<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/NCT04489537</url>
  </required_header>
  <id_info>
    <org_study_id>MAA-304</org_study_id>
    <nct_id>NCT04489537</nct_id>
  </id_info>
  <brief_title>Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Hemophilia A or B</brief_title>
  <official_title>Phase 3 Study to Evaluate the Efficacy and Safety of Subcutaneous Marzeptacog Alfa (Activated) For On Demand Treatment and Control of Bleeding Episodes in Subjects With Hemophilia A or Hemophilia B, With Inhibitors: The Crimson 1 Study</official_title>
  <sponsors>
    <lead_sponsor>
      <agency>Catalyst Biosciences</agency>
      <agency_class>Industry</agency_class>
    </lead_sponsor>
  </sponsors>
  <source>Catalyst Biosciences</source>
  <oversight_info>
    <has_dmc>Yes</has_dmc>
    <is_fda_regulated_drug>Yes</is_fda_regulated_drug>
    <is_fda_regulated_device>No</is_fda_regulated_device>
  </oversight_info>
  <brief_summary>
    <textblock>
      The purpose of the trial is to evaluate the safety and efficacy of MarzAA for on-demand&#xD;
      treatment and control of bleeding episodes in hemophilia A or B patients with inhibitors&#xD;
      compared with their standard of care (SOC).&#xD;
    </textblock>
  </brief_summary>
  <overall_status>Terminated</overall_status>
  <why_stopped>
    Sponsor decision (not a safety decision)&#xD;
  </why_stopped>
  <start_date type="Actual">May 4, 2021</start_date>
  <completion_date type="Actual">December 1, 2021</completion_date>
  <primary_completion_date type="Actual">November 15, 2021</primary_completion_date>
  <phase>Phase 3</phase>
  <study_type>Interventional</study_type>
  <has_expanded_access>No</has_expanded_access>
  <study_design_info>
    <allocation>Randomized</allocation>
    <intervention_model>Crossover Assignment</intervention_model>
    <primary_purpose>Treatment</primary_purpose>
    <masking>None (Open Label)</masking>
  </study_design_info>
  <primary_outcome>
    <measure>Bleeding episode treatment success</measure>
    <time_frame>24 hours after the first administration of study drug</time_frame>
    <description>Proportion of bleeding events treated with MarzAA achieving hemostatic efficacy based on a four-point scale according to the Investigator's assessment compared with standard of care.</description>
  </primary_outcome>
  <number_of_arms>2</number_of_arms>
  <enrollment type="Actual">18</enrollment>
  <condition>Hemophilia A With Inhibitor</condition>
  <condition>Hemophilia B With Inhibitor</condition>
  <arm_group>
    <arm_group_label>MarzAA</arm_group_label>
    <arm_group_type>Experimental</arm_group_type>
    <description>Coagulation Factor VIIa variant, 60 µg/kg by subcutaneous route, administered on-demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis</description>
  </arm_group>
  <arm_group>
    <arm_group_label>Standard of Care</arm_group_label>
    <arm_group_type>Active Comparator</arm_group_type>
    <description>Standard of care administered on-demand during bleeding episodes</description>
  </arm_group>
  <intervention>
    <intervention_type>Biological</intervention_type>
    <intervention_name>MarzAA</intervention_name>
    <description>A cross over design to assess the efficacy of a dosing regimen of 60 µg/kg of MarzAA compared with standard of care for the treatment of bleeding episodes.</description>
    <arm_group_label>MarzAA</arm_group_label>
    <arm_group_label>Standard of Care</arm_group_label>
  </intervention>
  <eligibility>
    <criteria>
      <textblock>
        Inclusion Criteria:&#xD;
&#xD;
          -  Diagnosis of congenital hemophilia A or B with inhibitors&#xD;
&#xD;
          -  Male or Female, age 12 or older&#xD;
&#xD;
          -  History of frequent bleeding episodes&#xD;
&#xD;
          -  Affirmation of informed consent with signature confirmation and assent for children&#xD;
             between ages 12 to 17 before any study related activities&#xD;
&#xD;
        Exclusion Criteria:&#xD;
&#xD;
          -  Previous participation in a clinical trial evaluating a modified rFVIIa agent&#xD;
&#xD;
          -  Received an investigational drug within 30 days or 5 half-lives or absence of clinical&#xD;
             effect&#xD;
&#xD;
          -  Known hypersensitivity to trial or related product&#xD;
&#xD;
          -  Known positive antibody to FVII or FVIIa detected by central lab at screening&#xD;
&#xD;
          -  Have a coagulation disorder other than hemophilia A or B&#xD;
&#xD;
          -  Be immunosuppressed&#xD;
&#xD;
          -  Significant contraindication to participate&#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>Phoenix Children's Hospital</name>
      <address>
        <city>Phoenix</city>
        <state>Arizona</state>
        <zip>85016</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Lombardi Comprehensive Cancer Center</name>
      <address>
        <city>Washington</city>
        <state>District of Columbia</state>
        <zip>20007</zip>
        <country>United States</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hematology Center after Prof. R. Yeolyan</name>
      <address>
        <city>Yerevan</city>
        <country>Armenia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>JSC &quot;K.Eristavi National Center of Experimental and Clinical Surgery&quot;</name>
      <address>
        <city>Tbilisi</city>
        <country>Georgia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Magyar Honvédség Egészségügyi Központ, Országos Haemophilia Központ</name>
      <address>
        <city>Budapest</city>
        <country>Hungary</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Nirmal Hospital</name>
      <address>
        <city>Gujrat</city>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>K.L.E.S Dr. Prabhakar Kore Hospital and Medical Research Centre</name>
      <address>
        <city>Karnad</city>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Sanjay Gandhi Postgraduate Institute of Medical Sciences</name>
      <address>
        <city>Lucknow</city>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>K.J Somaiya Hospital and Research Centre</name>
      <address>
        <city>Mumbai</city>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>MCGM - Comprehensive Thalassemia Care, Pediatric Hematology-Oncology &amp; BMT Centre</name>
      <address>
        <city>Mumbai</city>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Sahyadri Specialty Hospital</name>
      <address>
        <city>Mumbai</city>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Grant Medical Foundation, Ruby Hall Clinic</name>
      <address>
        <city>Pune</city>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>All India Institute of Medical Sciences, Rishikesh</name>
      <address>
        <city>Rishīkesh</city>
        <country>India</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Azienda Ospedaliero-Universitaria Careggi</name>
      <address>
        <city>Firenze</city>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano</name>
      <address>
        <city>Milan</city>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Azienda Ospedaliera Universitaria Federico II</name>
      <address>
        <city>Napoli</city>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Citta della Salute e della Scienza di Torino - Ospedale le Molinette</name>
      <address>
        <city>Torino</city>
        <country>Italy</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Ampang</name>
      <address>
        <city>Ampang</city>
        <country>Malaysia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Raja Perempuan Zainab II Hospital</name>
      <address>
        <city>Kota Bharu</city>
        <country>Malaysia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Queen Elizabeth Hospital Malaysia</name>
      <address>
        <city>Kota Kinabalu</city>
        <country>Malaysia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Tunku Azizah Kuala Lumpur</name>
      <address>
        <city>Kuala Lumpur</city>
        <country>Malaysia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Tuanku Ja'afar Hospital, Seremban</name>
      <address>
        <city>Sungai Petani</city>
        <country>Malaysia</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Instituto Nacional de Pediatria</name>
      <address>
        <city>Ciudad de mexico</city>
        <country>Mexico</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Instituto de Investigaciones Aplicada a la Neurociencia A.C.</name>
      <address>
        <city>Durango</city>
        <country>Mexico</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Investigación e Innovación en Medicina Traslacional S.A.P.I. de C.V.</name>
      <address>
        <city>Mexico City</city>
        <country>Mexico</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Korczowski Bartosz, Gabinet Lekarski</name>
      <address>
        <city>Rzeszów</city>
        <country>Poland</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Kirov Research Institute of Hematology and Blood Transfusion</name>
      <address>
        <city>Kirov</city>
        <country>Russian Federation</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Medis, Llc</name>
      <address>
        <city>Nizhny Novgorod</city>
        <country>Russian Federation</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Haemophilia Comprehensive Care Centre</name>
      <address>
        <city>Johannesburg</city>
        <country>South Africa</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital Universitario Vall d'Hebron</name>
      <address>
        <city>Barcelona</city>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Hospital de Malaga</name>
      <address>
        <city>Málaga</city>
        <country>Spain</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Changhua Christian Hospital (CCH)</name>
      <address>
        <city>Changhua City</city>
        <country>Taiwan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Taichung Veterans General Hospital</name>
      <address>
        <city>Taichung</city>
        <country>Taiwan</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Akdeniz University Medical Faculty Hospital</name>
      <address>
        <city>Antalya</city>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Istanbul University Faculty of Medicine</name>
      <address>
        <city>Istanbul</city>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Ege University Medical Faculty</name>
      <address>
        <city>İzmir</city>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Özel Acibadem Adana Hastanesi</name>
      <address>
        <city>Seyhan</city>
        <country>Turkey</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>&quot;Cherkasy Regional Oncological Dispensary of Cherkasy Regional Council&quot;, Regional Treatment and Diagnostic Center of Hematology</name>
      <address>
        <city>Cherkasy</city>
        <country>Ukraine</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>&quot;Kyiv City Clinical Hospital #9&quot; of Executive Body of Kyiv City Council (Kyiv City State Administration), City Scientific-Practical Centre of Diagnostics and Treatment</name>
      <address>
        <city>Kyiv</city>
        <country>Ukraine</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>&quot;Institute of Blood Pathology and Transfusion Medicine of National Academy of Medical Sciences of Ukraine&quot;, Haematology Department</name>
      <address>
        <city>Lviv</city>
        <country>Ukraine</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Queen Elizabeth Hospital Birmingham</name>
      <address>
        <city>Birmingham</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>Royal Liverpool University Hospital</name>
      <address>
        <city>Liverpool</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location>
    <facility>
      <name>University Hospital Southampton NHS Foundation Trust</name>
      <address>
        <city>Southampton</city>
        <country>United Kingdom</country>
      </address>
    </facility>
  </location>
  <location_countries>
    <country>Armenia</country>
    <country>Georgia</country>
    <country>Hungary</country>
    <country>India</country>
    <country>Italy</country>
    <country>Malaysia</country>
    <country>Mexico</country>
    <country>Poland</country>
    <country>Russian Federation</country>
    <country>South Africa</country>
    <country>Spain</country>
    <country>Taiwan</country>
    <country>Turkey</country>
    <country>Ukraine</country>
    <country>United Kingdom</country>
    <country>United States</country>
  </location_countries>
  <verification_date>December 2021</verification_date>
  <study_first_submitted>July 24, 2020</study_first_submitted>
  <study_first_submitted_qc>July 24, 2020</study_first_submitted_qc>
  <study_first_posted type="Actual">July 28, 2020</study_first_posted>
  <last_update_submitted>December 2, 2021</last_update_submitted>
  <last_update_submitted_qc>December 2, 2021</last_update_submitted_qc>
  <last_update_posted type="Actual">December 17, 2021</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>
    <mesh_term>Hemophilia B</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/NCT04489537
org study id: MAA-304
nct id: NCT04489537
lead sponsor: 
has dmc: Yes
is fda regulated drug: Yes
is fda regulated device: No
The purpose of the trial is to evaluate the safety and efficacy of MarzAA for on-demand
      treatment and control of bleeding episodes in hemophilia A or B patients with inhibitors
      compared with their standard of care (SOC).
allocation: Randomized
intervention model: Crossover Assignment
primary purpose: Treatment
masking: None (Open Label)
measure: Bleeding episode treatment success
time frame: 24 hours after the first administration of study drug
description: Proportion of bleeding events treated with MarzAA achieving hemostatic efficacy based on a four-point scale according to the Investigator's assessment compared with standard of care.
arm group label: MarzAA
arm group type: Experimental
description: Coagulation Factor VIIa variant, 60 µg/kg by subcutaneous route, administered on-demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis
arm group label: Standard of Care
arm group type: Active Comparator
description: Standard of care administered on-demand during bleeding episodes
intervention type: Biological
intervention name: MarzAA
description: A cross over design to assess the efficacy of a dosing regimen of 60 µg/kg of MarzAA compared with standard of care for the treatment of bleeding episodes.
arm group label: MarzAA
arm group label: Standard of Care
criteria: 
gender: All
minimum age: 12 Years
maximum age: N/A
healthy volunteers: No
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country: Armenia
country: Georgia
country: Hungary
country: India
country: Italy
country: Malaysia
country: Mexico
country: Poland
country: Russian Federation
country: South Africa
country: Spain
country: Taiwan
country: Turkey
country: Ukraine
country: United Kingdom
country: United States
responsible party type: Sponsor
mesh term: Hemophilia A
mesh term: Hemophilia B
sharing ipd: No