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Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders

Sponsor
Catalyst Biosciences (Industry)
Overall Status
Terminated
CT.gov ID
NCT04548791
Collaborator
(none)
19
Enrollment
20
Locations
3
Arms
6.6
Actual Duration (Months)
1
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of the trial is to evaluate the PK, bioavailability, PD, efficacy and safety of MarzAA for on demand treatment and control of bleeding episodes in adult subjects with inherited bleeding disorders.

Condition or Disease Intervention/Treatment Phase
  • Biological: Coagulation Factor VIIa variant
 Phase 1/Phase 2

Study Design

Study Type:
Interventional
Actual Enrollment :
19 participants
Allocation:
Non-Randomized
Intervention Model:
Sequential Assignment
Masking:
None (Open Label)
Primary Purpose:
Treatment
Official Title:
Phase 1/2 Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Efficacy of Marzeptacog Alfa (Activated) in Treatment of Episodic Bleeding in Subjects With Inherited Bleeding Disorders
Actual Study Start Date :
May 17, 2021
Actual Primary Completion Date :
Nov 15, 2021
Actual Study Completion Date :
Dec 3, 2021

Arms and Interventions

Arm Intervention/Treatment
Experimental: Cohort 1

For Phase 1, a Coagulation Factor VIIa variant by intravenous route, 18 μg/kg, followed by ascending doses by subcutaneous route, 10 μg/kg, 20 μg/kg, 30 μg/kg, 40 μg/kg, and 60 μg/kg, for PK and PD assessment. For Phase 2, Coagulation Factor VIIa, 20 μg/kg by subcutaneous route, administered on demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis.

Biological: Coagulation Factor VIIa variant
Single intravenous dose and ascending doses of subcutaneous injection of MarzAA, followed by a fixed dose of MarzAA for the treatment of bleeding episodes
Other Names:
  • MarzAA

    		•
    Experimental: Cohort 2

    For Phase 1, a Coagulation Factor VIIa variant by intravenous route, 18 μg/kg, followed by ascending doses by subcutaneous route, 30 μg/kg, 45 μg/kg, 60 μg/kg, 120 μg/kg, and 180 μg/kg, for PK and PD assessment. For Phase 2, Coagulation Factor VIIa, 60 μg/kg by subcutaneous route, administered on demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis.

    Biological: Coagulation Factor VIIa variant
    Single intravenous dose and ascending doses of subcutaneous injection of MarzAA, followed by a fixed dose of MarzAA for the treatment of bleeding episodes
    Other Names:
  • MarzAA

    		•
    Experimental: Cohort 3

    For Phase 1, a Coagulation Factor VIIa variant by intravenous route, 18 μg/kg, followed by ascending doses by subcutaneous route, 30 μg/kg, 45 μg/kg, 60 μg/kg, 120 μg/kg, and 180 μg/kg, for PK and PD assessment. For Phase 2, Coagulation Factor VIIa, 60 μg/kg by subcutaneous route, administered on demand during bleeding episodes for a maximum of 3 doses as needed for hemostasis.

    Biological: Coagulation Factor VIIa variant
    Single intravenous dose and ascending doses of subcutaneous injection of MarzAA, followed by a fixed dose of MarzAA for the treatment of bleeding episodes
    Other Names:
  • MarzAA

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Comparative MarzAA activity by dose level/stage and confirm the Phase 2 dose [Dosing period for each stage in a cohort will be approximately 5 to 11 days]

      Comparative pharmacokinetics by dose level/stage based on examination of AUX for each of the dose groups in each cohort.

    2. Bleeding episode treatment success [24 hours after the first administration of study drug]

      Proportion of bleeding events treated with MarzAA achieving hemostatic efficacy based on a four-point scale according to the Investigator's assessment

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    12 Years and Older
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Diagnosis of cohort: FVII deficiency, Glanzmann Thrombasthenia, or hemophilia A with inhibitors

    • Male or female, age 12 or older

    • History of frequent bleeding episodes

    • Affirmation of informed consent with signature confirmation and assent for children between ages 12 to 17 before any study related activities

    • Agreement to use highly effective birth control throughout the study if the subject has childbearing potential

    Exclusion Criteria:

    • Genotype of FVIID subjects with identified mutations by central lab at screening

    • Previous participation in a clinical trial evaluating a modified rFVIIa agent

    • Received an investigational drug within 30 days or 5 half-lives or absence of clinical effect, whichever is longer

    • Known hypersensitivity to trial or related product

    • Known positive antibody to FVII or FVIIa detected by central lab at screening

    • Be immunosuppressed

    • Significant contraindication to participate

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 UC Davis Medical Center Sacramento California United States 95817
    2 University of California -San Francisco San Francisco California United States 94143
    3 University of Colorado Hemophilia and Thrombosis Center Aurora Colorado United States 80045
    4 Rush University Chicago Illinois United States 60612
    5 Children's Hospital of Michigan Detroit Michigan United States 48201
    6 Michigan State University Center for Bleeding Disorders & Clotting Disorders East Lansing Michigan United States 49805
    7 East Carolina University Greenville North Carolina United States 27858
    8 Mazumdar Shaw Medical Centre Bengaluru India
    9 St. John's Medical College Hospital Bengaluru India
    10 Amrita Institute of Medical Sciences and Research Centre Kochi India
    11 K. J. Somaiya Hospital and Research Centre Mumbai India
    12 Sahyadri Super Speciality Hospital Pune India
    13 Careggi University Hospital Florence Italy
    14 Center for Thrombosis and Haemorrhagic Diseases Milan Italy
    15 Maggiore Polyclinic Hospital, IRCCS Ca' Granda Milan Italy
    16 Children's Hospital BambiNo Gesù, IRCCS (PEDS) Roma Italy
    17 City of Health and Science of Turin Turin Italy
    18 Territorial Clinical Hospital Barnaul Russian Federation
    19 National Medical Hematology Research Center under the Ministry of Healthcare of the Russian Federation Moscow Russian Federation
    20 Institute of Blood Pathology and Transfusion Medicine, Department of Surgery and Clinical Transfusiology Lviv Ukraine

    Sponsors and Collaborators

    • Catalyst Biosciences

    Investigators

    None specified.

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Catalyst Biosciences
    ClinicalTrials.gov Identifier:
    NCT04548791
    Other Study ID Numbers:
    • MAA-202
    First Posted:
    Sep 16, 2020
    Last Update Posted:
    Dec 21, 2021
    Last Verified:
    Dec 1, 2021
    Individual Participant Data (IPD) Sharing Statement:
    No
    Plan to Share IPD:
    No
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Hemostatic Disorders
    Hemophilia A
    Blood Coagulation Disorders
    Thrombasthenia
    Factor VII Deficiency

    Study Results

    No Results Posted as of Dec 21, 2021