<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/NCT01708564</url>
  </required_header>
  <id_info>
    <org_study_id>GTC-FVIIa-005-11</org_study_id>
    <secondary_id>2012-002285-13</secondary_id>
    <nct_id>NCT01708564</nct_id>
  </id_info>
  <brief_title>A Phase I Safety, Pharmacokinetics and Pharmacodynamics Study of Recombinant Factor VIIa in Adult Patients With Hemophilia A or B</brief_title>
  <acronym>rhFVIIa</acronym>
  <official_title>A Phase 1b, Dose Escalation Study to Assess the Safety, Pharmacokinetics and Pharmacodynamics of Coagulation Factor VIIa (Recombinant) in Congenital Hemophilia A or B Patients</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>rEVO Biologics</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>rEVO Biologics</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      This study will assess the pharmacokinetics and pharmacodynamics of rhFVIIa at three dose&#xD;
      levels. The results will help identify the most optimal doses to take forward to the Phase&#xD;
      2/3 studies where bleedings in hemophilia patients with inhibitors will be treated with&#xD;
      rhFVIIa.&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Completed</overall_status>
  <start_date>October 2012</start_date>
  <completion_date type="Actual">June 2013</completion_date>
  <primary_completion_date type="Actual">June 2013</primary_completion_date>
  <phase>Phase 1</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Non-Randomized</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Basic Science</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Factor VIIa concentration in patient plasma as measured by FVIIa PK and PD assays</measure>
    <time_frame>Up to 36 hours of dosing</time_frame>
  </primary_outcome>
  <secondary_outcome>
    <measure>Incidence of patients with treatment emergent adverse events</measure>
    <time_frame>Up to 28 days after dosing</time_frame>
  </secondary_outcome>
  <number_of_arms>3</number_of_arms>
  <enrollment type="Actual">15</enrollment>
  <condition>Hemophilia</condition>
  <arm_group>
    <arm_group_label>Cohort 1</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>10 patients administered a single low dose of rhFVIIa</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Cohort 2</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>10 patients administered a single intermediate dose of rhFVIIa</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Cohort 3</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>10 patients administered a single high dose of rhFVIIa</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>rhFVIIa</intervention_name>
    <description>Patients will be administered low, intermediate and high doses of rhFVIIa</description>
    <arm_group_label>Cohort 1</arm_group_label>
    <arm_group_label>Cohort 2</arm_group_label>
    <arm_group_label>Cohort 3</arm_group_label>
    <other_name>Coagulation Factor VIIa (Recombinant)</other_name>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. be male with a diagnosis of moderate or severe congenital hemophilia A and/or B (with&#xD;
             or without inhibitors)&#xD;
&#xD;
          2. be 18 years or older, up to and including 75 years of age&#xD;
&#xD;
          3. be capable of understanding and willing to comply with the conditions of the protocol&#xD;
&#xD;
          4. have read, understood and provided written informed consent&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. have any coagulation disorder other than hemophilia A or B&#xD;
&#xD;
          2. have a body weight &gt;105 kg (231 lb)&#xD;
&#xD;
          3. be immuno-suppressed (i.e., the patient should not receive systemic immunosuppressive&#xD;
             medication &lt;30 days prior to enrollment, CD4 counts at screening should be &gt;200/µl)&#xD;
&#xD;
          4. have a known allergy or hypersensitivity to rabbits&#xD;
&#xD;
          5. have platelet count &lt;100,000/mL&#xD;
&#xD;
          6. have had within one month prior to first administration of the study drug in this&#xD;
             study a major surgical procedure (e.g. orthopedic, abdominal)&#xD;
&#xD;
          7. have an active, ongoing bleeding for which the patient is being treated, or treatment&#xD;
             for a bleeding was stopped within 24 hours of the time of study drug administration&#xD;
&#xD;
          8. have received a Factor VII or FVIIa containing product (either plasma derived or&#xD;
             recombinant) within 72 hours prior to any study drug administration&#xD;
&#xD;
          9. have received an investigational drug within 30 days of the first study drug&#xD;
             administration, or is expected to receive such drug during participation in this study&#xD;
&#xD;
         10. have a clinically relevant hepatic (hepatic enzymes &gt;3 times the upper limit of&#xD;
             normal) and/or renal impairment (creatinine &gt;2 times the upper limit of normal)&#xD;
&#xD;
         11. have a history of arterial and/or venous thromboembolic events (such as myocardial&#xD;
             infarction, ischemic strokes, transient ischemic attacks, deep venous thrombosis or&#xD;
             pulmonary embolism) within 2 years prior to first dose of study drug, have an arterial&#xD;
             stent in place or have clinically significant atherosclerotic disease (e.g., angina&#xD;
             pectoris, peripheral vascular disease)&#xD;
&#xD;
         12. use any anticoagulant for arterial/venous obstructions and/or atrial fibrillation&#xD;
             within 7 days prior to first study drug administration&#xD;
&#xD;
         13. have an active malignancy (those with non-melanoma skin cancer are allowed)&#xD;
&#xD;
         14. have any life-threatening disease or other disease or condition which, according to&#xD;
             the investigator's judgment, could imply a potential hazard to the patient, interfere&#xD;
             with the trial participation or trial outcome&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <minimum_age>18 Years</minimum_age>
    <maximum_age>75 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <overall_official>
    <last_name>Johan Frieling, MD,PhD</last_name>
    <role>Study Director</role>
    <affiliation>rEVO Biologics</affiliation>
  </overall_official>
  <location>
    <facility>
      <name>UC Davis Health System Internal Medicine: Hematology &amp; Oncology</name>
      <address>
        <city>Sacramento</city>
        <state>California</state>
        <zip>95817</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>RUSH Hemophilia &amp; Thrombophilia Center</name>
      <address>
        <city>Chicago</city>
        <state>Illinois</state>
        <zip>60612</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Centre for Human Drug Research</name>
      <address>
        <city>Leiden</city>
        <country>Netherlands</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Netherlands</country>
    <country>United States</country>
  </location_countries>
  <verification_date>July 2013</verification_date>
  <study_first_submitted>October 15, 2012</study_first_submitted>
  <study_first_submitted_qc>October 15, 2012</study_first_submitted_qc>
  <study_first_posted type="Estimate">October 17, 2012</study_first_posted>
  <last_update_submitted>July 29, 2013</last_update_submitted>
  <last_update_submitted_qc>July 29, 2013</last_update_submitted_qc>
  <last_update_posted type="Estimate">July 30, 2013</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>
  <!-- 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/NCT01708564
org study id: GTC-FVIIa-005-11
secondary id: 2012-002285-13
nct id: NCT01708564
lead sponsor: 
has dmc: Yes
This study will assess the pharmacokinetics and pharmacodynamics of rhFVIIa at three dose
      levels. The results will help identify the most optimal doses to take forward to the Phase
      2/3 studies where bleedings in hemophilia patients with inhibitors will be treated with
      rhFVIIa.
allocation: Non-Randomized
intervention model: Single Group Assignment
primary purpose: Basic Science
masking: None (Open Label)
measure: Factor VIIa concentration in patient plasma as measured by FVIIa PK and PD assays
time frame: Up to 36 hours of dosing
measure: Incidence of patients with treatment emergent adverse events
time frame: Up to 28 days after dosing
arm group label: Cohort 1
arm group type: Experimental
description: 10 patients administered a single low dose of rhFVIIa
arm group label: Cohort 2
arm group type: Experimental
description: 10 patients administered a single intermediate dose of rhFVIIa
arm group label: Cohort 3
arm group type: Experimental
description: 10 patients administered a single high dose of rhFVIIa
intervention type: Biological
intervention name: rhFVIIa
description: Patients will be administered low, intermediate and high doses of rhFVIIa
arm group label: Cohort 1
arm group label: Cohort 2
arm group label: Cohort 3
other name: Coagulation Factor VIIa (Recombinant)
criteria: 
gender: Male
minimum age: 18 Years
maximum age: 75 Years
healthy volunteers: No
last name: Johan Frieling, MD,PhD
role: Study Director
affiliation: rEVO Biologics
facility: 
facility: 
facility: 
country: Netherlands
country: United States
responsible party type: Sponsor
mesh term: Hemophilia A