<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/NCT02971969</url>
  </required_header>
  <id_info>
    <org_study_id>101HEMB02</org_study_id>
    <secondary_id>2016-003430-25</secondary_id>
    <nct_id>NCT02971969</nct_id>
  </id_info>
  <brief_title>Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B</brief_title>
  <official_title>A Long-Term Follow-up Study to Evaluate the Safety, Tolerability, and Efficacy of Adeno-Associated Virus (AAV) rh10-Mediated Gene Transfer of Human Factor IX in Adults With Moderate/Severe to Severe Hemophilia B</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Ultragenyx Pharmaceutical Inc</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Ultragenyx Pharmaceutical Inc</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      A long-term follow-up study to evaluate the safety, tolerability, and efficacy of DTX101 in&#xD;
      adult males with moderate/severe to severe hemophilia B.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Hemophilia B is an X-linked recessive genetic bleeding disorder caused by mutations in the&#xD;
      factor IX (FIX) gene. FIX is produced in the liver and is critical for fibrin clot formation.&#xD;
      Hemophilia B is characterized by frequent, spontaneous internal bleeding that can lead to&#xD;
      chronic arthropathy (joint damage), intracranial hemorrhage, and even death. In patients with&#xD;
      moderate/severe to severe hemophilia B, the majority of bleeding episodes occur in the joints&#xD;
      and, if not treated, lead to debilitating damage and a decreased quality of life.&#xD;
&#xD;
      Study 101HEMB02 is a long-term follow-up study to evaluate the safety, tolerability, and&#xD;
      efficacy of AAVrh10-mediated gene therapy of human FIX in subjects with moderate/severe to&#xD;
      severe hemophilia B. The primary objective of the study is to determine the long-term safety&#xD;
      and efficacy of DTX101 following a single IV infusion (administered during Study 101HEMB01)&#xD;
      in adults with moderate/severe to severe hemophilia B.&#xD;
&#xD;
      This study was previously posted by Dimension Therapeutics, which has been acquired by&#xD;
      Ultragenyx.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date>January 2017</start_date>
  <completion_date type="Actual">November 6, 2021</completion_date>
  <primary_completion_date type="Actual">November 6, 2021</primary_completion_date>
  <study_type>Observational</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <observational_model>Cohort</observational_model>
    <time_perspective>Prospective</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Incidence of adverse events and serious adverse events by dosing group</measure>
    <time_frame>208 weeks</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Change from baseline in FIX activity level</measure>
    <time_frame>208 weeks</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Number of bleeding episodes requiring recombinant FIX infusion</measure>
    <time_frame>208 weeks</time_frame>
  </secondary_outcome>
  <enrollment type="Actual">6</enrollment>
  <condition>Hemophilia B</condition>
  <biospec_retention>Samples With DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      Blood&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Adult males with moderate/severe to severe Hemophilia B previously enrolled in 101HEMB01&#xD;
        clinical study&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Willing and able to provide written informed consent.&#xD;
&#xD;
          2. Completed the Week 52 visit in Study 101HEMB01.&#xD;
&#xD;
          3. Willing to stop prophylactic treatment with recombinant FIX at specified time points&#xD;
             during the study if medically acceptable.&#xD;
&#xD;
          4. Willing, able, and committed to comply with scheduled study site visits, study&#xD;
             procedures, and requirements.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Planned or current participation in another interventional clinical study that may&#xD;
             confound the efficacy or safety evaluation of DTX101 during the duration of this&#xD;
             study.&#xD;
&#xD;
          2. Any clinically significant medical condition that, in the opinion of the investigator,&#xD;
             would pose a risk to subject safety or would impede the study&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Medical Director</last_name>
    <role>Study Director</role>
    <affiliation>Ultragenyx Pharmaceutical Inc</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Arkansas Children's Hospital</name>
      <address>
        <city>Little Rock</city>
        <state>Arkansas</state>
        <zip>72202</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UF CRC - Clinical Research Center</name>
      <address>
        <city>Gainesville</city>
        <state>Florida</state>
        <zip>32610</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Michigan Hospital and Health Systems</name>
      <address>
        <city>Ann Arbor</city>
        <state>Michigan</state>
        <zip>48109-5872</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Haemophilia, Haemostasis &amp; Thrombosis Centre</name>
      <address>
        <city>Basingstoke</city>
        <state>Hampshire</state>
        <zip>RG24 9NA</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Manchester Haemophilia Comprehensive Care Center</name>
      <address>
        <city>Manchester</city>
        <zip>M13 9WL</zip>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <verification_date>January 2022</verification_date>
  <study_first_submitted>November 21, 2016</study_first_submitted>
  <study_first_submitted_qc>November 21, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">November 23, 2016</study_first_posted>
  <last_update_submitted>January 4, 2022</last_update_submitted>
  <last_update_submitted_qc>January 4, 2022</last_update_submitted_qc>
  <last_update_posted type="Actual">January 6, 2022</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>gene therapy</keyword>
  <keyword>Hemophilia B</keyword>
  <condition_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>Hemophilia A</mesh_term>
    <mesh_term>Hemophilia B</mesh_term>
  </condition_browse>
  <!-- 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/NCT02971969
org study id: 101HEMB02
secondary id: 2016-003430-25
nct id: NCT02971969
lead sponsor: 
has dmc: No
A long-term follow-up study to evaluate the safety, tolerability, and efficacy of DTX101 in
      adult males with moderate/severe to severe hemophilia B.
Hemophilia B is an X-linked recessive genetic bleeding disorder caused by mutations in the
      factor IX (FIX) gene. FIX is produced in the liver and is critical for fibrin clot formation.
      Hemophilia B is characterized by frequent, spontaneous internal bleeding that can lead to
      chronic arthropathy (joint damage), intracranial hemorrhage, and even death. In patients with
      moderate/severe to severe hemophilia B, the majority of bleeding episodes occur in the joints
      and, if not treated, lead to debilitating damage and a decreased quality of life.

      Study 101HEMB02 is a long-term follow-up study to evaluate the safety, tolerability, and
      efficacy of AAVrh10-mediated gene therapy of human FIX in subjects with moderate/severe to
      severe hemophilia B. The primary objective of the study is to determine the long-term safety
      and efficacy of DTX101 following a single IV infusion (administered during Study 101HEMB01)
      in adults with moderate/severe to severe hemophilia B.

      This study was previously posted by Dimension Therapeutics, which has been acquired by
      Ultragenyx.
observational model: Cohort
time perspective: Prospective
measure: Incidence of adverse events and serious adverse events by dosing group
time frame: 208 weeks
measure: Change from baseline in FIX activity level
time frame: 208 weeks
measure: Number of bleeding episodes requiring recombinant FIX infusion
time frame: 208 weeks
Blood
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: Male
minimum age: 18 Years
maximum age: N/A
healthy volunteers: No
last name: Medical Director
role: Study Director
affiliation: Ultragenyx Pharmaceutical Inc
facility: 
facility: 
facility: 
facility: 
facility: 
country: United Kingdom
country: United States
responsible party type: Sponsor
mesh term: Hemophilia A
mesh term: Hemophilia B