebo formulation was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week. The doses were administered for a total of 4 doses.</description>
          </group>
        </group_list>
        <measure>
          <title>Remission (Clinical and Endoscopic)</title>
          <description>Analysis of responders defined by a clinical component of: ulcerative colitis disease activity index (UC-DAI) score of less than or equal to 1 with 0 for rectal bleeding and 0 for stool frequency and an endoscopic component of: no mucosal friability (modified Baron score less than or equal to 1).</description>
          <population>Full analysis set (FAS) included all randomised and treated subjects. Two subjects in the rFXIII group had no UC-DAI score at any visit, including baseline and they were excluded from the analysis.</population>
          <units>Subjects</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="11"/>
                <count group_id="O2" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Number of Adverse Events (AEs)</title>
        <description>Number of adverse events reported from the first trial-related activity, after the subject was exposed to the trial drug, until the end of the post-treatment follow-up period.</description>
        <time_frame>Week 0 to 10</time_frame>
        <population>Safety analysis set included all randomised and treated subjects.</population>
        <group_list>
          <group group_id="O1">
            <title>rFXIII</title>
            <description>Recombinant factor XIII (rFXIII) was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) at a dose of 35 IU/kg. The doses were administered once every second week for a total of 4 doses.</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>Placebo formulation was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week. The doses were administered for a total of 4 doses.</description>
          </group>
        </group_list>
        <measure>
          <title>Number of Adverse Events (AEs)</title>
          <description>Number of adverse events reported from the first trial-related activity, after the subject was exposed to the trial drug, until the end of the post-treatment follow-up period.</description>
          <population>Safety analysis set included all randomised and treated subjects.</population>
          <units>events</units>
          <param>Number</param>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="13"/>
                <count group_id="O2" value="5"/>
              </count_list>
            </analyzed>
          </analyzed_list>
          <class_list>
            <class>
              <title>Adverse events</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="7"/>
                    <measurement group_id="O2" value="4"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Serious adverse events</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Severe adverse events</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="1"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Moderate adverse events</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Mild adverse events</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="6"/>
                    <measurement group_id="O2" value="2"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
            <class>
              <title>Fatal adverse events</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="O1" value="0"/>
                    <measurement group_id="O2" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Clearance (CL) of rFXIII</title>
        <description>The volume of plasma cleared of the drug per unit time.</description>
        <time_frame>Samples were collected before and up to 72 hours after the first dose of rFXIII.</time_frame>
        <population>It was not possible to obtain credible single dose pharmakokinetics (PK) for rFXIII in this trial due to a small number of subjects with required PK measurements and a spurious behaviour of individual PK profiles.</population>
        <group_list>
          <group group_id="O1">
            <title>rFXIII</title>
            <description>Recombinant factor XIII (rFXIII) was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) at a dose of 35 IU/kg. The doses were administered once every second week for a total of 4 doses.</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>Placebo formulation was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week. The doses were administered for a total of 4 doses.</description>
          </group>
        </group_list>
        <measure>
          <title>Clearance (CL) of rFXIII</title>
          <description>The volume of plasma cleared of the drug per unit time.</description>
          <population>It was not possible to obtain credible single dose pharmakokinetics (PK) for rFXIII in this trial due to a small number of subjects with required PK measurements and a spurious behaviour of individual PK profiles.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
      <outcome>
        <type>Secondary</type>
        <title>Maximum Concentration (Cmax) of rFXIII</title>
        <description>The peak plasma concentration of the drug after dose administration.</description>
        <time_frame>Samples were collected before and up to 72 hours after the first dose of rFXIII.</time_frame>
        <population>It was not possible to obtain credible single dose PK for rFXIII in this trial due to a small number of subjects with required PK measurements and a spurious behaviour of individual PK profiles.</population>
        <group_list>
          <group group_id="O1">
            <title>rFXIII</title>
            <description>Recombinant factor XIII (rFXIII) was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) at a dose of 35 IU/kg. The doses were administered once every second week for a total of 4 doses.</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>Placebo formulation was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week. The doses were administered for a total of 4 doses.</description>
          </group>
        </group_list>
        <measure>
          <title>Maximum Concentration (Cmax) of rFXIII</title>
          <description>The peak plasma concentration of the drug after dose administration.</description>
          <population>It was not possible to obtain credible single dose PK for rFXIII in this trial due to a small number of subjects with required PK measurements and a spurious behaviour of individual PK profiles.</population>
          <analyzed_list>
            <analyzed>
              <units>Participants</units>
              <scope>Measure</scope>
              <count_list>
                <count group_id="O1" value="0"/>
                <count group_id="O2" value="0"/>
              </count_list>
            </analyzed>
          </analyzed_list>
        </measure>
      </outcome>
    </outcome_list>

    <reported_events>
      <time_frame>Adverse events reported by the investigator from the first trial-related activity after the subject is exposed to the trial drug until the end of trial visit (Week 10).</time_frame>
      <desc>Safety analysis set was identical to the FAS which included all randomised and treated subjects.</desc>
      <group_list>
        <group group_id="E1">
          <title>rFXIII</title>
          <description>Recombinant factor XIII (rFXIII) was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) at a dose of 35 IU/kg. The doses were administered once every second week for a total of 4 doses.</description>
        </group>
        <group group_id="E2">
          <title>Placebo</title>
          <description>Placebo formulation was administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week. The doses were administered for a total of 4 doses.</description>
        </group>
      </group_list>
      <serious_events>
        <default_vocab>MedDRA (16.1)</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="1" subjects_at_risk="13"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Colitis ulcerative</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>5</frequency_threshold>
        <default_vocab>MedDRA (16.1)</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="2" subjects_at_risk="13"/>
                <counts group_id="E2" subjects_affected="2" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Blood and lymphatic system disorders</title>
            <event_list>
              <event>
                <sub_title>Anaemia</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Gastrointestinal disorders</title>
            <event_list>
              <event>
                <sub_title>Colitis ulcerative</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>General disorders</title>
            <event_list>
              <event>
                <sub_title>Asthenia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Fatigue</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Oedema peripheral</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Infections and infestations</title>
            <event_list>
              <event>
                <sub_title>Bronchitis</sub_title>
                <counts group_id="E1" events="0" subjects_affected="0" subjects_at_risk="13"/>
                <counts group_id="E2" events="1" subjects_affected="1" subjects_at_risk="5"/>
              </event>
              <event>
                <sub_title>Urinary tract infection</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Investigations</title>
            <event_list>
              <event>
                <sub_title>Blood creatinine increased</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Metabolism and nutrition disorders</title>
            <event_list>
              <event>
                <sub_title>Hypokalaemia</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
          <category>
            <title>Musculoskeletal and connective tissue disorders</title>
            <event_list>
              <event>
                <sub_title>Bone pain</sub_title>
                <counts group_id="E1" events="1" subjects_affected="1" subjects_at_risk="13"/>
                <counts group_id="E2" events="0" subjects_affected="0" subjects_at_risk="5"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>Novo Nordisk maintains the right to be informed of any Investigator plans for publication and to review any scientific paper, presentation, communication or other information concerning the investigation described in this protocol. Novo Nordisk reserves the right to prior review of such publications and to ask for delay of publication of individual site results until after the primary manuscript is accepted for publication.</restrictive_agreement>
    </certain_agreements>
    <limitations_and_caveats>The trial was terminated earlier than planned as the hypothesis of a correlation between low levels of FXIII and disease activity of UC could not be confirmed.</limitations_and_caveats>
    <point_of_contact>
      <name_or_title>Public Access to Clinical Trials</name_or_title>
      <organization>Novo Nordisk A/S</organization>
      <email>clinicaltrials@novonordisk.com</email>
    </point_of_contact>
  </clinical_results>
</clinical_study>


download date: ClinicalTrials.gov processed this data on September 20, 2023
link text: Link to the current ClinicalTrials.gov record.
url: https://clinicaltrials.gov/ct2/show/NCT01706159
org study id: NN8717-3946
secondary id: 2011-001568-22
secondary id: U1111-1120-3824
nct id: NCT01706159
lead sponsor: 
has dmc: No
This trial is conducted in Europe. The aim of the trial is to investigate the effect of
      recombinant factor XIII (rFXIII) administered to subjects with mild to moderate active
      ulcerative colitis (UC).
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Double (Participant, Investigator)
measure: Endoscopic Remission Defined as a Modified Baron Score of 0
time frame: At week 8
description: The primary endpoint was the binary variable ("responder" vs. "non-responder") where "responders" were the subjects with endoscopic remission (endoscopic mucosal healing) at Week 8, defined as a modified Baron score of 0. Subjects with a modified Baron score ≥1 were designated as "non-responders".
measure: Remission (Clinical and Endoscopic)
time frame: At Week 8
description: Analysis of responders defined by a clinical component of: ulcerative colitis disease activity index (UC-DAI) score of less than or equal to 1 with 0 for rectal bleeding and 0 for stool frequency and an endoscopic component of: no mucosal friability (modified Baron score less than or equal to 1).
measure: Number of Adverse Events (AEs)
time frame: Week 0 to 10
description: Number of adverse events reported from the first trial-related activity, after the subject was exposed to the trial drug, until the end of the post-treatment follow-up period.
measure: Clearance (CL) of rFXIII
time frame: Samples were collected before and up to 72 hours after the first dose of rFXIII.
description: The volume of plasma cleared of the drug per unit time.
measure: Maximum Concentration (Cmax) of rFXIII
time frame: Samples were collected before and up to 72 hours after the first dose of rFXIII.
description: The peak plasma concentration of the drug after dose administration.
arm group label: rFXIII
arm group type: Experimental
arm group label: Placebo
arm group type: Active Comparator
intervention type: Drug
intervention name: catridecacog
description: Catridecacog (recombinant factor XIII, rFXIII) will be administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week at a dose of 35 IU/kg
arm group label: rFXIII
other name: recombinant factor XIII
intervention type: Drug
intervention name: placebo
description: Placebo will be administered as intravenous (i.v.) injections (at an approximate rate of 1-2 mL/min) once every second week.
arm group label: Placebo
criteria: 
gender: All
minimum age: 18 Years
maximum age: 64 Years
healthy volunteers: No
last name: Global Clinical Registry (GCR, 1452)
role: Study Director
affiliation: Novo Nordisk A/S
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: Bulgaria
country: Croatia
country: Denmark
country: Hungary
country: Poland
country: Russian Federation
country: Ukraine
url: http://novonordisk-trials.com
description: Clinical Trials at Novo Nordisk
responsible party type: Sponsor
mesh term: Colitis
mesh term: Colitis, Ulcerative
mesh term: Inflammation
mesh term: Ulcer
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
limitations and caveats: The trial was terminated earlier than planned as the hypothesis of a correlation between low levels of FXIII and disease activity of UC could not be confirmed.
point of contact: 