<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/NCT03002480</url>
  </required_header>
  <id_info>
    <org_study_id>16-00501</org_study_id>
    <nct_id>NCT03002480</nct_id>
  </id_info>
  <brief_title>Individualizing Hemophilia Bypassing Agent Therapy Utilizing Thromboelastography</brief_title>
  <official_title>Individualizing Bypassing Agent Therapy Utilizing TEG in Hemophilia Patients With Inhibitors</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Children's Hospital Los Angeles</agency>
      <agency_class>Other</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Children's Hospital Los Angeles</source>
  <oversight_info>
    <has_dmc>No</has_dmc>
  </oversight_info>
  <brief_summary>
    <textblock>
      This pilot study will investigate the use of thromboelastograph (TEG) to determine the&#xD;
      primary bypassing agent for the management of bleeding in children and adults severe&#xD;
      hemophilia A patients with inhibitors.&#xD;
&#xD;
      The study will evaluate the TEG profile for each of the available bypassing agents (FEIBA and&#xD;
      rFVIIa) in each participating patient, which will then determine which agent provides the&#xD;
      most robust clot formation as measured by the TEG. This study will consist of screening visit&#xD;
      and 2-4 pharmacokinetic studies to determine the &quot;best&quot; bypassing agent based on the TEG&#xD;
      results. Patients will then be assigned that bypassing agent and dose for the treatment of&#xD;
      their bleeding episodes (Prophylaxis or On-demand). Each patient will be then followed for a&#xD;
      period of 6 months to monitor short-term safety of those patients whose bypassing agent was&#xD;
      modified.&#xD;
    </textblock>
  </brief_summary>
  <detailed_description>
    <textblock>
      Currently, there are no laboratory assays that can assess the pharmaceutical activity of the&#xD;
      available agents known collectively as bypassing agents, which negatively impacts the&#xD;
      treating physician's ability to manage these patients, leaving decisions on the dosing&#xD;
      regimen to trial and error. Both agents, FEIBA and recombinant activated factor VII (rFVIIa),&#xD;
      are approved for the treatment of bleeding in inhibitor patients with recommended dosing&#xD;
      regimens that are based on clinical trials. However, a significant amount of &quot;experimenting&quot;&#xD;
      with other dosing regimens is used by physicians and patients.&#xD;
&#xD;
      The goal of this project is to demonstrate that TEG is an excellent biomarker for predicting,&#xD;
      managing and individualizing the treatment with FEIBA and rFVIIa of this rare and difficult&#xD;
      to treat patient population.&#xD;
    </textblock>
  </detailed_description>
  <overall_status>Withdrawn</overall_status>
  <why_stopped>
    Study never opened.&#xD;
  </why_stopped>
  <start_date type="Anticipated">January 2017</start_date>
  <completion_date type="Anticipated">January 2019</completion_date>
  <primary_completion_date type="Anticipated">January 2019</primary_completion_date>
  <phase>N/A</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>N/A</allocation>
    <intervention_model>Single Group Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Elastic properties of a forming clot using thromboelastograph to determine best bypassing agent</measure>
    <time_frame>6 months</time_frame>
    <description>This study will assess the feasibility of TEG-guided individualization of bypassing agent treatment of bleeding for severe hemophilia A patients with inhibitors.</description>
  </primary_outcome>
  <secondary_outcome>
    <measure>Thrombin generation assay measuring the formation of thrombin during clot formation</measure>
    <time_frame>6 months</time_frame>
    <description>Thrombin generation assays will be performed to provide additional evidence supporting the TEG-guided individualization of bypassing agent treatment.</description>
  </secondary_outcome>
  <secondary_outcome>
    <measure>Number of participants with treatment-related adverse events</measure>
    <time_frame>1 year</time_frame>
    <description>The study will monitor short-term safety of patients whose bypassing agent treatment has been modified.</description>
  </secondary_outcome>
  <number_of_arms>1</number_of_arms>
  <enrollment type="Actual">0</enrollment>
  <condition>Hemophilia A With Inhibitors</condition>
  <arm_group>
    <arm_group_label>Severe Hemophilia A subjects w/ inhibitors</arm_group_label>
    <arm_group_type>Other</arm_group_type>
    <description>Males age 4-70 years diagnosed with severe hemophilia A with inhibitors who are currently treated with prophylaxis or on-demand treatment will be enrolled.</description>
  </arm_group>
  <intervention>
    <intervention_type>Other</intervention_type>
    <intervention_name>Thromboelastography-Guided Treatment Regimen</intervention_name>
    <description>The thromboelastograph (TEG) will be used to determine the &quot;best&quot; bypassing agent for the management of subjects with severe hemophilia A with inhibitors. Patients will be assigned that bypassing agent and dose for the treatment of their bleeding episodes and subsequently will be followed for a period of 6 months to determine their annual bleeding rate.</description>
    <arm_group_label>Severe Hemophilia A subjects w/ inhibitors</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          1. Males, 4-60 years old, inclusive on Prophylaxis or On-Demand treatment&#xD;
&#xD;
          2. Diagnosis of Hemophilia with active titer inhibitors (&gt; 0.6 BU)&#xD;
&#xD;
          3. Willing to alter their treatment regimen per study protocol&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          1. Bleeding disorder(s) other than hemophilia A with inhibitors&#xD;
&#xD;
          2. Thrombocytopenia (platelet count &lt;100,000K/µL)&#xD;
&#xD;
          3. Any concurrent clinically significant major disease that, in the opinion of the&#xD;
             investigator, would make the subject unsuitable for enrollment&#xD;
&#xD;
          4. Participation within the past 30 days in a clinical study involving investigational&#xD;
             drugs&#xD;
&#xD;
          5. Planned major surgery within 30 days prior to screening or during the study period&#xD;
      </textblock>
    </criteria>
    <gender>Male</gender>
    <minimum_age>4 Years</minimum_age>
    <maximum_age>70 Years</maximum_age>
    <healthy_volunteers>No</healthy_volunteers>
  </eligibility>
  <verification_date>January 2018</verification_date>
  <study_first_submitted>December 19, 2016</study_first_submitted>
  <study_first_submitted_qc>December 21, 2016</study_first_submitted_qc>
  <study_first_posted type="Estimate">December 23, 2016</study_first_posted>
  <last_update_submitted>January 26, 2018</last_update_submitted>
  <last_update_submitted_qc>January 26, 2018</last_update_submitted_qc>
  <last_update_posted type="Actual">January 30, 2018</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>
  <patient_data>
    <sharing_ipd>No</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/NCT03002480
org study id: 16-00501
nct id: NCT03002480
lead sponsor: 
has dmc: No
This pilot study will investigate the use of thromboelastograph (TEG) to determine the
      primary bypassing agent for the management of bleeding in children and adults severe
      hemophilia A patients with inhibitors.

      The study will evaluate the TEG profile for each of the available bypassing agents (FEIBA and
      rFVIIa) in each participating patient, which will then determine which agent provides the
      most robust clot formation as measured by the TEG. This study will consist of screening visit
      and 2-4 pharmacokinetic studies to determine the "best" bypassing agent based on the TEG
      results. Patients will then be assigned that bypassing agent and dose for the treatment of
      their bleeding episodes (Prophylaxis or On-demand). Each patient will be then followed for a
      period of 6 months to monitor short-term safety of those patients whose bypassing agent was
      modified.
Currently, there are no laboratory assays that can assess the pharmaceutical activity of the
      available agents known collectively as bypassing agents, which negatively impacts the
      treating physician's ability to manage these patients, leaving decisions on the dosing
      regimen to trial and error. Both agents, FEIBA and recombinant activated factor VII (rFVIIa),
      are approved for the treatment of bleeding in inhibitor patients with recommended dosing
      regimens that are based on clinical trials. However, a significant amount of "experimenting"
      with other dosing regimens is used by physicians and patients.

      The goal of this project is to demonstrate that TEG is an excellent biomarker for predicting,
      managing and individualizing the treatment with FEIBA and rFVIIa of this rare and difficult
      to treat patient population.
allocation: N/A
intervention model: Single Group Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Elastic properties of a forming clot using thromboelastograph to determine best bypassing agent
time frame: 6 months
description: This study will assess the feasibility of TEG-guided individualization of bypassing agent treatment of bleeding for severe hemophilia A patients with inhibitors.
measure: Thrombin generation assay measuring the formation of thrombin during clot formation
time frame: 6 months
description: Thrombin generation assays will be performed to provide additional evidence supporting the TEG-guided individualization of bypassing agent treatment.
measure: Number of participants with treatment-related adverse events
time frame: 1 year
description: The study will monitor short-term safety of patients whose bypassing agent treatment has been modified.
arm group label: Severe Hemophilia A subjects w/ inhibitors
arm group type: Other
description: Males age 4-70 years diagnosed with severe hemophilia A with inhibitors who are currently treated with prophylaxis or on-demand treatment will be enrolled.
intervention type: Other
intervention name: Thromboelastography-Guided Treatment Regimen
description: The thromboelastograph (TEG) will be used to determine the "best" bypassing agent for the management of subjects with severe hemophilia A with inhibitors. Patients will be assigned that bypassing agent and dose for the treatment of their bleeding episodes and subsequently will be followed for a period of 6 months to determine their annual bleeding rate.
arm group label: Severe Hemophilia A subjects w/ inhibitors
criteria: 
gender: Male
minimum age: 4 Years
maximum age: 70 Years
healthy volunteers: No
responsible party type: Sponsor
mesh term: Hemophilia A
sharing ipd: No