<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/NCT03818529</url>
  </required_header>
  <id_info>
    <org_study_id>ATHN 8</org_study_id>
    <nct_id>NCT03818529</nct_id>
  </id_info>
  <brief_title>ATHN 8: Previously Untreated Patients (PUPs) Matter Study</brief_title>
  <official_title>US Cohort Study of Previously Untreated Patients (PUPs) With Congenital Hemophilia</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>American Thrombosis and Hemostasis Network</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>CSL Behring</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
    <collaborator>
      <agency>Octapharma</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
    <collaborator>
      <agency>Takeda</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
  </sponsors>
  <source>American Thrombosis and Hemostasis Network</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>
      This is a multi-center cohort study of approximately 250 previously untreated patients (PUPs)&#xD;
      with congenital moderate to severe hemophilia A or B in a network of up to 50 US Hemophilia&#xD;
      Treatment Centers (HTCs). Participants will be followed as they receive their first 50&#xD;
      exposure days (ED) to clotting factor replacement product, both prospectively and&#xD;
      retrospectively. The data collected on evolving treatment practices will define the incidence&#xD;
      and risk factors for inhibitor development during the high risk period of first 50 ED and&#xD;
      improve the outcomes of this vulnerable population.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This multi-center, longitudinal, observational, prospective and retrospective study of&#xD;
      previously untreated patients (PUPs) with moderate to severe hemophilia A or B during the&#xD;
      initial 50 exposure days (ED) to clotting factor replacement product or until the development&#xD;
      of a confirmed inhibitor. The Primary Investigators have designed the study to utilize the&#xD;
      American Thrombosis and Hemostasis Network (ATHN) electronic infrastructure to leverage&#xD;
      existing data and enable the collection of more in-depth clinical and laboratory data on&#xD;
      PUPs. The study aligns with the National Hemophilia Foundation Medical and Scientific&#xD;
      Advisory Council (MASAC) recently issued Recommendation #243 which includes: &quot;Regardless of&#xD;
      which option is chosen, all PUPs should be enrolled in the ATHN data collection system or a&#xD;
      clinical trial to assess outcomes.&quot; Co-enrollment in the ATHNdataset by participants is&#xD;
      required. The total study duration is planned for 6 years.&#xD;
&#xD;
      The primary objective is to determine the percentage of patients with confirmed inhibitors&#xD;
      within the first 50 ED. Confirmed inhibitors are defined as two consecutive positive&#xD;
      inhibitor titers (per CDC laboratory criteria; &gt;0.5 Nijmegen Bethesda Units for hemophilia A&#xD;
      and &gt;0.3 Nijmegen Bethesda Units for hemophilia B) on different blood samples which result in&#xD;
      change in treatment recommendations.&#xD;
&#xD;
      Please note - the treatment regimen will be at the discretion of the participants' hemophilia&#xD;
      caregivers. No treatment products are being provided by the study nor will the participants&#xD;
      be paid. However, inhibitor titer testing will be provided at no cost to participants by the&#xD;
      Centers for Disease Control and Prevention (CDC).&#xD;
&#xD;
      All study procedures and follow-up will be timed to coincide with scheduled hemophilia care&#xD;
      whenever possible. Ad hoc, quarterly follow-up, annual and final visits are for participants&#xD;
      who have not met study endpoints (50 ED or inhibitor development) prior to enrollment.&#xD;
&#xD;
      Data collected will include eligibility, demographics, medical history (co-morbidities,&#xD;
      surgery/procedures, immunizations and allergies), hemophilia history (severity, genotype and&#xD;
      family history), birth history, inhibitor testing results, detailed treatment product(s)&#xD;
      usage, bleeding events, bleeding disorder related medical visits during the study, and EUHASS&#xD;
      adverse events.&#xD;
&#xD;
      Sub-studies&#xD;
&#xD;
      A number of sub-studies are planned with pharmaceutical sponsors to collect information from&#xD;
      patients about their specific product use. Participation in these product specific&#xD;
      sub-studies is optional and sub-study visits will be planned to coincide with HTC visits. The&#xD;
      sub-study will collect information from patients about their perception and use of treatment&#xD;
      products, physical activity levels and other general health questions. This data will be&#xD;
      collected via questionnaire.&#xD;
&#xD;
      Data Collection System&#xD;
&#xD;
      All data collected will be entered into electronic case report forms (eCRFs) within the&#xD;
      secure ATHN System by HTC site personnel. All participating study sites will have in place a&#xD;
      current, executed Data Use and Business Associate Agreement (DUBAA) with ATHN.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">October 3, 2018</start_date>
  <completion_date type="Actual">December 31, 2022</completion_date>
  <primary_completion_date type="Actual">January 26, 2022</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>Other</time_perspective>
  </study_design_info>
  <primary_outcome>
    <measure>Determine percentage of patients with confirmed inhibitors</measure>
    <time_frame>6 years</time_frame>
    <description>Participants will be followed to assess inhibitor development (per CDC laboratory criteria; &gt;0.5 Nijmegen Bethesda Units for hemophilia A and &gt;0.3 Nijmegen Bethesda Units for hemophilia B) within 50 exposure days. Blood specimens will be submitted to the CDC for inhibitor testing at various time points outlined in the protocol.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Determine risk factors including genetic variants associated with inhibitor development in PUPs</measure>
    <time_frame>6 years</time_frame>
    <description>The study will provide a systematic approach to data collection by using the ATHN System to provide database infrastructure and data collection methods to evaluate determinants of inhibitor formation.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Determine percentage of eligible participants enrolled at each site</measure>
    <time_frame>6 years</time_frame>
    <description>Sites will report number of eligible participants and number of participants enrolled to determine the percentage of enrollment.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Determine mean age of diagnosis and first exposure to factor treatment product</measure>
    <time_frame>6 years</time_frame>
    <description>Sites will document the date of diagnosis and the date of first exposure to clotting factor treatment product.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Determine the number of exposure days prior to inhibitor development</measure>
    <time_frame>6 years</time_frame>
    <description>Sites and participants will work together to provide detailed records of exposure days to clotting factor replacement products. The CDC will act as the central laboratory for the study and provide inhibitor testing.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Report bleeding complications that occur within the first 50 ED</measure>
    <time_frame>6 years</time_frame>
    <description>The documentation of bleeding events in the study records will be based on the review of bleeding and infusion records provided by the participant or the from medical chart review during the first 50 exposure days to clotting factor replacement product.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Summarize factor replacement dosing regimen prescribed to this study population within the first 50 ED</measure>
    <time_frame>6 years</time_frame>
    <description>A summary of the prescribed clotting factor replacement regimen will provide data on evolving treatment practices, including specific clotting factor replacement and non-factor products.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Report on the number of transient inhibitor, e.g., those which resolve without change in therapy</measure>
    <time_frame>6 years</time_frame>
    <description>The CDC is acting as the central laboratory for inhibitor testing. The CDC will report results to the participating HTC. If a blood specimen from a participant has an elevated result; potentially indicating the development of an inhibitor, a new blood specimen from the participant will be tested within 10 days of the first elevated result to determine if an inhibitor has developed. A transient inhibitor is defined as a positive inhibitor which is not confirmed on a consecutive repeat testing. Inhibitor testing results on all participants will be documented and reported by the HTC.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Report on targeted post-approval safety data for events related to clotting factor replacement products used by prospectively enroll participants</measure>
    <time_frame>6 years</time_frame>
    <description>Documentation of adverse events experienced by prospectively enrolled participants during the study period of first 50 exposure days to clotting factor replacement product will be reported. The types of adverse events to be reported are limited to Serious Adverse Events as defined by the European Union Haemophilia Safety Surveillance System (EUHASS).</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Sub-study modules will be developed to evaluate and report on cohorts of study participants who initiated treatment with a specific product</measure>
    <time_frame>6 years</time_frame>
    <description>Measure the number of participants who initiate treatment with a specific treatment product</description>
  </secondary_outcome>
  <enrollment type="Actual">237</enrollment>
  <condition>Hemophilia</condition>
  <condition>Hemophilia A</condition>
  <condition>Hemophilia B</condition>
  <condition>Hemophilia A With Inhibitor</condition>
  <condition>Hemophilia B With Inhibitor</condition>
  <condition>Haemophilia B Without Inhibitor</condition>
  <condition>Haemophilia A Without Inhibitor</condition>
  <condition>Haemophilia</condition>
  <eligibility>
    <study_pop>
      <textblock>
        The study will enroll a cohort of 250 participants born with hemophilia on or after January&#xD;
        1, 2010 who meet the eligibility criteria and are receiving care from one of the&#xD;
        participating HTCs.&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Congenital hemophilia A; FVIII &lt;/=5% or congenital hemophilia B; FIX &lt;/=5%;&#xD;
&#xD;
          -  Birth date on or after January 1, 2010;&#xD;
&#xD;
          -  Care established at one of the participating HTCs;&#xD;
&#xD;
          -  Co-enrollment in the ATHNdataset; and&#xD;
&#xD;
          -  Parent or authorized guardian can provide informed consent&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Patients who are referred to the HTC with no record of bleed and factor utilization&#xD;
             data&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>0 Years</minimum_age>
    <maximum_age>13 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Shannon Carpenter, MD, MS</last_name>
    <role>Principal Investigator</role>
    <affiliation>Children's Mercy Hospital Kansas City</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Courtney Thornburg, MD, MS</last_name>
    <role>Principal Investigator</role>
    <affiliation>University of California San Diego, Rady Children's Hospital San Diego</affiliation>
  </overall_official>
  <overall_official>
    <last_name>Marijke van den Berg, MD, PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Versiti</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Arizona Hemophilia and Thrombosis Center at Phoenix Children's Hospital</name>
      <address>
        <city>Phoenix</city>
        <state>Arizona</state>
        <zip>85016</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Valley Children's Hospital</name>
      <address>
        <city>Madera</city>
        <state>California</state>
        <zip>93636</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Rady Children's Hospital San Diego</name>
      <address>
        <city>San Diego</city>
        <state>California</state>
        <zip>92123</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UCSF Pediatric Hemophilia Treatment Center at Mission Bay</name>
      <address>
        <city>San Francisco</city>
        <state>California</state>
        <zip>94158</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Colorado Denver Hemophilia and Thrombosis Center</name>
      <address>
        <city>Aurora</city>
        <state>Colorado</state>
        <zip>80045</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Connecticut Bleeding and Clotting Disorders Center</name>
      <address>
        <city>Farmington</city>
        <state>Connecticut</state>
        <zip>06030</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's National Medical Center</name>
      <address>
        <city>Washington</city>
        <state>District of Columbia</state>
        <zip>20010</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Johns Hopkins All Children's Hospital</name>
      <address>
        <city>Saint Petersburg</city>
        <state>Florida</state>
        <zip>33701</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Children's Healthcare of Atlanta/Emory</name>
      <address>
        <city>Atlanta</city>
        <state>Georgia</state>
        <zip>30322</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Augusta University Hemophilia Treatment Center</name>
      <address>
        <city>Augusta</city>
        <state>Georgia</state>
        <zip>30912</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Rush University Medical Center</name>
      <address>
        <city>Chicago</city>
        <state>Illinois</state>
        <zip>60612</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Indiana Hemophilia and Thrombosis Center (IHTC)</name>
      <address>
        <city>Indianapolis</city>
        <state>Indiana</state>
        <zip>46260</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kansas City Regional Hemophilia Center</name>
      <address>
        <city>Kansas City</city>
        <state>Kansas</state>
        <zip>64108</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Maine Hemophilia and Thrombosis Center</name>
      <address>
        <city>Scarborough</city>
        <state>Maine</state>
        <zip>04074</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Boston Hemophilia Center at Children's Hospital of Boston</name>
      <address>
        <city>Boston</city>
        <state>Massachusetts</state>
        <zip>02115</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University of Michigan Hemophilia and Coagulation Disorders</name>
      <address>
        <city>Ann Arbor</city>
        <state>Michigan</state>
        <zip>48109</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Mayo Comprehensive Hemophilia Center</name>
      <address>
        <city>Rochester</city>
        <state>Minnesota</state>
        <zip>55905</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Cincinnati Children's Hospital Medical Center, Hemophilia &amp; Thrombosis Center</name>
      <address>
        <city>Cincinnati</city>
        <state>Ohio</state>
        <zip>45229</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>UHHS Cleveland</name>
      <address>
        <city>Cleveland</city>
        <state>Ohio</state>
        <zip>44106</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Northwest Ohio Hemophilia Treatment Center at the Toledo Hospital</name>
      <address>
        <city>Toledo</city>
        <state>Ohio</state>
        <zip>43606</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Oklahoma Center for Bleeding and Clotting Disorders</name>
      <address>
        <city>Oklahoma City</city>
        <state>Oklahoma</state>
        <zip>73104</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Oregon Health and Science University</name>
      <address>
        <city>Portland</city>
        <state>Oregon</state>
        <zip>97239</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>St Jude Children's Research Hospital</name>
      <address>
        <city>Memphis</city>
        <state>Tennessee</state>
        <zip>38105</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Vanderbilt University Medical Center</name>
      <address>
        <city>Nashville</city>
        <state>Tennessee</state>
        <zip>37212</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Utah Center for Bleeding &amp; Clotting Disorders at Primary Children's Hospital</name>
      <address>
        <city>Salt Lake City</city>
        <state>Utah</state>
        <zip>84113</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hemophilia Outreach Center</name>
      <address>
        <city>Green Bay</city>
        <state>Wisconsin</state>
        <zip>54311</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Blood Center of Wisconsin</name>
      <address>
        <city>Milwaukee</city>
        <state>Wisconsin</state>
        <zip>53201</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>United States</country>
  </location_countries>
  <verification_date>January 2023</verification_date>
  <study_first_submitted>January 23, 2019</study_first_submitted>
  <study_first_submitted_qc>January 24, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">January 28, 2019</study_first_posted>
  <last_update_submitted>January 30, 2023</last_update_submitted>
  <last_update_submitted_qc>January 30, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">February 1, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Hemophilia</keyword>
  <keyword>Previously Untreated Patients (PUPs)</keyword>
  <keyword>Clotting Factor Replacement Products</keyword>
  <keyword>Haemophilia</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/NCT03818529
org study id: ATHN 8
nct id: NCT03818529
lead sponsor: 
collaborator: 
collaborator: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
This is a multi-center cohort study of approximately 250 previously untreated patients (PUPs)
      with congenital moderate to severe hemophilia A or B in a network of up to 50 US Hemophilia
      Treatment Centers (HTCs). Participants will be followed as they receive their first 50
      exposure days (ED) to clotting factor replacement product, both prospectively and
      retrospectively. The data collected on evolving treatment practices will define the incidence
      and risk factors for inhibitor development during the high risk period of first 50 ED and
      improve the outcomes of this vulnerable population.
This multi-center, longitudinal, observational, prospective and retrospective study of
      previously untreated patients (PUPs) with moderate to severe hemophilia A or B during the
      initial 50 exposure days (ED) to clotting factor replacement product or until the development
      of a confirmed inhibitor. The Primary Investigators have designed the study to utilize the
      American Thrombosis and Hemostasis Network (ATHN) electronic infrastructure to leverage
      existing data and enable the collection of more in-depth clinical and laboratory data on
      PUPs. The study aligns with the National Hemophilia Foundation Medical and Scientific
      Advisory Council (MASAC) recently issued Recommendation #243 which includes: "Regardless of
      which option is chosen, all PUPs should be enrolled in the ATHN data collection system or a
      clinical trial to assess outcomes." Co-enrollment in the ATHNdataset by participants is
      required. The total study duration is planned for 6 years.

      The primary objective is to determine the percentage of patients with confirmed inhibitors
      within the first 50 ED. Confirmed inhibitors are defined as two consecutive positive
      inhibitor titers (per CDC laboratory criteria; >0.5 Nijmegen Bethesda Units for hemophilia A
      and >0.3 Nijmegen Bethesda Units for hemophilia B) on different blood samples which result in
      change in treatment recommendations.

      Please note - the treatment regimen will be at the discretion of the participants' hemophilia
      caregivers. No treatment products are being provided by the study nor will the participants
      be paid. However, inhibitor titer testing will be provided at no cost to participants by the
      Centers for Disease Control and Prevention (CDC).

      All study procedures and follow-up will be timed to coincide with scheduled hemophilia care
      whenever possible. Ad hoc, quarterly follow-up, annual and final visits are for participants
      who have not met study endpoints (50 ED or inhibitor development) prior to enrollment.

      Data collected will include eligibility, demographics, medical history (co-morbidities,
      surgery/procedures, immunizations and allergies), hemophilia history (severity, genotype and
      family history), birth history, inhibitor testing results, detailed treatment product(s)
      usage, bleeding events, bleeding disorder related medical visits during the study, and EUHASS
      adverse events.

      Sub-studies

      A number of sub-studies are planned with pharmaceutical sponsors to collect information from
      patients about their specific product use. Participation in these product specific
      sub-studies is optional and sub-study visits will be planned to coincide with HTC visits. The
      sub-study will collect information from patients about their perception and use of treatment
      products, physical activity levels and other general health questions. This data will be
      collected via questionnaire.

      Data Collection System

      All data collected will be entered into electronic case report forms (eCRFs) within the
      secure ATHN System by HTC site personnel. All participating study sites will have in place a
      current, executed Data Use and Business Associate Agreement (DUBAA) with ATHN.
observational model: Cohort
time perspective: Other
measure: Determine percentage of patients with confirmed inhibitors
time frame: 6 years
description: Participants will be followed to assess inhibitor development (per CDC laboratory criteria; >0.5 Nijmegen Bethesda Units for hemophilia A and >0.3 Nijmegen Bethesda Units for hemophilia B) within 50 exposure days. Blood specimens will be submitted to the CDC for inhibitor testing at various time points outlined in the protocol.
measure: Determine risk factors including genetic variants associated with inhibitor development in PUPs
time frame: 6 years
description: The study will provide a systematic approach to data collection by using the ATHN System to provide database infrastructure and data collection methods to evaluate determinants of inhibitor formation.
measure: Determine percentage of eligible participants enrolled at each site
time frame: 6 years
description: Sites will report number of eligible participants and number of participants enrolled to determine the percentage of enrollment.
measure: Determine mean age of diagnosis and first exposure to factor treatment product
time frame: 6 years
description: Sites will document the date of diagnosis and the date of first exposure to clotting factor treatment product.
measure: Determine the number of exposure days prior to inhibitor development
time frame: 6 years
description: Sites and participants will work together to provide detailed records of exposure days to clotting factor replacement products. The CDC will act as the central laboratory for the study and provide inhibitor testing.
measure: Report bleeding complications that occur within the first 50 ED
time frame: 6 years
description: The documentation of bleeding events in the study records will be based on the review of bleeding and infusion records provided by the participant or the from medical chart review during the first 50 exposure days to clotting factor replacement product.
measure: Summarize factor replacement dosing regimen prescribed to this study population within the first 50 ED
time frame: 6 years
description: A summary of the prescribed clotting factor replacement regimen will provide data on evolving treatment practices, including specific clotting factor replacement and non-factor products.
measure: Report on the number of transient inhibitor, e.g., those which resolve without change in therapy
time frame: 6 years
description: The CDC is acting as the central laboratory for inhibitor testing. The CDC will report results to the participating HTC. If a blood specimen from a participant has an elevated result; potentially indicating the development of an inhibitor, a new blood specimen from the participant will be tested within 10 days of the first elevated result to determine if an inhibitor has developed. A transient inhibitor is defined as a positive inhibitor which is not confirmed on a consecutive repeat testing. Inhibitor testing results on all participants will be documented and reported by the HTC.
measure: Report on targeted post-approval safety data for events related to clotting factor replacement products used by prospectively enroll participants
time frame: 6 years
description: Documentation of adverse events experienced by prospectively enrolled participants during the study period of first 50 exposure days to clotting factor replacement product will be reported. The types of adverse events to be reported are limited to Serious Adverse Events as defined by the European Union Haemophilia Safety Surveillance System (EUHASS).
measure: Sub-study modules will be developed to evaluate and report on cohorts of study participants who initiated treatment with a specific product
time frame: 6 years
description: Measure the number of participants who initiate treatment with a specific treatment product
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 0 Years
maximum age: 13 Years
healthy volunteers: No
last name: Shannon Carpenter, MD, MS
role: Principal Investigator
affiliation: Children's Mercy Hospital Kansas City
last name: Courtney Thornburg, MD, MS
role: Principal Investigator
affiliation: University of California San Diego, Rady Children's Hospital San Diego
last name: Marijke van den Berg, MD, PhD
role: Principal Investigator
affiliation: Versiti
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country: United States
responsible party type: Sponsor
mesh term: Hemophilia A
mesh term: Hemophilia B