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

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          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.

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          Author and article information

          Journal
          J. Thromb. Haemost.
          Journal of thrombosis and haemostasis : JTH
          Wiley
          1538-7836
          1538-7836
          Oct 2018
          : 16
          : 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
          10.1111/jth.14247
          30151972
          e67fc039-2e3b-4765-b159-36a0785d84ae

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