<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/NCT03006965</url>
  </required_header>
  <id_info>
    <org_study_id>PK HemoA-SP</org_study_id>
    <secondary_id>STH-OCT-2016-01</secondary_id>
    <nct_id>NCT03006965</nct_id>
  </id_info>
  <brief_title>Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using myPKFiT®</brief_title>
  <official_title>Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using an Online Medical Application Based on a Published Population Model and a Bayesian Algorithm</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Spanish Society of Thrombosis and Haemostasis</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
    <collaborator>
      <agency>Takeda</agency>
      <agency_class>Industry</agency_class>
    </collaborator>
    <collaborator>
      <agency>Instituto de Investigación Hospital Universitario La Paz</agency>
      <agency_class>Other</agency_class>
    </collaborator>
  </sponsors>
  <source>Spanish Society of Thrombosis and Haemostasis</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 purpose of this study is to describe the pharmacokinetic profile of patients with&#xD;
      hemophilia A in prophylaxis in Spain using myPKFit®&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      This pharmacokinetic profile will be obtained in only in patient using Advate® (INN-octocog&#xD;
      alfa) or Adynovi® (INN- rurioctocog alfa pegol), both recombinant intravenous FVIII products,&#xD;
      with myPKFiT®. This patients will be followed during the period of 12 months, and the&#xD;
      pharmacokinetic will be measured collecting at least 2 blood samples, in which the presence&#xD;
      of factor VIII will be determined by the usual clinical methods: chromogenic method or&#xD;
      coagulative method.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Completed</overall_status>
  <start_date type="Actual">November 11, 2016</start_date>
  <completion_date type="Actual">December 22, 2022</completion_date>
  <primary_completion_date type="Actual">December 22, 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>Pharmacokinetic profile: FVIII half-life measured with MyPKFiT</measure>
    <time_frame>3-4 hours postdose +/- 30 minutes</time_frame>
    <description>Change from Baseline to 12 months</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pharmacokinetic profile: FVIII half-life measured with MyPKFiT</measure>
    <time_frame>24-32 hours postdose +/- 60 minutes</time_frame>
    <description>Change from Baseline to 12 months</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pharmacokinetic profile: FVIII half-life measured with MyPKFiT</measure>
    <time_frame>48 hours postdose +/- 60 minutes</time_frame>
    <description>Change from Baseline to 12 months</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pharmacokinetic profile: FVIII half-life measured with MyPKFiT</measure>
    <time_frame>72 hours postdose</time_frame>
    <description>Change from Baseline to 12 months</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pharmacokinetic profile: Clearance measured with MyPKFiT</measure>
    <time_frame>3-4 hours postdose +/- 30 minutes</time_frame>
    <description>Change from Baseline to 12 months</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pharmacokinetic profile: Clearance measured with MyPKFiT</measure>
    <time_frame>24-32 hours postdose +/- 60 minutes</time_frame>
    <description>Change from Baseline to 12 months</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pharmacokinetic profile: Clearance measured with MyPKFiT</measure>
    <time_frame>48 hours postdose +/- 60 minutes</time_frame>
    <description>Change from Baseline to 12 months</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pharmacokinetic profile: Clearance measured with MyPKFiT</measure>
    <time_frame>72 hours postdose</time_frame>
    <description>Change from Baseline to 12 months</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pharmacokinetic profile: Volume in steady state measured with MyPKFiT</measure>
    <time_frame>3-4 hours postdose +/- 30 minutes</time_frame>
    <description>Change from Baseline to 12 months</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pharmacokinetic profile: Volume in steady state measured with MyPKFiT</measure>
    <time_frame>24-32 hours postdose +/- 60 minutes</time_frame>
    <description>Change from Baseline to 12 months</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pharmacokinetic profile: Volume in steady state measured with MyPKFiT</measure>
    <time_frame>48 hours postdose +/- 60 minutes</time_frame>
    <description>Change from Baseline to 12 months</description>
  </primary_outcome>
  <primary_outcome>
    <measure>Pharmacokinetic profile: Volume in steady state measured with MyPKFiT</measure>
    <time_frame>72 hours postdose</time_frame>
    <description>Change from Baseline to 12 months</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Hemorrhagic risk of physical activity</measure>
    <time_frame>baseline</time_frame>
    <description>Measured with Broderick scale</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Hemorrhagic risk of physical activity</measure>
    <time_frame>month 12</time_frame>
    <description>Measured with Broderick scale</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Joint condition</measure>
    <time_frame>baseline</time_frame>
    <description>Hemophilia Joint Health Score</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Joint condition</measure>
    <time_frame>month 12</time_frame>
    <description>Hemophilia Joint Health Score</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of total bleeding episodes in a year</measure>
    <time_frame>baseline</time_frame>
    <description>Measured by annual bleeding record</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of total bleeding episodes in a year</measure>
    <time_frame>month 12</time_frame>
    <description>Measured by annual bleeding record</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Annual Joint Bleeding Record</measure>
    <time_frame>baseline</time_frame>
    <description>Hemophilia Joint Health Score</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Annual Joint Bleeding Record</measure>
    <time_frame>month 12</time_frame>
    <description>Hemophilia Joint Health Score</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Self-adherence to treatment evaluation</measure>
    <time_frame>month 12</time_frame>
    <description>Measured by VERITAS- PRO scale</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Adverse Events</measure>
    <time_frame>baseline</time_frame>
    <description>Collect and identify adverse events</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Adverse Events</measure>
    <time_frame>month 12</time_frame>
    <description>Collect and identify adverse events</description>
  </secondary_outcome>
  <number_of_groups>1</number_of_groups>
  <enrollment type="Actual">50</enrollment>
  <condition>Hemophilia A</condition>
  <condition>Hemophilia</condition>
  <condition>Factor VIII Deficiency</condition>
  <arm_group>
    <arm_group_label>Hemophilia A patients</arm_group_label>
    <description>Group of patients in prophylactic treatment with Advate® (octocog alfa) or Adynovi® (rurioctocog alfa pegol), or patients using already myPKFit®.&#xD;
Patients will be given a dose of octocog alfa or rurioctocog alfa pegol according to usual clinical practice, and two blood samples will be taken in case of octocog alfa: one sample will be extracted 3-4h postdose (+/- 30 minutes), and the second sample will be extracted 24-32h postdose (+/- 60 minutes). In case of rurioctocog alfa pegol, the first sample is taken in the same conditions than octocog alfa, and the second sample will be extracted 48h postdose (+/- 120 minutes), and other sample post 72h(+/- 120 minutes) optional.</description>
  </arm_group>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>octocog alfa</intervention_name>
    <description>Pharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software</description>
    <arm_group_label>Hemophilia A patients</arm_group_label>
    <other_name>Advate®</other_name>
    <other_name>recombinant intravenous factor VIII</other_name>
  </intervention>
  <intervention>
    <intervention_type>Drug</intervention_type>
    <intervention_name>rurioctocog alfa pegol</intervention_name>
    <description>Pharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software</description>
    <arm_group_label>Hemophilia A patients</arm_group_label>
    <other_name>recombinant intravenous factor VIII</other_name>
  </intervention>
  <biospec_retention>Samples Without DNA</biospec_retention>
  <biospec_descr>
    <textblock>
      Whole blood samples collected in citrate plasma tubes to separate it and measure&#xD;
      FVIII:chromogenic and FVIII:one stage&#xD;
    </textblock>
  </biospec_descr>
  <eligibility>
    <study_pop>
      <textblock>
        Patients diagnosed with hemophilia A on prophylactic treatment with Advate® or Adynovi®&#xD;
      </textblock>
    </study_pop>
    <sampling_method>Non-Probability Sample</sampling_method>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Male patients.&#xD;
&#xD;
          -  Age between 1 and 65 years old.&#xD;
&#xD;
          -  Patients diagnosed with Hemophilia A who are on prophylactic treatment with Advate® or&#xD;
             Adynovi® or adjusted with myPKFIT.&#xD;
&#xD;
          -  Patients older than 18 who have signed the informed consent form.&#xD;
&#xD;
          -  In the case of mature minors, in addition to the consent signed by the legal guardian,&#xD;
             an assent of the minor must be obtain.&#xD;
&#xD;
          -  In the case of patients legally incapable for giving their consent, their primary&#xD;
             caregiver will consent as the patient's legal guardian.&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Withdrawal of informed consent.&#xD;
&#xD;
          -  Patients with any medical or psychological condition that according to the&#xD;
             researcher's criteria prevents them from following the usual clinical practice&#xD;
             procedures.&#xD;
&#xD;
          -  Patients with concomitant diagnosis of other haemostasis disorders.&#xD;
&#xD;
          -  Patients being treated for induction of immunologic tolerance at the time of&#xD;
             inclusion.&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <minimum_age>1 Year</minimum_age>
    <maximum_age>65 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>M Teresa Álvarez Román, MD,PhD</last_name>
    <role>Principal Investigator</role>
    <affiliation>Hospital Universitario La Paz, IdiPAZ, Universidad Autónoma de Madrid</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>Complexo Hospitalario Universitario A Coruña</name>
      <address>
        <city>A Coruna</city>
        <state>A Coruña</state>
        <zip>15006</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Universitario Central de Asturias</name>
      <address>
        <city>Oviedo</city>
        <state>Asturias</state>
        <zip>33011</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Sant Joan de Deu</name>
      <address>
        <city>Esplugues de Llobregat</city>
        <state>Barcelona</state>
        <zip>08950</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Universitario Son Espases</name>
      <address>
        <city>Palma</city>
        <state>Islas Baleares</state>
        <zip>07120</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Universitario Virgen de la Arrixaca</name>
      <address>
        <city>El Palmar</city>
        <state>Murcia</state>
        <zip>30120</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Virgen del Camino</name>
      <address>
        <city>Pamplona</city>
        <state>Navarra</state>
        <zip>31008</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Xeral de Vigo</name>
      <address>
        <city>Vigo</city>
        <state>Pontevedra</state>
        <zip>36212</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Universitario Cruces</name>
      <address>
        <city>Barakaldo</city>
        <state>Vizcaya</state>
        <zip>48903</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Vall D'Hebron</name>
      <address>
        <city>Barcelona</city>
        <zip>08035</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Universitario La Paz</name>
      <address>
        <city>Madrid</city>
        <zip>28046</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Regional Universitario de Málaga</name>
      <address>
        <city>Malaga</city>
        <zip>29010</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Materno-infantil de Málaga</name>
      <address>
        <city>Malaga</city>
        <zip>29011</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Universitario Virgen del Rocío</name>
      <address>
        <city>Sevilla</city>
        <zip>41013</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Universitari i Politècnic La Fe</name>
      <address>
        <city>Valencia</city>
        <zip>46026</zip>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Spain</country>
  </location_countries>
  <verification_date>June 2023</verification_date>
  <study_first_submitted>December 22, 2016</study_first_submitted>
  <study_first_submitted_qc>December 29, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 30, 2016</study_first_posted>
  <last_update_submitted>June 7, 2023</last_update_submitted>
  <last_update_submitted_qc>June 7, 2023</last_update_submitted_qc>
  <last_update_posted type="Actual">June 8, 2023</last_update_posted>
  <responsible_party>
    <responsible_party_type>Sponsor</responsible_party_type>
  </responsible_party>
  <keyword>Hemophilia</keyword>
  <keyword>Hemophilia A</keyword>
  <keyword>Factor VIII</keyword>
  <keyword>Advate</keyword>
  <keyword>Antithrombotic factor</keyword>
  <keyword>Pharmacokinetic dosing</keyword>
  <keyword>Hemarthrosis</keyword>
  <keyword>myPKFiT</keyword>
  <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>
    <mesh_term>BAX 855</mesh_term>
  </intervention_browse>
  <patient_data>
    <sharing_ipd>Undecided</sharing_ipd>
  </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/NCT03006965
org study id: PK HemoA-SP
secondary id: STH-OCT-2016-01
nct id: NCT03006965
lead sponsor: 
collaborator: 
collaborator: 
has dmc: No
is fda regulated drug: No
is fda regulated device: No
The purpose of this study is to describe the pharmacokinetic profile of patients with
      hemophilia A in prophylaxis in Spain using myPKFit®
This pharmacokinetic profile will be obtained in only in patient using Advate® (INN-octocog
      alfa) or Adynovi® (INN- rurioctocog alfa pegol), both recombinant intravenous FVIII products,
      with myPKFiT®. This patients will be followed during the period of 12 months, and the
      pharmacokinetic will be measured collecting at least 2 blood samples, in which the presence
      of factor VIII will be determined by the usual clinical methods: chromogenic method or
      coagulative method.
observational model: Cohort
time perspective: Other
measure: Pharmacokinetic profile: FVIII half-life measured with MyPKFiT
time frame: 3-4 hours postdose +/- 30 minutes
description: Change from Baseline to 12 months
measure: Pharmacokinetic profile: FVIII half-life measured with MyPKFiT
time frame: 24-32 hours postdose +/- 60 minutes
description: Change from Baseline to 12 months
measure: Pharmacokinetic profile: FVIII half-life measured with MyPKFiT
time frame: 48 hours postdose +/- 60 minutes
description: Change from Baseline to 12 months
measure: Pharmacokinetic profile: FVIII half-life measured with MyPKFiT
time frame: 72 hours postdose
description: Change from Baseline to 12 months
measure: Pharmacokinetic profile: Clearance measured with MyPKFiT
time frame: 3-4 hours postdose +/- 30 minutes
description: Change from Baseline to 12 months
measure: Pharmacokinetic profile: Clearance measured with MyPKFiT
time frame: 24-32 hours postdose +/- 60 minutes
description: Change from Baseline to 12 months
measure: Pharmacokinetic profile: Clearance measured with MyPKFiT
time frame: 48 hours postdose +/- 60 minutes
description: Change from Baseline to 12 months
measure: Pharmacokinetic profile: Clearance measured with MyPKFiT
time frame: 72 hours postdose
description: Change from Baseline to 12 months
measure: Pharmacokinetic profile: Volume in steady state measured with MyPKFiT
time frame: 3-4 hours postdose +/- 30 minutes
description: Change from Baseline to 12 months
measure: Pharmacokinetic profile: Volume in steady state measured with MyPKFiT
time frame: 24-32 hours postdose +/- 60 minutes
description: Change from Baseline to 12 months
measure: Pharmacokinetic profile: Volume in steady state measured with MyPKFiT
time frame: 48 hours postdose +/- 60 minutes
description: Change from Baseline to 12 months
measure: Pharmacokinetic profile: Volume in steady state measured with MyPKFiT
time frame: 72 hours postdose
description: Change from Baseline to 12 months
measure: Hemorrhagic risk of physical activity
time frame: baseline
description: Measured with Broderick scale
measure: Hemorrhagic risk of physical activity
time frame: month 12
description: Measured with Broderick scale
measure: Joint condition
time frame: baseline
description: Hemophilia Joint Health Score
measure: Joint condition
time frame: month 12
description: Hemophilia Joint Health Score
measure: Number of total bleeding episodes in a year
time frame: baseline
description: Measured by annual bleeding record
measure: Number of total bleeding episodes in a year
time frame: month 12
description: Measured by annual bleeding record
measure: Annual Joint Bleeding Record
time frame: baseline
description: Hemophilia Joint Health Score
measure: Annual Joint Bleeding Record
time frame: month 12
description: Hemophilia Joint Health Score
measure: Self-adherence to treatment evaluation
time frame: month 12
description: Measured by VERITAS- PRO scale
measure: Adverse Events
time frame: baseline
description: Collect and identify adverse events
measure: Adverse Events
time frame: month 12
description: Collect and identify adverse events
arm group label: Hemophilia A patients
description: Group of patients in prophylactic treatment with Advate® (octocog alfa) or Adynovi® (rurioctocog alfa pegol), or patients using already myPKFit®.
Patients will be given a dose of octocog alfa or rurioctocog alfa pegol according to usual clinical practice, and two blood samples will be taken in case of octocog alfa: one sample will be extracted 3-4h postdose (+/- 30 minutes), and the second sample will be extracted 24-32h postdose (+/- 60 minutes). In case of rurioctocog alfa pegol, the first sample is taken in the same conditions than octocog alfa, and the second sample will be extracted 48h postdose (+/- 120 minutes), and other sample post 72h(+/- 120 minutes) optional.
intervention type: Drug
intervention name: octocog alfa
description: Pharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software
arm group label: Hemophilia A patients
other name: Advate®
other name: recombinant intravenous factor VIII
intervention type: Drug
intervention name: rurioctocog alfa pegol
description: Pharmacokinetic Characterization of the Hemophilia A Population in Spain using an standalone web-based software
arm group label: Hemophilia A patients
other name: recombinant intravenous factor VIII
Whole blood samples collected in citrate plasma tubes to separate it and measure
      FVIII:chromogenic and FVIII:one stage
study pop: 
sampling method: Non-Probability Sample
criteria: 
gender: Male
minimum age: 1 Year
maximum age: 65 Years
healthy volunteers: No
last name: M Teresa Álvarez Román, MD,PhD
role: Principal Investigator
affiliation: Hospital Universitario La Paz, IdiPAZ, Universidad Autónoma de Madrid
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
facility: 
country: Spain
responsible party type: Sponsor
mesh term: Hemophilia A
mesh term: Factor VIII
mesh term: BAX 855
sharing ipd: Undecided