156
Participants
Start Date
November 13, 2019
Primary Completion Date
July 12, 2022
Study Completion Date
February 21, 2028
Concizumab
When patients are randomised/allocated to concizumab prophylaxis, they will receive a loading dose of 1.0 mg/kg concizumab at visit 2a (week (Wk) 0) (arm 2, 3 and 4) or visit 9a (Wk 24) (arm 1) followed by an initial daily dose of 0.20 mg/kg concizumab from treatment day 2. Within an initial 5-8-week dose adjustment period on 0.20 mg/kg concizumab, the patients can be increased or decreased in dose to 0.25 mg/kg or 0.15 mg/kg concizumab. A potential dose adjustment will take place at visit 4a.1 (Wk 6) or 9a.3 (Wk 30) and will be based on the concizumab exposure level measured at the previous visit 4a (Wk 6) or 9a.2 (Wk 28). Patients who have concizumab exposure levels of 200-4000 ng/mL will stay at 0.20 mg/kg concizumab. Patients in arm 1 will continue on-demand treatment with their usual replacement therapy until visit 9a (week 24; end of main part). In the extension part, patients in arm 1 will receive daily concizumab subcutaneous injections.
"UMHAT Tsaritsa Yoanna-ISUL", Sofia
Copenhagen Center for Heamatology, Copenhagen
The Alfred, Melbourne
Royal Children's Hospital, Parkville
Fiona Stanley Hospital - Haemophilia and Haemostasis Centre, Murdoch
Universitätsspital Zürich - Klinik für Medizinische Onkologie und Hämatologie, Zurich
A.O.U Città Salute Scienza Torino, Torino
Ramathibodi Hospital_Bangkok_0, Bangkok
Institute for Mother and Child Health Care of Serbia, Belgrade
North Estonia Medical Centre Foundation, Tallinn
CHU de Caen - Côte de Nacre, Caen
Haematology and Blood Bank Department, Algiers
M.S. Hershey Medical Center, Hershey
Clinical Centre of Vojvodina, Novi Sad
Trakya University, Edirne
CHU Constantine BEN BADIS/ Hematology department, Constantine
Hospital Universitario La Paz, Madrid
Hospital Regional Universitario de Málaga, Málaga
Centre Hospitalier Regional Et Universitaire de Brest-Hopital de La Cavale Blanche, Brest
University Clinical Centre Kragujevac, Kragujevac
Istanbul University Oncology Institute, Capa-ISTANBUL
Hôpital Pontchaillou, Rennes
Ege Universitesi Tip Fakultesi, Izmir
Daejeon Eulji University Hospital, Daejeon
Vanderbilt University Medical Center_Nashville_0, Nashville
Hospital Virgen del Rocío, Seville
Children's Hospital of Michigan, Detroit
Sheba MC The Israeli National Hemophilia Center, Tel Litwinsky
Versiti, CCBD, Milwaukee
Ondokuz Mayis University Medical Faculty Ped. Haematology, Samsun
Jeju National University Hospital, Jeju-do
Hospital Universitario Dr. José Eleuterio González, Monterrey
UKS - Hämostaseologie und Transfusionsmedizin, Homburg
Hospital Ampang, Ampang, Selangor
University Clinical Center of Republic Srpska (205), Banja Luka
University of Texas San Antonio, San Antonio
Institute of blood pathology and transfusion medicine, Lviv
Children's Hospital Los Angeles - Endocrinology, Los Angeles
Center for Inherited Blood Disorders, Orange
Hopital de Bicetre, Le Kremlin-Bicêtre
Morozovskaya municipal children hospital, Moscow
National Medical Research institution of haemotology, Moscow
Republican Hospital n.a. V. A. Baranov, Petrozavodsk
City out-patient clinic 37, City Hemophilia Centre, Saint Petersburg
J K Lon Hospital, Jaipur
Children Regional Clinical Hospital, Krasnodar
Sahyadri Speciality Hospital, Pune
Sahyadri Super Speciality Hospital, Pune
St. John's Medical college and Hospital, Bangalore
Hamltn Hth Sci/McMstr Child Hosp, Hamilton
KBC Zagreb, Rebro, Hemofilija centar, Zagreb
MH Eü. Központ -Orszagos Haemophilia Kozpont, Budapest
Nagoya University Hospital_Blood Transfusion, Aichi
Hiroshima University Hospital, Hematology, Hiroshima
Hyogo prefectural kobe children's hospital, Hyōgo
Osaka National Hospital, Osaka
Saitama Children's Med Centre_Hematology-Oncology, Saitama
Saitama Medical Univ. Hospital, Saitama
Shizuoka Children's Hospital, Shizuoka
National Center for Child Health and Development_Hematology, Tokyo
National Center for Child Health and Development, Tokyo
Ogikubo Hospital_Pediatries & Blood, Tokyo
Children Oncohaematology department Children's Hospital,, Vilnius
Vilnius University hospital Santaros klinikos, Vilnius
Uniwersytecki Szpital Kliniczny W Poznaniu, Poznan
Szpital Uniwersytecki, Oddzial Kliniczny Hematologii, Krakow
Instytut Hematologii i Transfuzjologii, Warsaw
Uniwersytecki Szpital Kliniczny nr 1 Klinika Hematoonkologii i Transplantacji Szpiku, Lublin
ULS São João, E.P.E., Porto
Pietersburg Hospital, Polokwane
Severance Hospital, Yonsei University Health System, Seoul
Koagulationsmottagning, Malmo
Koagulationsmottagningen, Solna
Gazi University, Ankara
Acibadem Adana Hastanesi, Adana
Hacettepe University Medical Faculty, Ankara
"National specialized children clinic OHMATDYT", Kyiv
Belfast City Hospital, Belfast
Royal Free Haemophilia Comprehensive Care Center, London
Royal Free Haemophilia Comprehensive Care Centre, London
Royal Hallamshire Hospital, Sheffield
Lead Sponsor
Novo Nordisk A/S
INDUSTRY