Phase 1, single-dose escalating study of marzeptacog alfa (activated), a recombinant factor VIIa variant, in patients with severe hemophilia.

Abstract

Essentials Marzeptacog alfa (activated) [MarzAA] is a novel variant of activated human factor VII. A phase 1 dose escalation trial of MarzAA was conducted in subjects with severe hemophilia. MarzAA was safe and tolerated at intravenous doses up to 30 μg kg-1 Data observed support further trials for hemophilia patients with inhibitors to factors VIII/IX.

Journal

Journal ID (iso-abbrev): J. Thromb. Haemost.

Title: Journal of thrombosis and haemostasis : JTH

Publisher: Wiley

ISSN (Electronic): 1538-7836

ISSN (Print): 1538-7836

Publication date (Electronic): Oct 2018

Volume: 16

Issue: 10

Affiliations

[1 ] Comprehensive Hemophilia and Thrombosis Center, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.

[2 ] Phoenix Pharma Pty Ltd, Mount Croix, Port Elizabeth, South Africa.

[3 ] Semmelweis University 1st Department of Medicine, Budapest, Hungary.

[4 ] National Hemophilia Center and Hemostasis Department, Medical Center of the Hungarian Defense Forces, Budapest, Hungary.

[5 ] University Department of Haematology, Manchester Royal Infirmary, Manchester, UK.

[6 ] Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, IL, USA.

[7 ] KD Haemophilia and Thrombosis Centre, Royal Free Hospital, London, UK.

[8 ] Department of Medicine, Hemophilia Center, Castelfranco Veneto Hospital, Castelfranco, Italy.

[9 ] University of California San Diego, San Diego, CA, USA.

[10 ] Applied BioMath, Concord, MA, USA.

[11 ] Early Clinical Development, Pfizer Worldwide R&D, Pfizer Inc., Cambridge, MA, USA.

[12 ] Rare Disease Research Unit, Pfizer Inc., Cambridge, MA, USA.

Article

DOI: 10.1111/jth.14247

PubMed ID: 30151972

SO-VID: e67fc039-2e3b-4765-b159-36a0785d84ae