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

Primary Purpose

Factor VII Deficiency, Glanzmann Thrombasthenia, Hemophilia A With Inhibitor

Status
Terminated
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
Coagulation Factor VIIa variant
Sponsored by
Catalyst Biosciences
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Factor VII Deficiency

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • UC Davis Medical Center
  • University of California -San Francisco
  • University of Colorado Hemophilia and Thrombosis Center
  • Rush University
  • Children's Hospital of Michigan
  • Michigan State University Center for Bleeding Disorders & Clotting Disorders
  • East Carolina University
  • Mazumdar Shaw Medical Centre
  • St. John's Medical College Hospital
  • Amrita Institute of Medical Sciences and Research Centre
  • K. J. Somaiya Hospital and Research Centre
  • Sahyadri Super Speciality Hospital
  • Careggi University Hospital
  • Center for Thrombosis and Haemorrhagic Diseases
  • Maggiore Polyclinic Hospital, IRCCS Ca' Granda
  • Children's Hospital BambiNo Gesù, IRCCS (PEDS)
  • City of Health and Science of Turin
  • Territorial Clinical Hospital
  • National Medical Hematology Research Center under the Ministry of Healthcare of the Russian Federation
  • Institute of Blood Pathology and Transfusion Medicine, Department of Surgery and Clinical Transfusiology

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

Cohort 1

Cohort 2

Cohort 3

Arm Description

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.

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.

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.

Outcomes

Primary Outcome Measures

Comparative MarzAA activity by dose level/stage and confirm the Phase 2 dose
Comparative pharmacokinetics by dose level/stage based on examination of AUX for each of the dose groups in each cohort.
Bleeding episode treatment success
Proportion of bleeding events treated with MarzAA achieving hemostatic efficacy based on a four-point scale according to the Investigator's assessment

Secondary Outcome Measures

Full Information

First Posted
September 8, 2020
Last Updated
December 6, 2021
Sponsor
Catalyst Biosciences
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1. Study Identification

Unique Protocol Identification Number
NCT04548791
Brief Title
Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders
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
Study Type
Interventional

2. Study Status

Record Verification Date
December 2021
Overall Recruitment Status
Terminated
Why Stopped
Company decision (not a safety issue)
Study Start Date
May 17, 2021 (Actual)
Primary Completion Date
November 15, 2021 (Actual)
Study Completion Date
December 3, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Catalyst Biosciences

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Factor VII Deficiency, Glanzmann Thrombasthenia, Hemophilia A With Inhibitor

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Sequential Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
19 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Cohort 1
Arm Type
Experimental
Arm Description
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.
Arm Title
Cohort 2
Arm Type
Experimental
Arm Description
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.
Arm Title
Cohort 3
Arm Type
Experimental
Arm Description
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.
Intervention Type
Biological
Intervention Name(s)
Coagulation Factor VIIa variant
Other Intervention Name(s)
MarzAA
Intervention Description
Single intravenous dose and ascending doses of subcutaneous injection of MarzAA, followed by a fixed dose of MarzAA for the treatment of bleeding episodes
Primary Outcome Measure Information:
Title
Comparative MarzAA activity by dose level/stage and confirm the Phase 2 dose
Description
Comparative pharmacokinetics by dose level/stage based on examination of AUX for each of the dose groups in each cohort.
Time Frame
Dosing period for each stage in a cohort will be approximately 5 to 11 days
Title
Bleeding episode treatment success
Description
Proportion of bleeding events treated with MarzAA achieving hemostatic efficacy based on a four-point scale according to the Investigator's assessment
Time Frame
24 hours after the first administration of study drug

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility 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
Facility Information:
Facility Name
UC Davis Medical Center
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
University of California -San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
University of Colorado Hemophilia and Thrombosis Center
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Rush University
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Children's Hospital of Michigan
City
Detroit
State/Province
Michigan
ZIP/Postal Code
48201
Country
United States
Facility Name
Michigan State University Center for Bleeding Disorders & Clotting Disorders
City
East Lansing
State/Province
Michigan
ZIP/Postal Code
49805
Country
United States
Facility Name
East Carolina University
City
Greenville
State/Province
North Carolina
ZIP/Postal Code
27858
Country
United States
Facility Name
Mazumdar Shaw Medical Centre
City
Bengaluru
Country
India
Facility Name
St. John's Medical College Hospital
City
Bengaluru
Country
India
Facility Name
Amrita Institute of Medical Sciences and Research Centre
City
Kochi
Country
India
Facility Name
K. J. Somaiya Hospital and Research Centre
City
Mumbai
Country
India
Facility Name
Sahyadri Super Speciality Hospital
City
Pune
Country
India
Facility Name
Careggi University Hospital
City
Florence
Country
Italy
Facility Name
Center for Thrombosis and Haemorrhagic Diseases
City
Milan
Country
Italy
Facility Name
Maggiore Polyclinic Hospital, IRCCS Ca' Granda
City
Milan
Country
Italy
Facility Name
Children's Hospital BambiNo Gesù, IRCCS (PEDS)
City
Roma
Country
Italy
Facility Name
City of Health and Science of Turin
City
Turin
Country
Italy
Facility Name
Territorial Clinical Hospital
City
Barnaul
Country
Russian Federation
Facility Name
National Medical Hematology Research Center under the Ministry of Healthcare of the Russian Federation
City
Moscow
Country
Russian Federation
Facility Name
Institute of Blood Pathology and Transfusion Medicine, Department of Surgery and Clinical Transfusiology
City
Lviv
Country
Ukraine

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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