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

NCT ID: NCT04489537

Last Updated: 2021-12-17

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE3

Total Enrollment

18 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-05-04

Study Completion Date

2021-12-01

Brief Summary

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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).

Detailed Description

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Conditions

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Hemophilia A With Inhibitor Hemophilia B With Inhibitor

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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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

Group Type EXPERIMENTAL

MarzAA

Intervention Type 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.

Standard of Care

Standard of care administered on-demand during bleeding episodes

Group Type ACTIVE_COMPARATOR

MarzAA

Intervention Type 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.

Interventions

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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.

Intervention Type BIOLOGICAL

Eligibility Criteria

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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
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Catalyst Biosciences

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Phoenix Children's Hospital

Phoenix, Arizona, United States

Site Status

Lombardi Comprehensive Cancer Center

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

Site Status

Hematology Center after Prof. R. Yeolyan

Yerevan, , Armenia

Site Status

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

Tbilisi, , Georgia

Site Status

Magyar Honvédség Egészségügyi Központ, Országos Haemophilia Központ

Budapest, , Hungary

Site Status

Nirmal Hospital

Gujrāt, , India

Site Status

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

Kārnād, , India

Site Status

Sanjay Gandhi Postgraduate Institute of Medical Sciences

Lucknow, , India

Site Status

K.J Somaiya Hospital and Research Centre

Mumbai, , India

Site Status

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

Mumbai, , India

Site Status

Sahyadri Specialty Hospital

Mumbai, , India

Site Status

Grant Medical Foundation, Ruby Hall Clinic

Pune, , India

Site Status

All India Institute of Medical Sciences, Rishikesh

Rishikesh, , India

Site Status

Azienda Ospedaliero-Universitaria Careggi

Florence, , Italy

Site Status

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico di Milano

Milan, , Italy

Site Status

Azienda Ospedaliera Universitaria Federico II

Napoli, , Italy

Site Status

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

Torino, , Italy

Site Status

Hospital Ampang

Ampang, , Malaysia

Site Status

Raja Perempuan Zainab II Hospital

Kota Bharu, , Malaysia

Site Status

Queen Elizabeth Hospital Malaysia

Kota Kinabalu, , Malaysia

Site Status

Hospital Tunku Azizah Kuala Lumpur

Kuala Lumpur, , Malaysia

Site Status

Tuanku Ja'afar Hospital, Seremban

Sungai Petani, , Malaysia

Site Status

Instituto de Investigaciones Aplicada a la Neurociencia A.C.

Durango, , Mexico

Site Status

Instituto Nacional de Pediatria

Mexico City, , Mexico

Site Status

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

Mexico City, , Mexico

Site Status

Korczowski Bartosz, Gabinet Lekarski

Rzeszów, , Poland

Site Status

Kirov Research Institute of Hematology and Blood Transfusion

Kirov, , Russia

Site Status

Medis, Llc

Nizhny Novgorod, , Russia

Site Status

Haemophilia Comprehensive Care Centre

Johannesburg, , South Africa

Site Status

Hospital Universitario Vall d'Hebron

Barcelona, , Spain

Site Status

Hospital de Malaga

Málaga, , Spain

Site Status

Changhua Christian Hospital (CCH)

Changhua, , Taiwan

Site Status

Taichung Veterans General Hospital

Taichung, , Taiwan

Site Status

Akdeniz University Medical Faculty Hospital

Antalya, , Turkey (Türkiye)

Site Status

Istanbul University Faculty of Medicine

Istanbul, , Turkey (Türkiye)

Site Status

Ege University Medical Faculty

Izmir, , Turkey (Türkiye)

Site Status

Özel Acibadem Adana Hastanesi

Seyhan, , Turkey (Türkiye)

Site Status

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

Cherkasy, , Ukraine

Site Status

"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

Site Status

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

Lviv, , Ukraine

Site Status

Queen Elizabeth Hospital Birmingham

Birmingham, , United Kingdom

Site Status

Royal Liverpool University Hospital

Liverpool, , United Kingdom

Site Status

University Hospital Southampton NHS Foundation Trust

Southampton, , United Kingdom

Site Status

Countries

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United States Armenia Georgia Hungary India Italy Malaysia Mexico Poland Russia South Africa Spain Taiwan Turkey (Türkiye) Ukraine United Kingdom

Other Identifiers

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MAA-304

Identifier Type: -

Identifier Source: org_study_id