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

Primary Purpose

Hemophilia A With Inhibitor, Hemophilia B With Inhibitor

Status
Terminated
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
MarzAA
Sponsored by
Catalyst Biosciences
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A With Inhibitor

Eligibility Criteria

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

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

Sites / Locations

  • Phoenix Children's Hospital
  • Lombardi Comprehensive Cancer Center
  • Hematology Center after Prof. R. Yeolyan
  • JSC "K.Eristavi National Center of Experimental and Clinical Surgery"
  • Magyar Honvédség Egészségügyi Központ, Országos Haemophilia Központ
  • Nirmal Hospital
  • K.L.E.S Dr. Prabhakar Kore Hospital and Medical Research Centre
  • Sanjay Gandhi Postgraduate Institute of Medical Sciences
  • K.J Somaiya Hospital and Research Centre
  • MCGM - Comprehensive Thalassemia Care, Pediatric Hematology-Oncology & BMT Centre
  • Sahyadri Specialty Hospital
  • Grant Medical Foundation, Ruby Hall Clinic
  • All India Institute of Medical Sciences, Rishikesh
  • Azienda Ospedaliero-Universitaria Careggi
  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano
  • Azienda Ospedaliera Universitaria Federico II
  • Citta della Salute e della Scienza di Torino - Ospedale le Molinette
  • Hospital Ampang
  • Raja Perempuan Zainab II Hospital
  • Queen Elizabeth Hospital Malaysia
  • Hospital Tunku Azizah Kuala Lumpur
  • Tuanku Ja'afar Hospital, Seremban
  • Instituto Nacional de Pediatria
  • Instituto de Investigaciones Aplicada a la Neurociencia A.C.
  • Investigación e Innovación en Medicina Traslacional S.A.P.I. de C.V.
  • Korczowski Bartosz, Gabinet Lekarski
  • Kirov Research Institute of Hematology and Blood Transfusion
  • Medis, Llc
  • Haemophilia Comprehensive Care Centre
  • Hospital Universitario Vall d'Hebron
  • Hospital de Malaga
  • Changhua Christian Hospital (CCH)
  • Taichung Veterans General Hospital
  • Akdeniz University Medical Faculty Hospital
  • Istanbul University Faculty of Medicine
  • Ege University Medical Faculty
  • Özel Acibadem Adana Hastanesi
  • "Cherkasy Regional Oncological Dispensary of Cherkasy Regional Council", Regional Treatment and Diagnostic Center of Hematology
  • "Kyiv City Clinical Hospital #9" of Executive Body of Kyiv City Council (Kyiv City State Administration), City Scientific-Practical Centre of Diagnostics and Treatment
  • "Institute of Blood Pathology and Transfusion Medicine of National Academy of Medical Sciences of Ukraine", Haematology Department
  • Queen Elizabeth Hospital Birmingham
  • Royal Liverpool University Hospital
  • University Hospital Southampton NHS Foundation Trust

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

MarzAA

Standard of Care

Arm 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

Standard of care administered on-demand during bleeding episodes

Outcomes

Primary Outcome Measures

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 compared with standard of care.

Secondary Outcome Measures

Full Information

First Posted
July 24, 2020
Last Updated
December 2, 2021
Sponsor
Catalyst Biosciences
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1. Study Identification

Unique Protocol Identification Number
NCT04489537
Brief Title
Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Hemophilia A or B
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
Study Type
Interventional

2. Study Status

Record Verification Date
December 2021
Overall Recruitment Status
Terminated
Why Stopped
Sponsor decision (not a safety decision)
Study Start Date
May 4, 2021 (Actual)
Primary Completion Date
November 15, 2021 (Actual)
Study Completion Date
December 1, 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
Data Monitoring Committee
Yes

5. Study Description

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).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A With Inhibitor, Hemophilia B With Inhibitor

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Crossover Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
18 (Actual)

8. Arms, Groups, and Interventions

Arm Title
MarzAA
Arm Type
Experimental
Arm 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 Title
Standard of Care
Arm Type
Active Comparator
Arm Description
Standard of care administered on-demand during bleeding episodes
Intervention Type
Biological
Intervention Name(s)
MarzAA
Intervention 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.
Primary Outcome Measure Information:
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 compared with standard of care.
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 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
Facility Information:
Facility Name
Phoenix Children's Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016
Country
United States
Facility Name
Lombardi Comprehensive Cancer Center
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20007
Country
United States
Facility Name
Hematology Center after Prof. R. Yeolyan
City
Yerevan
Country
Armenia
Facility Name
JSC "K.Eristavi National Center of Experimental and Clinical Surgery"
City
Tbilisi
Country
Georgia
Facility Name
Magyar Honvédség Egészségügyi Központ, Országos Haemophilia Központ
City
Budapest
Country
Hungary
Facility Name
Nirmal Hospital
City
Gujrat
Country
India
Facility Name
K.L.E.S Dr. Prabhakar Kore Hospital and Medical Research Centre
City
Karnad
Country
India
Facility Name
Sanjay Gandhi Postgraduate Institute of Medical Sciences
City
Lucknow
Country
India
Facility Name
K.J Somaiya Hospital and Research Centre
City
Mumbai
Country
India
Facility Name
MCGM - Comprehensive Thalassemia Care, Pediatric Hematology-Oncology & BMT Centre
City
Mumbai
Country
India
Facility Name
Sahyadri Specialty Hospital
City
Mumbai
Country
India
Facility Name
Grant Medical Foundation, Ruby Hall Clinic
City
Pune
Country
India
Facility Name
All India Institute of Medical Sciences, Rishikesh
City
Rishīkesh
Country
India
Facility Name
Azienda Ospedaliero-Universitaria Careggi
City
Firenze
Country
Italy
Facility Name
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano
City
Milan
Country
Italy
Facility Name
Azienda Ospedaliera Universitaria Federico II
City
Napoli
Country
Italy
Facility Name
Citta della Salute e della Scienza di Torino - Ospedale le Molinette
City
Torino
Country
Italy
Facility Name
Hospital Ampang
City
Ampang
Country
Malaysia
Facility Name
Raja Perempuan Zainab II Hospital
City
Kota Bharu
Country
Malaysia
Facility Name
Queen Elizabeth Hospital Malaysia
City
Kota Kinabalu
Country
Malaysia
Facility Name
Hospital Tunku Azizah Kuala Lumpur
City
Kuala Lumpur
Country
Malaysia
Facility Name
Tuanku Ja'afar Hospital, Seremban
City
Sungai Petani
Country
Malaysia
Facility Name
Instituto Nacional de Pediatria
City
Ciudad de mexico
Country
Mexico
Facility Name
Instituto de Investigaciones Aplicada a la Neurociencia A.C.
City
Durango
Country
Mexico
Facility Name
Investigación e Innovación en Medicina Traslacional S.A.P.I. de C.V.
City
Mexico City
Country
Mexico
Facility Name
Korczowski Bartosz, Gabinet Lekarski
City
Rzeszów
Country
Poland
Facility Name
Kirov Research Institute of Hematology and Blood Transfusion
City
Kirov
Country
Russian Federation
Facility Name
Medis, Llc
City
Nizhny Novgorod
Country
Russian Federation
Facility Name
Haemophilia Comprehensive Care Centre
City
Johannesburg
Country
South Africa
Facility Name
Hospital Universitario Vall d'Hebron
City
Barcelona
Country
Spain
Facility Name
Hospital de Malaga
City
Málaga
Country
Spain
Facility Name
Changhua Christian Hospital (CCH)
City
Changhua City
Country
Taiwan
Facility Name
Taichung Veterans General Hospital
City
Taichung
Country
Taiwan
Facility Name
Akdeniz University Medical Faculty Hospital
City
Antalya
Country
Turkey
Facility Name
Istanbul University Faculty of Medicine
City
Istanbul
Country
Turkey
Facility Name
Ege University Medical Faculty
City
İzmir
Country
Turkey
Facility Name
Özel Acibadem Adana Hastanesi
City
Seyhan
Country
Turkey
Facility Name
"Cherkasy Regional Oncological Dispensary of Cherkasy Regional Council", Regional Treatment and Diagnostic Center of Hematology
City
Cherkasy
Country
Ukraine
Facility Name
"Kyiv City Clinical Hospital #9" of Executive Body of Kyiv City Council (Kyiv City State Administration), City Scientific-Practical Centre of Diagnostics and Treatment
City
Kyiv
Country
Ukraine
Facility Name
"Institute of Blood Pathology and Transfusion Medicine of National Academy of Medical Sciences of Ukraine", Haematology Department
City
Lviv
Country
Ukraine
Facility Name
Queen Elizabeth Hospital Birmingham
City
Birmingham
Country
United Kingdom
Facility Name
Royal Liverpool University Hospital
City
Liverpool
Country
United Kingdom
Facility Name
University Hospital Southampton NHS Foundation Trust
City
Southampton
Country
United Kingdom

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 Hemophilia A or B

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