<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/NCT03793426</url>
  </required_header>
  <id_info>
    <org_study_id>FORMA-07</org_study_id>
    <nct_id>NCT03793426</nct_id>
  </id_info>
  <brief_title>Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency</brief_title>
  <official_title>Post-marketing Observational Study on the Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Octapharma</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Octapharma</source>
  <oversight_info>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      Open-label, Uncontrolled, Multicenter Observational Study on the Safety and Efficacy of&#xD;
      Fibryga in Congenital Fibrinogen Deficiency&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      There is a need to increase the body of data on treatment effectiveness and safety in the&#xD;
      ultra-rare setting of congenital fibrinogen deficiency. Real-world evidence (RWE) derived&#xD;
      from non-interventional studies can describe product utilization, demonstrate value, and&#xD;
      facilitate benefit-risk assessments; RWE can only be fully assessed once a product is&#xD;
      launched and used in a real-life setting.&#xD;
&#xD;
      This post-marketing, observational study is designed to collect information concerning&#xD;
      safety, efficacy, and outcomes of Fibryga administration in routine clinical use in patients&#xD;
      of any age with congenital afibrinogenemia or hypofibrinogenemia. Documentation of the&#xD;
      administration of Fibryga in clinical practice for the treatment of both minor and major&#xD;
      bleeding events (BEs) will not only enhance the knowledge on the efficacy and safety profile&#xD;
      of Fibryga, but will also gather information that cannot be obtained in the same way in&#xD;
      controlled clinical studies. These observational data will support the safety and efficacy&#xD;
      data generated with Fibryga in good clinical practice (GCP) clinical studies, providing&#xD;
      benefit for both physicians and patients.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Recruiting</overall_status>
  <start_date type="Actual">January 28, 2021</start_date>
  <completion_date type="Anticipated">October 2027</completion_date>
  <primary_completion_date type="Anticipated">October 2027</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>The incidence of thromboembolic adverse drug reactions (ADRs)</measure>
    <time_frame>Day 0-28</time_frame>
    <description>The incidence of thromboembolic ADRs in patients receiving Fibryga for on-demand treatment of bleeding, including major bleeding, will be documented</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Hemostatic efficacy of Fibryga for all bleeding events (BEs) collected in the study will be assessed by the investigator using a 4-point hemostatic efficacy scale</measure>
    <time_frame>Within 2-24 hours following treatment of BEs</time_frame>
    <description>The hemostatic efficacy of Fibryga for all BEs collected in the study will be assessed by the investigator using a 4-point hemostatic efficacy scale including the four items: 'excellent,' 'good,' moderate,' and 'none'. These data will be transformed into a dichotomous result, with 'treatment success-yes' defined as a rating of 'excellent' or 'good' and 'treatment success-no' defined as a rating of 'moderate' or 'none'.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Dosage of Fibryga</measure>
    <time_frame>Within 2-24 hours following treatment of BEs</time_frame>
    <description>Fibryga will be individually dosed as per the locally approved package insert. Actual dosage administered will be documented</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Duration of BEs</measure>
    <time_frame>Within 2-24 hours following treatment of BEs</time_frame>
    <description>Details of BE duration will be documented</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Incidence of treatment-emergent adverse events (safety)</measure>
    <time_frame>Day 0-28</time_frame>
    <description>All ADRs in patients receiving Fibryga for on-demand treatment of BEs, including major BEs, will be documented</description>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Anticipated">25</enrollment>
  <condition>Congenital Fibrinogen Deficiency</condition>
  <arm_group>
    <arm_group_label>Fibryga</arm_group_label>
    <description>Fibryga (human plasma-derived fibrinogen concentrate)</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>Fibryga</intervention_name>
    <description>Human plasma-derived fibrinogen concentrate</description>
    <arm_group_label>Fibryga</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        Approximately 25 patients with a documented diagnosis of congenital afibrinogenemia or&#xD;
        hypofibrinogenemia are planned to be documented in this study&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Patients of any age with a documented diagnosis of congenital afibrinogenemia or&#xD;
             hypofibrinogenemia expected to require on-demand in-hospital treatment for BEs with&#xD;
             Fibryga&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Bleeding disorder other than congenital fibrinogen deficiency&#xD;
&#xD;
          -  Patients with acquired fibrinogen deficiency or dysfibrinogenemia&#xD;
&#xD;
          -  Suspicion of an anti-fibrinogen inhibitor as indicated by previous in vivo recovery,&#xD;
             if available, of &lt;0.5 (mg/dL)/(mg/kg); there is currently no standard test for&#xD;
             inhibitors&#xD;
&#xD;
          -  Participation in an interventional clinical study at the time of or within 4 weeks&#xD;
             prior to enrolment&#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_contact>
    <last_name>Gita Pezeshki, PhD</last_name>
    <phone>+12016041125</phone>
    <email>gita.pezeshki@octapharma.com</email>
  </overall_contact>
  <overall_contact_backup>
    <last_name>Sylvia Werner, MS</last_name>
    <phone>+12016041149</phone>
    <email>sylvia.werner@octapharma.com</email>
  </overall_contact_backup>
  <location>
    <facility>
      <name>Gerinnungszentrum rhein-ruhr</name>
      <address>
        <city>Duisburg</city>
        <country>Germany</country>
      </address>
    </facility>
    <status>Recruiting</status>
  </location>
  <location>
    <facility>
      <name>Medizinische Klinik 2 / Institut für Transfusionsmedizin Universitätsklinikum</name>
      <address>
        <city>Frankfurt</city>
        <country>Germany</country>
      </address>
    </facility>
    <status>Recruiting</status>
  </location>
  <location>
    <facility>
      <name>Gerinnungszentrum Hochtaunus</name>
      <address>
        <city>Hamburg</city>
        <country>Germany</country>
      </address>
    </facility>
    <status>Recruiting</status>
  </location>
  <location_countries>
    <country>Germany</country>
  </location_countries>
  <verification_date>August 2023</verification_date>
  <study_first_submitted>December 19, 2018</study_first_submitted>
  <study_first_submitted_qc>January 3, 2019</study_first_submitted_qc>
  <study_first_posted type="Actual">January 4, 2019</study_first_posted>
  <last_update_submitted>August 15, 2023</last_update_submitted>
  <last_update_submitted_qc>August 15, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">August 16, 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>Afibrinogenemia</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/NCT03793426
org study id: FORMA-07
nct id: NCT03793426
lead sponsor: 
is fda regulated drug: Yes
is fda regulated device: No
Open-label, Uncontrolled, Multicenter Observational Study on the Safety and Efficacy of
      Fibryga in Congenital Fibrinogen Deficiency
There is a need to increase the body of data on treatment effectiveness and safety in the
      ultra-rare setting of congenital fibrinogen deficiency. Real-world evidence (RWE) derived
      from non-interventional studies can describe product utilization, demonstrate value, and
      facilitate benefit-risk assessments; RWE can only be fully assessed once a product is
      launched and used in a real-life setting.

      This post-marketing, observational study is designed to collect information concerning
      safety, efficacy, and outcomes of Fibryga administration in routine clinical use in patients
      of any age with congenital afibrinogenemia or hypofibrinogenemia. Documentation of the
      administration of Fibryga in clinical practice for the treatment of both minor and major
      bleeding events (BEs) will not only enhance the knowledge on the efficacy and safety profile
      of Fibryga, but will also gather information that cannot be obtained in the same way in
      controlled clinical studies. These observational data will support the safety and efficacy
      data generated with Fibryga in good clinical practice (GCP) clinical studies, providing
      benefit for both physicians and patients.
observational model: Cohort
time perspective: Prospective
measure: The incidence of thromboembolic adverse drug reactions (ADRs)
time frame: Day 0-28
description: The incidence of thromboembolic ADRs in patients receiving Fibryga for on-demand treatment of bleeding, including major bleeding, will be documented
measure: Hemostatic efficacy of Fibryga for all bleeding events (BEs) collected in the study will be assessed by the investigator using a 4-point hemostatic efficacy scale
time frame: Within 2-24 hours following treatment of BEs
description: The hemostatic efficacy of Fibryga for all BEs collected in the study will be assessed by the investigator using a 4-point hemostatic efficacy scale including the four items: 'excellent,' 'good,' moderate,' and 'none'. These data will be transformed into a dichotomous result, with 'treatment success-yes' defined as a rating of 'excellent' or 'good' and 'treatment success-no' defined as a rating of 'moderate' or 'none'.
measure: Dosage of Fibryga
time frame: Within 2-24 hours following treatment of BEs
description: Fibryga will be individually dosed as per the locally approved package insert. Actual dosage administered will be documented
measure: Duration of BEs
time frame: Within 2-24 hours following treatment of BEs
description: Details of BE duration will be documented
measure: Incidence of treatment-emergent adverse events (safety)
time frame: Day 0-28
description: All ADRs in patients receiving Fibryga for on-demand treatment of BEs, including major BEs, will be documented
arm group label: Fibryga
description: Fibryga (human plasma-derived fibrinogen concentrate)
intervention type: Biological
intervention name: Fibryga
description: Human plasma-derived fibrinogen concentrate
arm group label: Fibryga
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: N/A
maximum age: N/A
healthy volunteers: No
last name: Gita Pezeshki, PhD
phone: +12016041125
email: gita.pezeshki@octapharma.com
last name: Sylvia Werner, MS
phone: +12016041149
email: sylvia.werner@octapharma.com
facility: 
status: Recruiting
facility: 
status: Recruiting
facility: 
status: Recruiting
country: Germany
responsible party type: Sponsor
mesh term: Afibrinogenemia