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Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Hemophilia A or B

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04489537
Recruitment Status : Terminated (Sponsor decision (not a safety decision))
First Posted : July 28, 2020
Last Update Posted : December 17, 2021
Sponsor:
Information provided by (Responsible Party):
Catalyst Biosciences

Study Description
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Brief Summary:
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).

Condition or disease Intervention/treatment Phase
Hemophilia A With Inhibitor Hemophilia B With Inhibitor Biological: MarzAA Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 18 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : May 4, 2021
Actual Primary Completion Date : November 15, 2021
Actual Study Completion Date : December 1, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Bleeding Hemophilia

Arms and Interventions
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Arm Intervention/treatment
Experimental: MarzAA
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
 Biological: MarzAA
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.
Active Comparator: Standard of Care
Standard of care administered on-demand during bleeding episodes
 Biological: MarzAA
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.


Outcome Measures
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Primary Outcome Measures :
  1. Bleeding episode treatment success [ Time Frame: 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 compared with standard of care.


Eligibility Criteria
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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Diagnosis of congenital hemophilia A or B 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

Exclusion Criteria:

  • 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
  • Known hypersensitivity to trial or related product
  • Known positive antibody to FVII or FVIIa detected by central lab at screening
  • Have a coagulation disorder other than hemophilia A or B
  • Be immunosuppressed
  • Significant contraindication to participate
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04489537


Locations
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Sponsors and Collaborators
Catalyst Biosciences
More Information
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Responsible Party: Catalyst Biosciences
ClinicalTrials.gov Identifier: NCT04489537    
Other Study ID Numbers: MAA-304
First Posted: July 28, 2020    Key Record Dates
Last Update Posted: December 17, 2021
Last Verified: December 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked