<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/NCT04461639</url>
  </required_header>
  <id_info>
    <org_study_id>20904</org_study_id>
    <nct_id>NCT04461639</nct_id>
  </id_info>
  <brief_title>Study to Learn More About the Safety of Drug Jivi Over a Long Period of Time in Previously Treated Patients With Hemophilia A (Bleeding Disorder Resulting From a Lack of FVIII) Who Are Receiving Jivi Regularly at Their Treating Doctors to Prevent Bleeding</brief_title>
  <acronym>HA-SAFE</acronym>
  <official_title>Observational Study Evaluating Long-term Safety of Real-world Treatment With Damoctocog Alfa Pegol in Previously Treated Patients With Hemophilia A</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Bayer</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Bayer</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>
      In this observational study researchers want to learn more about the safety of drug Jivi over&#xD;
      a long period of time. Jivi (generic name: Damoctocog alfa pegol) is an approved blood&#xD;
      clotting Factor VIII (FVIII) medication for the treatment of hemophilia A (bleeding disorder&#xD;
      resulting from a lack of FVIII). It is manufactured via recombinant technology and has an&#xD;
      extended half-live, i.e. it will stay longer in the body than other FVIII products. Therefore&#xD;
      Jivi acts longer in the body which reduces the frequency of drug injections. This study will&#xD;
      enroll previously treated patients with hemophilia A who are receiving Jivi regularly at&#xD;
      their treating doctors to prevent bleeding. Observation for each patient will last for at&#xD;
      least 4 years, and medical data will be collected during patients' routine visits at their&#xD;
      treating doctors.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Active, not recruiting</overall_status>
  <start_date type="Actual">May 14, 2021</start_date>
  <completion_date type="Anticipated">June 30, 2028</completion_date>
  <primary_completion_date type="Anticipated">June 30, 2028</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>Number of participants with safety events</measure>
    <time_frame>At least 4 years</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Duration of safety events</measure>
    <time_frame>At least 4 years</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of participants with safety events leading to a change of treatment</measure>
    <time_frame>At least 4 years</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of participants with safety events per intensity</measure>
    <time_frame>At least 4 years</time_frame>
    <description>The maximum intensity of each safety event should be assigned to one of the following categories: mild, moderate or severe</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of participants with safety events with outcome of death</measure>
    <time_frame>At least 4 years</time_frame>
  </primary_outcome>
  <primary_outcome>
    <measure>Number of participants with safety events related to inhibitor development</measure>
    <time_frame>At least 4 years</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Number of adverse reactions (ARs) that are defined within the system organ classes nervous system and psychiatric disorders</measure>
    <time_frame>At least 4 years</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of adverse reactions (ARs) related to hepatic or renal function</measure>
    <time_frame>At least 4 years</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in creatinine</measure>
    <time_frame>At least 4 years</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in estimated glomerular filtration rate (eGFR)</measure>
    <time_frame>At least 4 years</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in alanine transaminase (ALT)</measure>
    <time_frame>At least 4 years</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in aspartate aminotransferase (AST)</measure>
    <time_frame>At least 4 years</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Change from baseline in bilirubin</measure>
    <time_frame>At least 4 years</time_frame>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Testing for PEG plasma levels (baseline and end of study)</measure>
    <time_frame>At least 4 years</time_frame>
    <description>PEG (Polyethylene Glycol)-plasma levels at baseline and end of study will be analyzed only if PEG-plasma levels were collected in local routine clinical practice at the investigator's discretion.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of patients with abnormal findings as assessed by neurological examination</measure>
    <time_frame>At least 4 years</time_frame>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">62</enrollment>
  <condition>Hemophilia A</condition>
  <arm_group>
    <arm_group_label>Damoctocog alfa pegol</arm_group_label>
    <description>Participants with hemophilia A received damoctocog alfa pegol as prophylaxis treatment prescribed by the physician as part of normal clinical practice.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>Damoctocog alfa pegol (Jivi, BAY94-9027)</intervention_name>
    <description>Different prophylaxis regimens with damoctocog alfa pegol following approved local labels or any other regimen prescribed by the physician as part of normal clinical practice</description>
    <arm_group_label>Damoctocog alfa pegol</arm_group_label>
  </intervention>
  <eligibility>
    <study_pop>
      <textblock>
        All previously treated patients (PTPs) with hemophilia A receiving prophylaxis with&#xD;
        damoctocog alfa pegol will be eligible to be enrolled in the study. Indications and&#xD;
        contra-indications according to the local market authorization will be carefully considered&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Signed informed consent/assent will be obtained before any study-related activities&#xD;
&#xD;
          -  PTPs with hemophilia A assigned to Jivi prophylaxis treatment&#xD;
&#xD;
          -  Negative FVIII inhibitor test before study entry&#xD;
&#xD;
          -  Decision to initiate treatment with commercially available Jivi has been made by the&#xD;
             treating physician before and independently from the decision to include the patient&#xD;
             in this study&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Known or suspected contraindications to Jivi or related products&#xD;
&#xD;
          -  Mental incapacity, unwillingness or other barriers precluding adequate understanding&#xD;
             or cooperation&#xD;
&#xD;
          -  Participation in an investigational program with interventions outside of routine&#xD;
             clinical practice&#xD;
      </textblock>
    </criteria>
    <gender>All</gender>
    <minimum_age>12 Years</minimum_age>
    <maximum_age>N/A</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <location>
    <facility>
      <name>Many Locations</name>
      <address>
        <city>Multiple Locations</city>
        <country>Austria</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Many Locations</name>
      <address>
        <city>Multiple Locations</city>
        <country>Germany</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Many Locations</name>
      <address>
        <city>Multiple Locations</city>
        <country>Greece</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Many Locations</name>
      <address>
        <city>Multiple Locations</city>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Many Locations</name>
      <address>
        <city>Multiple Locations</city>
        <country>Russian Federation</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Many Locations</name>
      <address>
        <city>Multiple Locations</city>
        <country>Slovenia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Many Locations</name>
      <address>
        <city>Multiple Locations</city>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Austria</country>
    <country>Germany</country>
    <country>Greece</country>
    <country>Italy</country>
    <country>Russian Federation</country>
    <country>Slovenia</country>
    <country>Spain</country>
  </location_countries>
  <removed_countries>
    <country>Belgium</country>
    <country>Denmark</country>
    <country>France</country>
    <country>Luxembourg</country>
    <country>Netherlands</country>
    <country>Norway</country>
    <country>Sweden</country>
    <country>United Kingdom</country>
  </removed_countries>
  <verification_date>September 2023</verification_date>
  <study_first_submitted>June 18, 2020</study_first_submitted>
  <study_first_submitted_qc>July 2, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 8, 2020</study_first_posted>
  <last_update_submitted>September 6, 2023</last_update_submitted>
  <last_update_submitted_qc>September 6, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">September 7, 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>Factor VIII</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>No</sharing_ipd>
    <ipd_description>Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA &quot;Principles for responsible clinical trial data sharing&quot;. This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.&#xD;
Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.</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/NCT04461639
org study id: 20904
nct id: NCT04461639
lead sponsor: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
In this observational study researchers want to learn more about the safety of drug Jivi over
      a long period of time. Jivi (generic name: Damoctocog alfa pegol) is an approved blood
      clotting Factor VIII (FVIII) medication for the treatment of hemophilia A (bleeding disorder
      resulting from a lack of FVIII). It is manufactured via recombinant technology and has an
      extended half-live, i.e. it will stay longer in the body than other FVIII products. Therefore
      Jivi acts longer in the body which reduces the frequency of drug injections. This study will
      enroll previously treated patients with hemophilia A who are receiving Jivi regularly at
      their treating doctors to prevent bleeding. Observation for each patient will last for at
      least 4 years, and medical data will be collected during patients' routine visits at their
      treating doctors.
observational model: Cohort
time perspective: Prospective
measure: Number of participants with safety events
time frame: At least 4 years
measure: Duration of safety events
time frame: At least 4 years
measure: Number of participants with safety events leading to a change of treatment
time frame: At least 4 years
measure: Number of participants with safety events per intensity
time frame: At least 4 years
description: The maximum intensity of each safety event should be assigned to one of the following categories: mild, moderate or severe
measure: Number of participants with safety events with outcome of death
time frame: At least 4 years
measure: Number of participants with safety events related to inhibitor development
time frame: At least 4 years
measure: Number of adverse reactions (ARs) that are defined within the system organ classes nervous system and psychiatric disorders
time frame: At least 4 years
measure: Number of adverse reactions (ARs) related to hepatic or renal function
time frame: At least 4 years
measure: Change from baseline in creatinine
time frame: At least 4 years
measure: Change from baseline in estimated glomerular filtration rate (eGFR)
time frame: At least 4 years
measure: Change from baseline in alanine transaminase (ALT)
time frame: At least 4 years
measure: Change from baseline in aspartate aminotransferase (AST)
time frame: At least 4 years
measure: Change from baseline in bilirubin
time frame: At least 4 years
measure: Testing for PEG plasma levels (baseline and end of study)
time frame: At least 4 years
description: PEG (Polyethylene Glycol)-plasma levels at baseline and end of study will be analyzed only if PEG-plasma levels were collected in local routine clinical practice at the investigator's discretion.
measure: Number of patients with abnormal findings as assessed by neurological examination
time frame: At least 4 years
arm group label: Damoctocog alfa pegol
description: Participants with hemophilia A received damoctocog alfa pegol as prophylaxis treatment prescribed by the physician as part of normal clinical practice.
intervention type: Drug
intervention name: Damoctocog alfa pegol (Jivi, BAY94-9027)
description: Different prophylaxis regimens with damoctocog alfa pegol following approved local labels or any other regimen prescribed by the physician as part of normal clinical practice
arm group label: Damoctocog alfa pegol
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: All
minimum age: 12 Years
maximum age: N/A
healthy volunteers: No
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: Austria
country: Germany
country: Greece
country: Italy
country: Russian Federation
country: Slovenia
country: Spain
country: Belgium
country: Denmark
country: France
country: Luxembourg
country: Netherlands
country: Norway
country: Sweden
country: United Kingdom
responsible party type: Sponsor
mesh term: Hemophilia A
mesh term: Factor VIII
sharing ipd: No
ipd description: Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014.
Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.