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

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

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).

Study Overview

• Status: Terminated
• Conditions: Hemophilia A With Inhibitor; Hemophilia B With Inhibitor
• Intervention / Treatment: Biological: MarzAA

• Study Type: Interventional
• Enrollment (Actual): 18
• Phase: Phase 3

Contacts and Locations

Study Locations

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

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	24 hours after the first administration of study drug

Collaborators and Investigators

• Sponsor: Catalyst Biosciences

Study record dates

Study Major Dates

• Study Start (Actual): May 4, 2021
• Primary Completion (Actual): November 15, 2021
• Study Completion (Actual): December 1, 2021

Study Registration Dates

• First Submitted: July 24, 2020
• First Submitted That Met QC Criteria: July 24, 2020
• First Posted (Actual): July 28, 2020

Study Record Updates

• Last Update Posted (Actual): December 17, 2021
• Last Update Submitted That Met QC Criteria: December 2, 2021
• Last Verified: December 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Blood Coagulation Disorders; Hemophilia A; Hemophilia B
• Other Study ID Numbers: MAA-304

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No