<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/NCT03824522</url>
  </required_header>
  <id_info>
    <org_study_id>261603</org_study_id>
    <nct_id>NCT03824522</nct_id>
  </id_info>
  <brief_title>Post Marketing Surveillance Study for ADYNOVATE in South Korea</brief_title>
  <official_title>Post Marketing Surveillance Study for ADYNOVATE in South Korea</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Takeda</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Takeda Pharma Korea Co. Ltd.</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Takeda</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
    <is_fda_regulated_drug>No</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The main aim of this study is to check for side effects and medical problems in participants&#xD;
      with hemophilia A who are receiving ADYNOVATE in routine clinical practice. Also, it will be&#xD;
      checked how effective ADYNOVATE is at stopping a bleed, at preventing bleeding episodes when&#xD;
      used routinely for prevention and at controlling hemophilia-related bleeding during&#xD;
      surgeries.&#xD;
&#xD;
      Participants with hemophilia A who will be newly prescribed with ADYNOVATE treatment or have&#xD;
      already been taking ADYNOVATE treatment according to the physician's decision will be&#xD;
      included in this study.&#xD;
&#xD;
      Participants will visit the study clinic as a part of their routine clinical practice and&#xD;
      will be contacted by phone calls/home visits by home care workers for collection of treatment&#xD;
      related data during the 6-month observation period.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">February 25, 2019</start_date>
  <completion_date type="Anticipated">January 30, 2024</completion_date>
  <primary_completion_date type="Anticipated">January 30, 2024</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</measure>
    <time_frame>Throughout the duration of patient participation of up to approximately 6 months</time_frame>
    <description>Serious adverse events and non-serious adverse events</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Frequency of adverse events</measure>
    <time_frame>Throughout the duration of patient participation of up to approximately 6 months</time_frame>
    <description>Serious adverse events and non-serious adverse events</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Incidence of inhibitor titers for FVIII antibodies</measure>
    <time_frame>Throughout the duration of patient participation of up to approximately 6 months</time_frame>
    <description>By titer levels, high and lower titer categories</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of treated bleeds</measure>
    <time_frame>Throughout the duration of patient participation of up to approximately 6 months</time_frame>
    <description>Number of treated bleeds throughout the study period</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>ADYNOVATE units required for bleed resolution</measure>
    <time_frame>At bleed resolution, throughout the duration of patient participation of up to approximately 6 months</time_frame>
    <description>Number of ADYNOVATE units required for bleed resolution</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of ADYNOVATE infusions needed for the treatment of bleeding episodes</measure>
    <time_frame>At bleed resolution, throughout the duration of patient participation of up to approximately 6 months</time_frame>
    <description>The number of ADYNOVATE infusions needed for each bleeding episode is determined by the participant, his/her caregiver, and/or clinician treating the participant, and is based upon the participant's response to treatment</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Hemostatic effectiveness assessment of bleeding episodes treated with ADYNOVATE during prophylaxis treatment</measure>
    <time_frame>Throughout the duration of patient participation of up to approximately 6 months</time_frame>
    <description>Using a 4-point ordinal scale (Excellent, Good, Fair, or None)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Hemostatic effectiveness of ADYNOVATE for participants on an on-demand regimen</measure>
    <time_frame>At bleed resolution, throughout the duration of patient participation of up to approximately 6 months</time_frame>
    <description>Using a 4-point ordinal scale (Excellent, Good, Fair, or None)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Assessment of perioperative effectiveness of ADYNOVATE</measure>
    <time_frame>Throughout the duration of patient participation of up to approximately 6 months</time_frame>
    <description>Using a 4-point ordinal scale (Excellent, Good, Fair, or None)</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Clinically significant changes in laboratory results</measure>
    <time_frame>Throughout the duration of patient participation of up to approximately 6 months</time_frame>
    <description>Number of clinically significant changes in laboratory results</description>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Anticipated">600</enrollment>
  <condition>Hemophilia A</condition>
  <arm_group>
    <arm_group_label>All Study Participants</arm_group_label>
    <description>Participants who are newly prescribed with Adynovate and participants previously treated with Adynovate will be treated with ADYNOVATE for hemophilia A at the time of enrollment according to a regimen determined by the study site treating physician.</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>ADYNOVATE</intervention_name>
    <description>Pegylated recombinant human factor VIII</description>
    <arm_group_label>All Study Participants</arm_group_label>
    <other_name>PEGylated rFVIII</other_name>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Hemophilia A participants in South Korea who will be newly prescribed with ADYNOVATE or&#xD;
        already been prescribed with ADYNOVATE will be included in this study.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria&#xD;
&#xD;
        Participants with a diagnosis of hemophilia A who will be newly prescribed with ADYNOVATE&#xD;
        or already have been prescribed ADYNOVATE according to the study physician's judgment shall&#xD;
        be included in this study if:&#xD;
&#xD;
          -  The participant or legally authorized representative has given written informed&#xD;
             consent to participate in the study.&#xD;
&#xD;
          -  The participant is indicated for treatment according to the ADYNOVATE South Korea&#xD;
             prescribing information (PI).&#xD;
&#xD;
        Exclusion Criteria&#xD;
&#xD;
        Participants should be excluded from this study if:&#xD;
&#xD;
          -  The participant or legally authorized representative does not wish to participate in&#xD;
             the study.&#xD;
&#xD;
          -  Any of the contraindications included in the PI for ADYNOVATE apply.&#xD;
&#xD;
          -  Participant is enrolled in an interventional trial using an investigational product&#xD;
             other than ADYNOVATE.&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>N/A</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Study Director</last_name>
    <role>Study Director</role>
    <affiliation>Takeda</affiliation>
  </overall_official>
  <overall_contact>
    <last_name>Takeda Contact</last_name>
    <phone>+1-877-825-3327</phone>
    <email>medinfoUS@takeda.com</email>
  </overall_contact>
  <location>
    <facility>
      <name>Kyungpook National University Chilgok Hospital</name>
      <address>
        <city>Daegu</city>
        <zip>41404</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Site Contact</last_name>
      <phone>+82-10-8677-5701</phone>
      <email>phojyk@gmail.com</email>
    </contact>
    <investigator>
      <last_name>Ji Yooun Kim, Ph.D.</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Kyungpook National University Hospital</name>
      <address>
        <city>Daegu</city>
        <zip>41944</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Site Contact</last_name>
      <phone>+82-53-200-5704</phone>
      <email>phojyk@gmail.com</email>
    </contact>
    <investigator>
      <last_name>Ji Yooun Kim, Ph.D</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Daegu Catholic University Medical Center</name>
      <address>
        <city>Daegu</city>
        <zip>42472</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Site Contact</last_name>
      <phone>+82-53-650-4248</phone>
      <email>ejchoi2@cu.ac.kr</email>
    </contact>
    <investigator>
      <last_name>Eunjin Choi, Ph.D.</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Severance Hospital Yonsei University Health System</name>
      <address>
        <city>Seoul</city>
        <zip>03722</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Site Contact</last_name>
      <phone>+82-10-3740-9149</phone>
      <email>CJ@yuhs.ac</email>
    </contact>
    <investigator>
      <last_name>Chuhl Joo Lyu, Ph.D</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Kyung Hee University Hospital at Gangdong</name>
      <address>
        <city>Seoul</city>
        <zip>05278</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Site Contact</last_name>
      <phone>+82-10-4618-3918</phone>
      <email>pysmd@khnmc.or.kr</email>
    </contact>
    <investigator>
      <last_name>YoungShil Park, Ph.D</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Kim Hugh Chul Internal Medicine</name>
      <address>
        <city>Seoul</city>
        <zip>05538</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Site Contact</last_name>
      <phone>+82-2-478-0300</phone>
      <email>hughkim2@naver.com; hughkim7@naver.com</email>
    </contact>
    <investigator>
      <last_name>Hugh Chul Kim, Ph.D</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Korea Hemophilia Foundation (Seoul)</name>
      <address>
        <city>Seoul</city>
        <zip>06641</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Site Contact</last_name>
      <phone>+82-2-3473-6107</phone>
      <email>gowho@daum.net</email>
    </contact>
    <investigator>
      <last_name>Ki-Young Yoo, Ph.D.</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location>
    <facility>
      <name>Ulsan University Hospital</name>
      <address>
        <city>Ulsan</city>
        <zip>44033</zip>
        <country>Korea, Republic of</country>
      </address>
    </facility>
    <status>Recruiting</status>
    <contact>
      <last_name>Site Contact</last_name>
      <phone>+82-10-9709-9256</phone>
      <email>sang@uuh.ulsan.kr</email>
    </contact>
    <investigator>
      <last_name>Sang Kyun Park, Ph.D</last_name>
      <role>Principal Investigator</role>
    </investigator>
  </location>
  <location_countries>
    <country>Korea, Republic of</country>
  </location_countries>
  <link>
    <url>https://clinicaltrials.takeda.com/study-detail/5f6b5fc74db2bf003ab46012</url>
    <description>To obtain more information on the study, click here/on this link</description>
  </link>
  <verification_date>March 2023</verification_date>
  <study_first_submitted>January 29, 2019</study_first_submitted>
  <study_first_submitted_qc>January 29, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">January 31, 2019</study_first_posted>
  <last_update_submitted>March 17, 2023</last_update_submitted>
  <last_update_submitted_qc>March 17, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">March 21, 2023</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>
  <intervention_browse>
    <!-- CAUTION:  The following MeSH terms are assigned with an imperfect algorithm            -->
    <mesh_term>BAX 855</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>Yes</sharing_ipd>
    <ipd_description>Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.</ipd_description>
    <ipd_info_type>Study Protocol</ipd_info_type>
    <ipd_info_type>Statistical Analysis Plan (SAP)</ipd_info_type>
    <ipd_info_type>Informed Consent Form (ICF)</ipd_info_type>
    <ipd_info_type>Clinical Study Report (CSR)</ipd_info_type>
    <ipd_access_criteria>IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.</ipd_access_criteria>
    <ipd_url>https://vivli.org/ourmember/takeda/</ipd_url>
  </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/NCT03824522
org study id: 261603
nct id: NCT03824522
lead sponsor: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
The main aim of this study is to check for side effects and medical problems in participants
      with hemophilia A who are receiving ADYNOVATE in routine clinical practice. Also, it will be
      checked how effective ADYNOVATE is at stopping a bleed, at preventing bleeding episodes when
      used routinely for prevention and at controlling hemophilia-related bleeding during
      surgeries.

      Participants with hemophilia A who will be newly prescribed with ADYNOVATE treatment or have
      already been taking ADYNOVATE treatment according to the physician's decision will be
      included in this study.

      Participants will visit the study clinic as a part of their routine clinical practice and
      will be contacted by phone calls/home visits by home care workers for collection of treatment
      related data during the 6-month observation period.
observational model: Cohort
time perspective: Prospective
measure: Incidence of adverse events
time frame: Throughout the duration of patient participation of up to approximately 6 months
description: Serious adverse events and non-serious adverse events
measure: Frequency of adverse events
time frame: Throughout the duration of patient participation of up to approximately 6 months
description: Serious adverse events and non-serious adverse events
measure: Incidence of inhibitor titers for FVIII antibodies
time frame: Throughout the duration of patient participation of up to approximately 6 months
description: By titer levels, high and lower titer categories
measure: Number of treated bleeds
time frame: Throughout the duration of patient participation of up to approximately 6 months
description: Number of treated bleeds throughout the study period
measure: ADYNOVATE units required for bleed resolution
time frame: At bleed resolution, throughout the duration of patient participation of up to approximately 6 months
description: Number of ADYNOVATE units required for bleed resolution
measure: Number of ADYNOVATE infusions needed for the treatment of bleeding episodes
time frame: At bleed resolution, throughout the duration of patient participation of up to approximately 6 months
description: The number of ADYNOVATE infusions needed for each bleeding episode is determined by the participant, his/her caregiver, and/or clinician treating the participant, and is based upon the participant's response to treatment
measure: Hemostatic effectiveness assessment of bleeding episodes treated with ADYNOVATE during prophylaxis treatment
time frame: Throughout the duration of patient participation of up to approximately 6 months
description: Using a 4-point ordinal scale (Excellent, Good, Fair, or None)
measure: Hemostatic effectiveness of ADYNOVATE for participants on an on-demand regimen
time frame: At bleed resolution, throughout the duration of patient participation of up to approximately 6 months
description: Using a 4-point ordinal scale (Excellent, Good, Fair, or None)
measure: Assessment of perioperative effectiveness of ADYNOVATE
time frame: Throughout the duration of patient participation of up to approximately 6 months
description: Using a 4-point ordinal scale (Excellent, Good, Fair, or None)
measure: Clinically significant changes in laboratory results
time frame: Throughout the duration of patient participation of up to approximately 6 months
description: Number of clinically significant changes in laboratory results
arm group label: All Study Participants
description: Participants who are newly prescribed with Adynovate and participants previously treated with Adynovate will be treated with ADYNOVATE for hemophilia A at the time of enrollment according to a regimen determined by the study site treating physician.
intervention type: Biological
intervention name: ADYNOVATE
description: Pegylated recombinant human factor VIII
arm group label: All Study Participants
other name: PEGylated rFVIII
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
healthy volunteers: No
last name: Study Director
role: Study Director
affiliation: Takeda
last name: Takeda Contact
phone: +1-877-825-3327
email: medinfoUS@takeda.com
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
facility: 
status: Recruiting
contact: 
investigator: 
country: Korea, Republic of
url: https://clinicaltrials.takeda.com/study-detail/5f6b5fc74db2bf003ab46012
description: To obtain more information on the study, click here/on this link
responsible party type: Sponsor
mesh term: Hemophilia A
mesh term: BAX 855
sharing ipd: Yes
ipd description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
ipd info type: Study Protocol
ipd info type: Statistical Analysis Plan (SAP)
ipd info type: Informed Consent Form (ICF)
ipd info type: Clinical Study Report (CSR)
ipd access criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
ipd url: https://vivli.org/ourmember/takeda/