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

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ClinicalTrials.gov Identifier: NCT04548791
Recruitment Status : Recruiting
First Posted : September 16, 2020
Last Update Posted : June 10, 2021
Sponsor:
Information provided by (Responsible Party):
Catalyst Biosciences

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
Factor VII Deficiency Glanzmann Thrombasthenia Hemophilia A With Inhibitor Biological: Coagulation Factor VIIa variant Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 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
Estimated Primary Completion Date : December 2021
Estimated Study Completion Date : March 2022


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 Name: 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 Name: 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 Name: MarzAA




Primary Outcome Measures :
  1. Comparative MarzAA activity by dose level/stage and confirm the Phase 2 dose [ Time Frame: 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 [ 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



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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 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

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): NCT04548791


Contacts
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Contact: Howard Levy, MD, PhD, MMM +1.650.266.8671 hlevy@catbio.com
Contact: Linda Neuman, MD +1.650.266.8667 lneuman@catbio.com

Locations
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Sponsors and Collaborators
Catalyst Biosciences
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Responsible Party: Catalyst Biosciences
ClinicalTrials.gov Identifier: NCT04548791    
Other Study ID Numbers: MAA-202
First Posted: September 16, 2020    Key Record Dates
Last Update Posted: June 10, 2021
Last Verified: June 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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Hemostatic Disorders
Hemophilia A
Blood Coagulation Disorders
Thrombasthenia
Factor VII Deficiency
Blood Coagulation Disorders, Inherited
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Vascular Diseases
Cardiovascular Diseases
Blood Platelet Disorders