<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/NCT03754790</url>
  </required_header>
  <id_info>
    <org_study_id>LTE15174</org_study_id>
    <secondary_id>2018-002880-25</secondary_id>
    <secondary_id>U1111-1210-0018</secondary_id>
    <nct_id>NCT03754790</nct_id>
  </id_info>
  <brief_title>Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX</brief_title>
  <acronym>ATLAS-OLE</acronym>
  <official_title>An Open-label, Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Genzyme, a Sanofi Company</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Sanofi</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>
    <is_us_export>No</is_us_export>
  </oversight_info>
  <brief_summary>
    <textblock>
      Primary Objective:&#xD;
&#xD;
      To characterize the long-term safety and tolerability of fitusiran&#xD;
&#xD;
      Secondary Objectives:&#xD;
&#xD;
        -  To characterize the efficacy and long-term efficacy of fitusiran as assessed by the&#xD;
           frequency of:&#xD;
&#xD;
             -  Bleeding episodes&#xD;
&#xD;
             -  Spontaneous bleeding episodes&#xD;
&#xD;
             -  Joint bleeding episodes&#xD;
&#xD;
        -  To characterize the effects of fitusiran on health-related quality of life (HRQOL)&#xD;
           measures in participants ≥17 years of age&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      The estimated total time on the study for a participant up to 90 months (including screening,&#xD;
      treatment period and safety follow up period).&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">January 9, 2019</start_date>
  <completion_date type="Anticipated">December 18, 2026</completion_date>
  <primary_completion_date type="Anticipated">December 18, 2026</primary_completion_date>
  <phase>Phase 3</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Number of participants with treatment emergent adverse events (TEAEs)</measure>
    <time_frame>from study baseline (day 1) up to maximum 88 months</time_frame>
    <description>The number of participants experiencing any TEAEs, serious TEAEs, discontinuation due to TEAEs and death will be reported</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Annualized bleeding rate (ABR)</measure>
    <time_frame>from first ever dose of fitusiran (day 1) up to maximum 78 months</time_frame>
    <description>Annualized bleeding rate (ABR) in the treatment period</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Annualized spontaneous bleeding rate</measure>
    <time_frame>from first ever dose of fitusiran (day 1) up to maximum 78 months</time_frame>
    <description>Annualized spontaneous bleeding rate in the treatment period</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Annualized joint bleeding rate</measure>
    <time_frame>from first ever dose of fitusiran (day 1) up to maximum 78 months</time_frame>
    <description>Annualized joint bleeding rate in the treatment period</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Changes in Haem-A-quality of life (QoL) score</measure>
    <time_frame>from first ever dose of fitusiran (day 1) up to maximum 78 months</time_frame>
    <description>Change in Haem A QoL physical health score and total score in the treatment period (in participants ≥17 years of age) from first ever dose of fitusiran (day 1) to month 48</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">355</enrollment>
  <condition>Hemophilia</condition>
  <arm_group>
    <arm_group_label>Fitusiran</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Participants will be administered fitusiran as a subcutaneous injection once monthly or every other month for up to 48 months post initiation of modified IMP dose/frequency or until fitusiran becomes commercially available, whichever comes first.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Fitusiran</intervention_name>
    <description>Pharmaceutical form:solution for injection Route of administration: subcutaneous</description>
    <arm_group_label>Fitusiran</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion criteria :&#xD;
&#xD;
          -  Participant must be at least 12 years of age inclusive, at the time of signing the&#xD;
             informed consent&#xD;
&#xD;
          -  Participants with severe hemophilia A or B who have completed a Phase 3 fitusiran&#xD;
             clinical trial&#xD;
&#xD;
          -  Male&#xD;
&#xD;
          -  Capable of giving signed informed consent which includes compliance with the&#xD;
             requirements and restrictions listed in the informed consent form (ICF) and in the&#xD;
             protocol. In countries where legal age of majority is above 18 years, a specific ICF&#xD;
             must also be signed by the participant's legally authorized representative&#xD;
&#xD;
        Exclusion criteria:&#xD;
&#xD;
          -  Completion of a surgical procedure within 14 days prior to screening, or currently&#xD;
             receiving additional factor concentrate or BPA infusion for postoperative hemostasis&#xD;
&#xD;
          -  Current participation in immune tolerance induction treatment (ITI)&#xD;
&#xD;
          -  Current use of factor concentrates or bypassing agents (BPAs) as regularly&#xD;
             administered prophylaxis designed to prevent spontaneous bleeding episodes except for&#xD;
             participants requiring factor concentrates or BPAs prophylaxis during the study dosing&#xD;
             pause period&#xD;
&#xD;
          -  Use of compounds other than factor concentrates or BPAs for hemophilia treatment&#xD;
&#xD;
          -  Current or prior participation in a gene therapy trial&#xD;
&#xD;
          -  Alanine aminotransferase (ALT) and/or Aspartate aminotransferase (AST) &gt;1.5 × upper&#xD;
             limit of normal reference range (ULN) for patients who are naïve to fitusiran at study&#xD;
             start; ALT and/or AST &gt; 5 x ULN for patients who were in the fitusiran arm in the&#xD;
             parent study&#xD;
&#xD;
          -  Additional exclusions for participants not currently participating in a fitusiran&#xD;
             trial at the time of enrollment in the lower dose cohort:&#xD;
&#xD;
               -  Clinically significant liver disease&#xD;
&#xD;
               -  History of arterial or venous thromboembolism&#xD;
&#xD;
        The above information is not intended to contain all considerations relevant to a patient's&#xD;
        potential participation in a clinical trial.&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <minimum_age>12 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Clinical Sciences &amp; Operations</last_name>
    <role>Study Director</role>
    <affiliation>Sanofi</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Investigational Site Number :8400009</name>
      <address>
        <city>Phoenix</city>
        <state>Arizona</state>
        <zip>85016</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :8400019</name>
      <address>
        <city>Los Angeles</city>
        <state>California</state>
        <zip>90027</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :8400016</name>
      <address>
        <city>Orange</city>
        <state>California</state>
        <zip>92868</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :8400008</name>
      <address>
        <city>Jacksonville</city>
        <state>Florida</state>
        <zip>32207</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :8400002</name>
      <address>
        <city>Tampa</city>
        <state>Florida</state>
        <zip>33607</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :8400001</name>
      <address>
        <city>Chicago</city>
        <state>Illinois</state>
        <zip>60612</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :8400011</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02114</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :8400012</name>
      <address>
        <city>Ann Arbor</city>
        <state>Michigan</state>
        <zip>48109</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :8400007</name>
      <address>
        <city>Las Vegas</city>
        <state>Nevada</state>
        <zip>89135</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :8400006</name>
      <address>
        <city>Akron</city>
        <state>Ohio</state>
        <zip>44308</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :0360001</name>
      <address>
        <city>Camperdown</city>
        <state>New South Wales</state>
        <zip>2050</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :0360003</name>
      <address>
        <city>Prahran</city>
        <state>Victoria</state>
        <zip>3181</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :0360002</name>
      <address>
        <city>Murdoch</city>
        <state>Western Australia</state>
        <zip>6961</zip>
        <country>Australia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1240001</name>
      <address>
        <city>Montreal</city>
        <state>Quebec</state>
        <zip>H1T 2M4</zip>
        <country>Canada</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1560004</name>
      <address>
        <city>Beijing</city>
        <zip>100045</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1560007</name>
      <address>
        <city>Beijing</city>
        <zip>100730</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1560014</name>
      <address>
        <city>Changsha</city>
        <zip>410008</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1560009</name>
      <address>
        <city>Chengdu</city>
        <zip>610091</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1560002</name>
      <address>
        <city>Guangzhou</city>
        <zip>510515</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1560011</name>
      <address>
        <city>Guiyang</city>
        <zip>550004</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1560005</name>
      <address>
        <city>Hangzhou</city>
        <zip>89147</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1560008</name>
      <address>
        <city>Jinan</city>
        <zip>250013</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1560012</name>
      <address>
        <city>Kunming</city>
        <zip>650000</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1560013</name>
      <address>
        <city>Lanzhou</city>
        <zip>730000</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1560010</name>
      <address>
        <city>Qingdao</city>
        <zip>266555</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1560003</name>
      <address>
        <city>Shanghai</city>
        <zip>200025</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1560006</name>
      <address>
        <city>Suzhou</city>
        <zip>215006</zip>
        <country>China</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :2080001</name>
      <address>
        <city>Copenhagen</city>
        <zip>2100</zip>
        <country>Denmark</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :2500002</name>
      <address>
        <city>Lyon</city>
        <zip>69677</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :2500003</name>
      <address>
        <city>Paris</city>
        <zip>75015</zip>
        <country>France</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :2760001</name>
      <address>
        <city>Berlin</city>
        <zip>10249</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :2760003</name>
      <address>
        <city>Leipzig</city>
        <zip>04103</zip>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :3480002</name>
      <address>
        <city>Budapest</city>
        <zip>1134</zip>
        <country>Hungary</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :3560001</name>
      <address>
        <city>Bangalore</city>
        <zip>560034</zip>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :3560005</name>
      <address>
        <city>Lucknow</city>
        <zip>226003</zip>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :3560007</name>
      <address>
        <city>Mumbai</city>
        <zip>400 022</zip>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :3560002</name>
      <address>
        <city>Pune</city>
        <zip>411001</zip>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :3560010</name>
      <address>
        <city>Pune</city>
        <zip>411004</zip>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :3560004</name>
      <address>
        <city>Vellore</city>
        <zip>632004</zip>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :3720001</name>
      <address>
        <city>Crumlin</city>
        <state>Dublin</state>
        <zip>12</zip>
        <country>Ireland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :3720002</name>
      <address>
        <city>Dublin</city>
        <zip>DUBLIN 8</zip>
        <country>Ireland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :3760001</name>
      <address>
        <city>Ramat Gan</city>
        <zip>52662</zip>
        <country>Israel</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :3800002</name>
      <address>
        <city>Milano</city>
        <state>Lombardia</state>
        <zip>20121</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :3800004</name>
      <address>
        <city>Padua</city>
        <state>Veneto</state>
        <zip>35128</zip>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :3920002</name>
      <address>
        <city>Nishinomiya</city>
        <state>Hyogo</state>
        <zip>663-8501</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :3920005</name>
      <address>
        <city>Isehara</city>
        <state>Kanagawa</state>
        <zip>2591193</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :3920003</name>
      <address>
        <city>Kitakyushu</city>
        <zip>8078555</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :3920001</name>
      <address>
        <city>Nagoya</city>
        <zip>4668560</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :3920004</name>
      <address>
        <city>Saitama</city>
        <zip>330-8777</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :3920008</name>
      <address>
        <city>Tokyo</city>
        <zip>1600023</zip>
        <country>Japan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :4100001</name>
      <address>
        <city>Busan</city>
        <state>Busan-gwangyeoksi</state>
        <zip>602-739</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :4100002</name>
      <address>
        <city>Daejeon</city>
        <state>Daejeon-gwangyeoksi</state>
        <zip>35233</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :4100003</name>
      <address>
        <city>Seoul</city>
        <state>Seoul-teukbyeolsi</state>
        <zip>03722</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :4100004</name>
      <address>
        <city>Seoul</city>
        <zip>3722</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :4580003</name>
      <address>
        <city>Ampang</city>
        <zip>68000</zip>
        <country>Malaysia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :4580001</name>
      <address>
        <city>Johor Bahru</city>
        <zip>80100</zip>
        <country>Malaysia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :4580002</name>
      <address>
        <city>Kota Kinabalu</city>
        <zip>88586</zip>
        <country>Malaysia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :7100002</name>
      <address>
        <city>Port Elizabeth</city>
        <zip>6001</zip>
        <country>South Africa</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1580002</name>
      <address>
        <city>Changhua</city>
        <zip>500</zip>
        <country>Taiwan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1580008</name>
      <address>
        <city>Taichung</city>
        <zip>40201</zip>
        <country>Taiwan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1580004</name>
      <address>
        <city>Taichung</city>
        <zip>40705</zip>
        <country>Taiwan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1580003</name>
      <address>
        <city>Taipei</city>
        <zip>100</zip>
        <country>Taiwan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1580001</name>
      <address>
        <city>Taipei</city>
        <zip>110</zip>
        <country>Taiwan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1580005</name>
      <address>
        <city>Taipei</city>
        <zip>112</zip>
        <country>Taiwan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :1580007</name>
      <address>
        <city>Taoyuan City</city>
        <zip>33305</zip>
        <country>Taiwan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :7920002</name>
      <address>
        <city>Adana</city>
        <zip>01130</zip>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :7920004</name>
      <address>
        <city>Akdeniz</city>
        <zip>07059</zip>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :7920012</name>
      <address>
        <city>Bornova</city>
        <zip>35100</zip>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :7920006</name>
      <address>
        <city>Gaziantep</city>
        <zip>27100</zip>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :7920005</name>
      <address>
        <city>Istanbul</city>
        <zip>34093</zip>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :7920003</name>
      <address>
        <city>Izmir</city>
        <zip>TR-35100</zip>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :7920008</name>
      <address>
        <city>Kayseri</city>
        <zip>38039</zip>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :7920007</name>
      <address>
        <city>Samsun</city>
        <zip>55200</zip>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :8040001</name>
      <address>
        <city>Kyiv</city>
        <zip>04060</zip>
        <country>Ukraine</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :8040003</name>
      <address>
        <city>Kyiv</city>
        <zip>1135</zip>
        <country>Ukraine</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :8040002</name>
      <address>
        <city>Lviv</city>
        <zip>79044</zip>
        <country>Ukraine</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :8040004</name>
      <address>
        <city>Mykolaiv</city>
        <zip>54058</zip>
        <country>Ukraine</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :8260001</name>
      <address>
        <city>London</city>
        <state>London, City Of</state>
        <zip>SE1 9RT</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigational Site Number :8260004</name>
      <address>
        <city>Glasgow</city>
        <zip>G4 0SF</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Australia</country>
    <country>Canada</country>
    <country>China</country>
    <country>Denmark</country>
    <country>France</country>
    <country>Germany</country>
    <country>Hungary</country>
    <country>India</country>
    <country>Ireland</country>
    <country>Israel</country>
    <country>Italy</country>
    <country>Japan</country>
    <country>Korea, Republic of</country>
    <country>Malaysia</country>
    <country>South Africa</country>
    <country>Taiwan</country>
    <country>Turkey</country>
    <country>Ukraine</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <verification_date>December 2022</verification_date>
  <study_first_submitted>November 25, 2018</study_first_submitted>
  <study_first_submitted_qc>November 26, 2018</study_first_submitted_qc>
  <study_first_posted type="Actual">November 27, 2018</study_first_posted>
  <last_update_submitted>December 12, 2022</last_update_submitted>
  <last_update_submitted_qc>December 12, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">December 14, 2022</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>Hemophilia A</mesh_term>
  </condition_browse>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_description>Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org</ipd_description>
  </patient_data>
  <!-- Results have not yet been posted for this study                                          -->
</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/NCT03754790
org study id: LTE15174
secondary id: 2018-002880-25
secondary id: U1111-1210-0018
nct id: NCT03754790
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
is us export: No
Primary Objective:

      To characterize the long-term safety and tolerability of fitusiran

      Secondary Objectives:

        -  To characterize the efficacy and long-term efficacy of fitusiran as assessed by the
           frequency of:

             -  Bleeding episodes

             -  Spontaneous bleeding episodes

             -  Joint bleeding episodes

        -  To characterize the effects of fitusiran on health-related quality of life (HRQOL)
           measures in participants ≥17 years of age
The estimated total time on the study for a participant up to 90 months (including screening,
      treatment period and safety follow up period).
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Number of participants with treatment emergent adverse events (TEAEs)
time frame: from study baseline (day 1) up to maximum 88 months
description: The number of participants experiencing any TEAEs, serious TEAEs, discontinuation due to TEAEs and death will be reported
measure: Annualized bleeding rate (ABR)
time frame: from first ever dose of fitusiran (day 1) up to maximum 78 months
description: Annualized bleeding rate (ABR) in the treatment period
measure: Annualized spontaneous bleeding rate
time frame: from first ever dose of fitusiran (day 1) up to maximum 78 months
description: Annualized spontaneous bleeding rate in the treatment period
measure: Annualized joint bleeding rate
time frame: from first ever dose of fitusiran (day 1) up to maximum 78 months
description: Annualized joint bleeding rate in the treatment period
measure: Changes in Haem-A-quality of life (QoL) score
time frame: from first ever dose of fitusiran (day 1) up to maximum 78 months
description: Change in Haem A QoL physical health score and total score in the treatment period (in participants ≥17 years of age) from first ever dose of fitusiran (day 1) to month 48
arm group label: Fitusiran
arm group type: Experimental
description: Participants will be administered fitusiran as a subcutaneous injection once monthly or every other month for up to 48 months post initiation of modified IMP dose/frequency or until fitusiran becomes commercially available, whichever comes first.
intervention type: Drug
intervention name: Fitusiran
description: Pharmaceutical form:solution for injection Route of administration: subcutaneous
arm group label: Fitusiran
criteria: 
gender: Male
minimum age: 12 Years
maximum age: N/A
healthy volunteers: No
last name: Clinical Sciences & Operations
role: Study Director
affiliation: Sanofi
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: Australia
country: Canada
country: China
country: Denmark
country: France
country: Germany
country: Hungary
country: India
country: Ireland
country: Israel
country: Italy
country: Japan
country: Korea, Republic of
country: Malaysia
country: South Africa
country: Taiwan
country: Turkey
country: Ukraine
country: United Kingdom
country: United States
responsible party type: Sponsor
mesh term: Hemophilia A
sharing ipd: Yes
ipd description: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org