<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/NCT01693653</url>
  </required_header>
  <id_info>
    <org_study_id>12-01182</org_study_id>
    <nct_id>NCT01693653</nct_id>
  </id_info>
  <brief_title>Tocilizumab for the Treatment of Behcet's Syndrome</brief_title>
  <official_title>Tocilizumab for the Treatment of Behcet's Syndrome</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>NYU Langone Health</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Genentech, Inc.</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>NYU Langone Health</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      This is a double-blind placebo controlled study targeting individuals with active Behcet's&#xD;
      Syndrome who have oral ulcers and are resistant (have not responded after 4 weeks) to&#xD;
      conventional treatments. Maximum allowable dose of colchicine (0.6mg twice a day) and stable&#xD;
      dose for 4 weeks before enrollment. Prednisone or equivalent (&lt; 10mg/day) permitted if dose&#xD;
      stable for 6 weeks prior to enrollment.&#xD;
&#xD;
      The study will investigate the safety of tocilizumab for this vasculitic condition in&#xD;
      addition to its efficacy.&#xD;
&#xD;
      The planned sample size is 30 participants per arm for a total of 60 participants. The study&#xD;
      would be for 3 months, with a safety follow up at 2 months after study termination.&#xD;
&#xD;
      Study participants will stay on their current treatments and either tocilizumab or placebo&#xD;
      infusions will be given every 4 weeks in addition. Patients will be randomized to Actemra IV&#xD;
      8mg/kg Q 4 weeks X 3 doses or placebo.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Behcet's syndrome is a vasculitis that causes oral and genital ulcerations, skin lesions, eye&#xD;
      disease and arthritis, in addition to vascular complications with thrombophlebitis,&#xD;
      thrombosis and rarely central nervous system involvement. IL-6 activity has been suggested in&#xD;
      the pathogenesis in some studies. Tocilizumab with its unique mode of action among biologic&#xD;
      agents may be a good candidate in this orphan disease.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    low enrollment&#xD;
  </why_stopped>
  <start_date>September 2012</start_date>
  <completion_date type="Actual">November 2013</completion_date>
  <primary_completion_date type="Actual">November 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>Triple (Participant, Care Provider, Investigator)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Primary Outcome</measure>
    <time_frame>9 months</time_frame>
    <description>The study was terminated. No data were collected for this Outcome Measure.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Genital Ulcers</measure>
    <time_frame>9 months</time_frame>
    <description>The study was terminated. No data were collected for this outcome measure.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Oral Ulcers</measure>
    <time_frame>9 months</time_frame>
    <description>The study was terminated. No data were collected for this outcome measure.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Treatment Failures</measure>
    <time_frame>9 months</time_frame>
    <description>The study was terminated. No data were collected for this outcome measure.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Oral Ulcer Pain</measure>
    <time_frame>9 months</time_frame>
    <description>The study was terminated. No data were collected for this outcome measure.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Gential Ulcer Pain</measure>
    <time_frame>9 months</time_frame>
    <description>The study was terminated. No data were collected for this outcome measure.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>BSAS</measure>
    <time_frame>9 months</time_frame>
    <description>The study was terminated. No data were collected for this outcome measure.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>MDHAQ</measure>
    <time_frame>9 months</time_frame>
    <description>The study was terminated. No data were collected for this outcome measure.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>BDCAF</measure>
    <time_frame>9 months</time_frame>
    <description>The study was terminated. No data were collected for this outcome measure.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Safety</measure>
    <time_frame>9 moths</time_frame>
    <description>The study was terminated. No data were collected for this outcome measure.</description>
  </secondary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">1</enrollment>
  <condition>Behcet Syndrome</condition>
  <arm_group>
    <arm_group_label>Tocilizumab</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
    <description>tocilizumab infusion every 4 weeks over 3 months</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_type>Placebo Comparator</arm_group_type>
    <description>placebo infusion 0.9% sodium chloride every 4 weeks over 3 months</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Tocilizumab</intervention_name>
    <description>Intravenous infusions every 4 weeks for 3 doses.</description>
    <arm_group_label>Placebo</arm_group_label>
    <arm_group_label>Tocilizumab</arm_group_label>
    <other_name>actemra</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Any patient with BS with at least 1 active oral ulcer resistant (have not responded&#xD;
             after 4 weeks to colchicine or local measures.&#xD;
&#xD;
          2. dose (maximum allowable 0.6mg twice a day ) stable for 4 weeks and prednisone or&#xD;
             equivalent (maximum dose &lt; 10mg/day) stable for 6 weeks prior to enrollment.&#xD;
&#xD;
          3. Patients must have Behcet's syndrome based on International Study Group criteria.&#xD;
&#xD;
        Patients will be included in the trial based on the following criteria:&#xD;
&#xD;
          -  Normal organ function, except if abnormal due to the disease under investigation such&#xD;
             as mucocutaneous involvement or joint involvement.&#xD;
&#xD;
          -  Men and women of reproductive potential must agree to use an acceptable method of&#xD;
             birth control during treatment and for twelve months after completion of treatment.&#xD;
&#xD;
          -  Subject has provided written informed consent.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Patients with eye, CNS, vascular involvement such as DVT, thrombosis, or aneurysms.&#xD;
&#xD;
          2. Patients who are currently being treated or have been exposed in the last 3 months to&#xD;
             other immunosuppressive medications (azathioprine, TNF inhibitors, methotrexate,&#xD;
             mycophenolate mofetil). Patients who are currently being treated or have been exposed&#xD;
             to Cyclosporine or cyclophosphamide in the past 6 months will be excluded).&#xD;
&#xD;
          3. Major surgery (including joint surgery) within 8 weeks prior to screening or planned&#xD;
             major surgery within 6 months following randomization.&#xD;
&#xD;
          4. Treatment with any investigational agent within 4 weeks (or 5 half-lives of the&#xD;
             investigational drug, whichever is longer) of screening.&#xD;
&#xD;
          5. Previous treatment with any cell-depleting therapies, including investigational agents&#xD;
             or approved therapies, some examples are CAMPATH, anti-CD4, anti-CD5, anti¬CD3,&#xD;
             anti-CD19 and anti-CD20 (please note exceptions above).&#xD;
&#xD;
          6. Treatment with intravenous gamma globulin, plasmapheresis or Prosorba column within 6&#xD;
             months of baseline.&#xD;
&#xD;
          7. Immunization with a live/attenuated vaccine within 4 weeks prior to baseline.&#xD;
&#xD;
          8. Previous treatment with TCZ (an exception to this criterion may be granted for single&#xD;
             dose exposure upon application to the sponsor on a case-by-case basis).&#xD;
&#xD;
          9. Any previous treatment with alkylating agents such as chlorambucil, or with total&#xD;
             lymphoid irradiation.&#xD;
&#xD;
         10. History of severe allergic or anaphylactic reactions to human, humanized or murine&#xD;
             monoclonal antibodies.&#xD;
&#xD;
         11. Evidence of serious uncontrolled concomitant cardiovascular, nervous system, pulmonary&#xD;
             (including obstructive pulmonary disease), renal, hepatic, endocrine (include&#xD;
             uncontrolled diabetes mellitus) or gastrointestinal disease (including complicated&#xD;
             diverticulitis, ulcerative colitis, or Crohn's disease.)&#xD;
&#xD;
         12. Current liver disease as determined by principal investigator unless related to&#xD;
             primary disease under investigation&#xD;
&#xD;
         13. Known active current or history of recurrent bacterial, viral, fungal, mycobacterial&#xD;
             or other infections (including but not limited to tuberculosis and atypical&#xD;
             mycobacterial disease, Hepatitis B and C, and herpes zoster, but excluding fungal&#xD;
             infections of nail beds).&#xD;
&#xD;
         14. Any major episode of infection requiring hospitalization or treatment with IV&#xD;
             antibiotics within 4 weeks of screening or oral antibiotics within 2 weeks prior to&#xD;
             screening.&#xD;
&#xD;
         15. Active TB requiring treatment within the previous 3 years. Patients should be screened&#xD;
             for latent TB and, if positive, treated following local practice guidelines prior to&#xD;
             initiating TCZ. Patients treated for tuberculosis with no recurrence in 3 years are&#xD;
             permitted. (Appendix 8)&#xD;
&#xD;
         16. Primary or secondary immunodeficiency (history of or currently active) unless related&#xD;
             to primary disease under investigation.&#xD;
&#xD;
         17. Evidence of active malignant disease, malignancies diagnosed within the previous 10&#xD;
             years (including hematological malignancies and solid tumors, except basal and&#xD;
             squamous cell carcinoma of the skin or carcinoma in situ of the cervix uteri that has&#xD;
             been excised and cured), or breast cancer diagnosed within the previous 20 years&#xD;
             unless related to primary disease under investigation.&#xD;
&#xD;
         18. Pregnant women or nursing (breast feeding) mothers.&#xD;
&#xD;
         19. Patients with reproductive potential not willing to use an effective method of&#xD;
             contraception.&#xD;
&#xD;
         20. History of alcohol, drug or chemical abuse within 1 year prior to screening.&#xD;
&#xD;
         21. Neuropathies or other conditions that might interfere with pain evaluation unless&#xD;
             related to primary disease under investigation.&#xD;
&#xD;
         22. Patients with lack of peripheral venous access.&#xD;
&#xD;
             Laboratory Exclusion criteria (at screening):&#xD;
&#xD;
         23. Serum creatinine &gt; 1.6 mg/dL (141 µmol/L) in female patients and &gt; 1.9 mg/dL (168&#xD;
             µmol/L) in male patients. Patients with serum creatinine values exceeding limits may&#xD;
             be eligible for the study if their estimated glomerular filtration rates (GFR) are&#xD;
             &gt;30.&#xD;
&#xD;
         24. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) &gt; 1.5 times upper&#xD;
             limit of normal (ULN)&#xD;
&#xD;
         25. Total Bilirubin &gt; ULN&#xD;
&#xD;
         26. Platelet count &lt; 100 x 109/L (100,000/mm3)&#xD;
&#xD;
         27. Hemoglobin &lt; 85 g/L (8.5 g/dL; 5.3 mmol/L)&#xD;
&#xD;
         28. White Blood Cells &lt; 3.0 x 109/L (3000/mm3)&#xD;
&#xD;
         29. Absolute Neutrophil Count &lt; 2.0 x 109/L (2000/mm3)&#xD;
&#xD;
         30. Absolute Lymphocyte Count &lt; 0.5 x 109/L (500/mm3)&#xD;
&#xD;
         31. Positive Hepatitis BsAg, or Hepatitis C antibody&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Yusuf Yazici, MD</last_name>
    <role>Principal Investigator</role>
    <affiliation>NYU Langone Medical Center</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>NYU Center for Musculoskeletal Care</name>
      <address>
        <city>New York</city>
        <state>New York</state>
        <zip>10016</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>February 2019</verification_date>
  <study_first_submitted>September 19, 2012</study_first_submitted>
  <study_first_submitted_qc>September 24, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">September 26, 2012</study_first_posted>
  <results_first_submitted>October 14, 2015</results_first_submitted>
  <results_first_submitted_qc>April 26, 2016</results_first_submitted_qc>
  <results_first_posted type="Estimate">June 2, 2016</results_first_posted>
  <last_update_submitted>February 13, 2019</last_update_submitted>
  <last_update_submitted_qc>February 13, 2019</last_update_submitted_qc>
  <last_update_posted type="Actual">February 15, 2019</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Behcet's syndrome</keyword>
  <keyword>oral ulcers</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Behcet Syndrome</mesh_term>
    <mesh_term>Syndrome</mesh_term>
  </condition_browse>

  <clinical_results>

    <participant_flow>
      <group_list>
        <group group_id="P1">
          <title>Tocilizumab</title>
          <description>tocilizumab infusion every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
        </group>
        <group group_id="P2">
          <title>Placebo</title>
          <description>placebo infusion 0.9% sodium chloride every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 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="1"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="1"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </milestone>
            <milestone>
              <title>NOT COMPLETED</title>
              <participants_list>
                <participants group_id="P1" count="0"/>
                <participants group_id="P2" count="0"/>
              </participants_list>
            </milestone>
          </milestone_list>
        </period>
      </period_list>
    </participant_flow>

    <baseline>
      <group_list>
        <group group_id="B1">
          <title>Tocilizumab</title>
          <description>tocilizumab infusion every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
        </group>
        <group group_id="B2">
          <title>Placebo</title>
          <description>placebo infusion 0.9% sodium chloride every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 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="1"/>
            <count group_id="B2" value="0"/>
            <count group_id="B3" value="1"/>
          </count_list>
        </analyzed>
      </analyzed_list>
      <measure_list>
        <measure>
          <title>Age</title>
          <units>Participants</units>
          <param>Count of Participants</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <title>&lt;=18 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
                <category>
                  <title>Between 18 and 65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B3" value="1"/>
                  </measurement_list>
                </category>
                <category>
                  <title>&gt;=65 years</title>
                  <measurement_list>
                    <measurement group_id="B1" value="0"/>
                    <measurement group_id="B3" value="0"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Sex/Gender, Customized</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B3" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Race/Ethnicity, Customized</title>
          <units>particpants</units>
          <param>Number</param>
          <class_list>
            <class>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B3" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
        <measure>
          <title>Region of Enrollment</title>
          <units>participants</units>
          <param>Number</param>
          <class_list>
            <class>
              <title>United States</title>
              <category_list>
                <category>
                  <measurement_list>
                    <measurement group_id="B1" value="1"/>
                    <measurement group_id="B3" value="1"/>
                  </measurement_list>
                </category>
              </category_list>
            </class>
          </class_list>
        </measure>
      </measure_list>
    </baseline>

    <outcome_list>
      <outcome>
        <type>Primary</type>
        <title>Primary Outcome</title>
        <description>The study was terminated. No data were collected for this Outcome Measure.</description>
        <time_frame>9 months</time_frame>
        <population>The study was terminated. No data were collected for this Outcome Measure.</population>
        <group_list>
          <group group_id="O1">
            <title>Tocilizumab</title>
            <description>tocilizumab infusion every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>placebo infusion 0.9% sodium chloride every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
        </group_list>
        <measure>
          <title>Primary Outcome</title>
          <description>The study was terminated. No data were collected for this Outcome Measure.</description>
          <population>The study was terminated. No data were collected for this Outcome Measure.</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>Genital Ulcers</title>
        <description>The study was terminated. No data were collected for this outcome measure.</description>
        <time_frame>9 months</time_frame>
        <population>Terminated due to low enrollments. Data for 1 subject not analyzed.</population>
        <group_list>
          <group group_id="O1">
            <title>Tocilizumab</title>
            <description>tocilizumab infusion every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>placebo infusion 0.9% sodium chloride every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
        </group_list>
        <measure>
          <title>Genital Ulcers</title>
          <description>The study was terminated. No data were collected for this outcome measure.</description>
          <population>Terminated due to low enrollments. Data for 1 subject not analyzed.</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>Oral Ulcers</title>
        <description>The study was terminated. No data were collected for this outcome measure.</description>
        <time_frame>9 months</time_frame>
        <population>Terminated due to low enrollments. Data for 1 subject not analyzed.</population>
        <group_list>
          <group group_id="O1">
            <title>Tocilizumab</title>
            <description>tocilizumab infusion every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>placebo infusion 0.9% sodium chloride every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
        </group_list>
        <measure>
          <title>Oral Ulcers</title>
          <description>The study was terminated. No data were collected for this outcome measure.</description>
          <population>Terminated due to low enrollments. Data for 1 subject not analyzed.</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>Treatment Failures</title>
        <description>The study was terminated. No data were collected for this outcome measure.</description>
        <time_frame>9 months</time_frame>
        <population>Terminated due to low enrollments. Data for 1 subject not analyzed.</population>
        <group_list>
          <group group_id="O1">
            <title>Tocilizumab</title>
            <description>tocilizumab infusion every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>placebo infusion 0.9% sodium chloride every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
        </group_list>
        <measure>
          <title>Treatment Failures</title>
          <description>The study was terminated. No data were collected for this outcome measure.</description>
          <population>Terminated due to low enrollments. Data for 1 subject not analyzed.</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>Oral Ulcer Pain</title>
        <description>The study was terminated. No data were collected for this outcome measure.</description>
        <time_frame>9 months</time_frame>
        <population>Terminated due to low enrollments. Data for 1 subject not analyzed.</population>
        <group_list>
          <group group_id="O1">
            <title>Tocilizumab</title>
            <description>tocilizumab infusion every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>placebo infusion 0.9% sodium chloride every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
        </group_list>
        <measure>
          <title>Oral Ulcer Pain</title>
          <description>The study was terminated. No data were collected for this outcome measure.</description>
          <population>Terminated due to low enrollments. Data for 1 subject not analyzed.</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>Gential Ulcer Pain</title>
        <description>The study was terminated. No data were collected for this outcome measure.</description>
        <time_frame>9 months</time_frame>
        <population>Terminated due to low enrollments. Data for 1 subject not analyzed.</population>
        <group_list>
          <group group_id="O1">
            <title>Tocilizumab</title>
            <description>tocilizumab infusion every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>placebo infusion 0.9% sodium chloride every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
        </group_list>
        <measure>
          <title>Gential Ulcer Pain</title>
          <description>The study was terminated. No data were collected for this outcome measure.</description>
          <population>Terminated due to low enrollments. Data for 1 subject not analyzed.</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>BSAS</title>
        <description>The study was terminated. No data were collected for this outcome measure.</description>
        <time_frame>9 months</time_frame>
        <population>Terminated due to low enrollments. Data for 1 subject not analyzed.</population>
        <group_list>
          <group group_id="O1">
            <title>Tocilizumab</title>
            <description>tocilizumab infusion every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>placebo infusion 0.9% sodium chloride every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
        </group_list>
        <measure>
          <title>BSAS</title>
          <description>The study was terminated. No data were collected for this outcome measure.</description>
          <population>Terminated due to low enrollments. Data for 1 subject not analyzed.</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>MDHAQ</title>
        <description>The study was terminated. No data were collected for this outcome measure.</description>
        <time_frame>9 months</time_frame>
        <population>Terminated due to low enrollments. Data for 1 subject not analyzed.</population>
        <group_list>
          <group group_id="O1">
            <title>Tocilizumab</title>
            <description>tocilizumab infusion every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>placebo infusion 0.9% sodium chloride every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
        </group_list>
        <measure>
          <title>MDHAQ</title>
          <description>The study was terminated. No data were collected for this outcome measure.</description>
          <population>Terminated due to low enrollments. Data for 1 subject not analyzed.</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>BDCAF</title>
        <description>The study was terminated. No data were collected for this outcome measure.</description>
        <time_frame>9 months</time_frame>
        <population>Terminated due to low enrollments. Data for 1 subject not analyzed.</population>
        <group_list>
          <group group_id="O1">
            <title>Tocilizumab</title>
            <description>tocilizumab infusion every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>placebo infusion 0.9% sodium chloride every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
        </group_list>
        <measure>
          <title>BDCAF</title>
          <description>The study was terminated. No data were collected for this outcome measure.</description>
          <population>Terminated due to low enrollments. Data for 1 subject not analyzed.</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>Safety</title>
        <description>The study was terminated. No data were collected for this outcome measure.</description>
        <time_frame>9 moths</time_frame>
        <population>Terminated due to low enrollments. Data for 1 subject not analyzed.</population>
        <group_list>
          <group group_id="O1">
            <title>Tocilizumab</title>
            <description>tocilizumab infusion every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
          <group group_id="O2">
            <title>Placebo</title>
            <description>placebo infusion 0.9% sodium chloride every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
          </group>
        </group_list>
        <measure>
          <title>Safety</title>
          <description>The study was terminated. No data were collected for this outcome measure.</description>
          <population>Terminated due to low enrollments. Data for 1 subject not analyzed.</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>
      <desc>serious and non serious adverse events were not observed.</desc>
      <group_list>
        <group group_id="E1">
          <title>Tocilizumab</title>
          <description>tocilizumab infusion every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
        </group>
        <group group_id="E2">
          <title>Placebo</title>
          <description>placebo infusion 0.9% sodium chloride every 4 weeks over 3 months&#xD;
Tocilizumab: Intravenous infusions every 4 weeks for 3 doses.</description>
        </group>
      </group_list>
      <serious_events>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, all-cause mortality</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="0"/>
              </event>
              <event>
                <sub_title>Total, serious adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="0"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </serious_events>
      <other_events>
        <frequency_threshold>0</frequency_threshold>
        <category_list>
          <category>
            <title>Total</title>
            <event_list>
              <event>
                <sub_title>Total, other adverse events</sub_title>
                <counts group_id="E1" subjects_affected="0" subjects_at_risk="1"/>
                <counts group_id="E2" subjects_affected="0" subjects_at_risk="0"/>
              </event>
            </event_list>
          </category>
        </category_list>
      </other_events>
    </reported_events>

    <certain_agreements>
      <pi_employee>Principal Investigators are NOT employed by the organization sponsoring the study.</pi_employee>
      <restrictive_agreement>There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed. </restrictive_agreement>
    </certain_agreements>
    <point_of_contact>
      <name_or_title>Yusuf Yazici</name_or_title>
      <organization>NYU Langone Medical Center, Center for Musculoskeletal Care</organization>
      <phone>646-501-7400</phone>
      <email>yusuf.yazici@nyumc.org</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/NCT01693653
org study id: 12-01182
nct id: NCT01693653
lead sponsor: 
collaborator: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
This is a double-blind placebo controlled study targeting individuals with active Behcet's
      Syndrome who have oral ulcers and are resistant (have not responded after 4 weeks) to
      conventional treatments. Maximum allowable dose of colchicine (0.6mg twice a day) and stable
      dose for 4 weeks before enrollment. Prednisone or equivalent (< 10mg/day) permitted if dose
      stable for 6 weeks prior to enrollment.

      The study will investigate the safety of tocilizumab for this vasculitic condition in
      addition to its efficacy.

      The planned sample size is 30 participants per arm for a total of 60 participants. The study
      would be for 3 months, with a safety follow up at 2 months after study termination.

      Study participants will stay on their current treatments and either tocilizumab or placebo
      infusions will be given every 4 weeks in addition. Patients will be randomized to Actemra IV
      8mg/kg Q 4 weeks X 3 doses or placebo.
Behcet's syndrome is a vasculitis that causes oral and genital ulcerations, skin lesions, eye
      disease and arthritis, in addition to vascular complications with thrombophlebitis,
      thrombosis and rarely central nervous system involvement. IL-6 activity has been suggested in
      the pathogenesis in some studies. Tocilizumab with its unique mode of action among biologic
      agents may be a good candidate in this orphan disease.
allocation: Randomized
intervention model: Parallel Assignment
primary purpose: Treatment
masking: Triple (Participant, Care Provider, Investigator)
measure: Primary Outcome
time frame: 9 months
description: The study was terminated. No data were collected for this Outcome Measure.
measure: Genital Ulcers
time frame: 9 months
description: The study was terminated. No data were collected for this outcome measure.
measure: Oral Ulcers
time frame: 9 months
description: The study was terminated. No data were collected for this outcome measure.
measure: Treatment Failures
time frame: 9 months
description: The study was terminated. No data were collected for this outcome measure.
measure: Oral Ulcer Pain
time frame: 9 months
description: The study was terminated. No data were collected for this outcome measure.
measure: Gential Ulcer Pain
time frame: 9 months
description: The study was terminated. No data were collected for this outcome measure.
measure: BSAS
time frame: 9 months
description: The study was terminated. No data were collected for this outcome measure.
measure: MDHAQ
time frame: 9 months
description: The study was terminated. No data were collected for this outcome measure.
measure: BDCAF
time frame: 9 months
description: The study was terminated. No data were collected for this outcome measure.
measure: Safety
time frame: 9 moths
description: The study was terminated. No data were collected for this outcome measure.
arm group label: Tocilizumab
arm group type: Active Comparator
description: tocilizumab infusion every 4 weeks over 3 months
arm group label: Placebo
arm group type: Placebo Comparator
description: placebo infusion 0.9% sodium chloride every 4 weeks over 3 months
intervention type: Drug
intervention name: Tocilizumab
description: Intravenous infusions every 4 weeks for 3 doses.
arm group label: Placebo
arm group label: Tocilizumab
other name: actemra
criteria: 
gender: All
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Yusuf Yazici, MD
role: Principal Investigator
affiliation: NYU Langone Medical Center
facility: 
country: United States
responsible party type: Sponsor
mesh term: Behcet Syndrome
mesh term: Syndrome
participant flow: 
baseline: 
outcome list: 
reported events: 
certain agreements: 
point of contact: 