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

NCT04489537 | Terminated Phase 3 DMC FDA Drug

• 1 other identifier: MAA-304
• Study Type: interventional
• Target: 18
• Locations: 16 countries
• Sites: 43

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).

Conditions

Hemophilia A With Inhibitor; Hemophilia B With Inhibitor

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (2)

MarzAA

EXPERIMENTAL

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

Standard of Care

ACTIVE COMPARATOR

Standard of care administered on-demand during bleeding episodes

Biological: MarzAA

Interventions

MarzAA BIOLOGICAL

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.

MarzAA; Standard of Care

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Catalyst Biosciences: lead

Study Sites (43)

Phoenix Children's Hospital

Phoenix, Arizona, 85016, United States

Location

Lombardi Comprehensive Cancer Center

Washington D.C., District of Columbia, 20007, United States

Location

Hematology Center after Prof. R. Yeolyan

Yerevan, Armenia

Location

JSC "K.Eristavi National Center of Experimental and Clinical Surgery"

Tbilisi, Georgia

Location

Magyar Honvédség Egészségügyi Központ, Országos Haemophilia Központ

Budapest, Hungary

Location

Nirmal Hospital

Gujrāt, India

Location

K.L.E.S Dr. Prabhakar Kore Hospital and Medical Research Centre

Kārnād, India

Location

Sanjay Gandhi Postgraduate Institute of Medical Sciences

Lucknow, India

Location

K.J Somaiya Hospital and Research Centre

Mumbai, India

Location

MCGM - Comprehensive Thalassemia Care, Pediatric Hematology-Oncology & BMT Centre

Mumbai, India

Location

Sahyadri Specialty Hospital

Mumbai, India

Location

Grant Medical Foundation, Ruby Hall Clinic

Pune, India

Location

All India Institute of Medical Sciences, Rishikesh

Rishikesh, India

Location

Azienda Ospedaliero-Universitaria Careggi

Florence, Italy

Location

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano

Milan, Italy

Location

Azienda Ospedaliera Universitaria Federico II

Napoli, Italy

Location

Citta della Salute e della Scienza di Torino - Ospedale le Molinette

Torino, Italy

Location

Hospital Ampang

Ampang, Malaysia

Location

Raja Perempuan Zainab II Hospital

Kota Bharu, Malaysia

Location

Queen Elizabeth Hospital Malaysia

Kota Kinabalu, Malaysia

Location

Hospital Tunku Azizah Kuala Lumpur

Kuala Lumpur, Malaysia

Location

Tuanku Ja'afar Hospital, Seremban

Sungai Petani, Malaysia

Location

Instituto de Investigaciones Aplicada a la Neurociencia A.C.

Durango, Mexico

Location

Instituto Nacional de Pediatria

Mexico City, Mexico

Location

Investigación e Innovación en Medicina Traslacional S.A.P.I. de C.V.

Mexico City, Mexico

Location

Korczowski Bartosz, Gabinet Lekarski

Rzeszów, Poland

Location

Kirov Research Institute of Hematology and Blood Transfusion

Kirov, Russia

Location

Medis, Llc

Nizhny Novgorod, Russia

Location

Haemophilia Comprehensive Care Centre

Johannesburg, South Africa

Location

Hospital Universitario Vall d'Hebron

Barcelona, Spain

Location

Hospital de Malaga

Málaga, Spain

Location

Changhua Christian Hospital (CCH)

Changhua, Taiwan

Location

Taichung Veterans General Hospital

Taichung, Taiwan

Location

Akdeniz University Medical Faculty Hospital

Antalya, Turkey (Türkiye)

Location

Istanbul University Faculty of Medicine

Istanbul, Turkey (Türkiye)

Location

Ege University Medical Faculty

Izmir, Turkey (Türkiye)

Location

Özel Acibadem Adana Hastanesi

Seyhan, Turkey (Türkiye)

Location

"Cherkasy Regional Oncological Dispensary of Cherkasy Regional Council", Regional Treatment and Diagnostic Center of Hematology

Cherkasy, Ukraine

Location

"Kyiv City Clinical Hospital #9" of Executive Body of Kyiv City Council (Kyiv City State Administration), City Scientific-Practical Centre of Diagnostics and Treatment

Kyiv, Ukraine

Location

"Institute of Blood Pathology and Transfusion Medicine of National Academy of Medical Sciences of Ukraine", Haematology Department

Lviv, Ukraine

Location

Queen Elizabeth Hospital Birmingham

Birmingham, United Kingdom

Location

Royal Liverpool University Hospital

Liverpool, United Kingdom

Location

University Hospital Southampton NHS Foundation Trust

Southampton, United Kingdom

Location

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 24, 2020
• First Posted: July 28, 2020
• Study Start: May 4, 2021
• Primary Completion: November 15, 2021
• Study Completion: December 1, 2021
• Last Updated: December 17, 2021

Record last verified: 2021-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (2); ME (3); IN (8); AR (1); IT (4); TU (4); HU (1); ES (2); GE (1); GB (3); ZA (1); TW (2); RU (2); PL (1); UA (3); MY (5)