152
Participants
Start Date
September 27, 2016
Primary Completion Date
September 15, 2017
Study Completion Date
May 12, 2022
Emicizumab
Participants received emicizumab prophylaxis subcutaneously at the specified dose for each arm. After at least 24 weeks on prophylactic emicizumab, individuals who experienced suboptimal bleeding control on emicizumab (according to protocol-defined criteria) had the opportunity to increase their dose to 3 mg/kg weekly. Upon implementation of protocol version 4 (20-Dec-2019), treatment duration was extended. During this study prolongation, each participant had the option to choose a preferred emicizumab dosing regimen among those permitted (i.e., emicizumab 1.5 mg/kg once every week \[QW\], 3 mg/kg once every 2 weeks \[Q2W\], or 6 mg/kg once every 4 weeks \[Q4W\]) and continue on that dosing regimen until discontinuation from the study.
Factor VIII (FVIII)
FVIII was allowed to treat bleeds on an episodic basis, per the local prescribing information. Specific dosages of FVIII were not mandated in the study. Breakthrough bleeds were to be treated with the lowest FVIII dose expected to achieve hemostasis, which may have been lower than the participant's prior FVIII dose. To avoid bleeds before adequate emicizumab level is reached, patients in Arm D continued their regular FVIII prophylaxis until the second emicizumab loading dose. Concomitant routine FVIII prophylaxis was not permissible otherwise during the study.
St James's Hospital, Dublin
Taipei Medical University Hospital, Taipei
Changhua Christian Hospital, Chang-hua
ICIC, San José
Charlotte Maxeke Johannesburg Hospital; Haemophilia Comprehensive Care Center, Johannesburg
The Alfred Hospital, Melbourne; Thrombosis and Haemostasis Unit, Melbourne
Royal Adelaide Hospital, Adelaide
The Perth Blood Institute, Nedlands
Cornell Univ Medical College; Hematology-Oncolog, New York
Georgetown Uni Medical Center; Lombardi Cancer Center, Washington D.C.
"IRCCS Ca' Granda Ospedale Maggiore Policlinico; Centro Emofilia e Trombosi Angelo Bianchi e Bonomi", Milan
Hospital Universitario la Paz; Servicio de Hematologia, Madrid
Winship Cancer Institute, Atlanta
University of Florida, Gainesville
Taichung Veterans General Hospital, Taichung
Hospital Universitario Virgen del Rocio; Servicio de Hematologia, Seville
Children's Hospital of Michigan; Pediatrics, Detroit
AOU Careggi; SOD Malattie Emorragiche, Florence
Universitätsklinikum Bonn; Institut für Experimentelle Hämatologie und Transfusionsmedizin, Bonn
Hopital Cardio-vasculaire Louis Pradel; Hemostase clinique, Bron
Groupe Hospitalier Necker Enfants Malades, Paris
Santa Monica Oncology Center, Santa Monica
CH de Bicetre; Centre de Traitement d' Hemophilie, Le Kremlin-Bicêtre
Bloodworks Northwest (formerly Puget Sound Blood Center); Hemophilia, Seattle
Dana-Farber Cancer Institute; Brigham and Women's Cancer Center, Boston
Nagoya University Hospital, Aichi
St. Marianna University Hospital, Kanagawa
Sendai Medical Center, Miyagi
Nara Medical University Hospital, Nara
NHO Osaka National Hospital, Osaka
Tokyo Medical University Hospital, Tokyo
Ogikubo Hospital, Tokyo
Uniwersyteckie Centrum Kliniczne; Klinika Hematologii i Transplantologii, Gdansk
SPSK Nr1 Klinika Hematoo&Transpl.Szpiku, Lublin
ALVAMED Lekarskie Gabinety Specjalistyczne, Poznan
Instytut Hematologii i Transfuzjologii; Klinika Zaburzeń Hemostazy i Chorób Wewnętrznych, Warsaw
Severance Hospital, Seoul
Cardiff and Vale NHS Trust, Cardiff
Royal Free Hospital; Haemophilia Centre, London
Collaborators (1)
Chugai Pharmaceutical
INDUSTRY
Hoffmann-La Roche
INDUSTRY