134
Participants
Start Date
October 21, 2019
Primary Completion Date
December 27, 2021
Study Completion Date
February 21, 2027
Concizumab
Concizumab will be administered daily subcutaneously (s.c., under the skin). When patients are randomised to concizumab prophylaxis they will receive a loading dose of 1.0 mg/kg concizumab at visit 2a (week 0: arm 2, 3 \& 4) or visit 9a (week 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 (week 6: arm 2, 3 \& 4) or 9a.3 (week 30: arm 1) and will be based on the concizumab exposure level measured at the previous visit 4a (week 4) or 9a.2 (week 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 bypassing product until visit 9a (week 24: end of main part for arm 1).
Klin. Abt. f. Hämatologie und Hämostaseologie, AKH Wien, Vienna
UMHAT Tsaritsa Yoanna-ISUL EAD, Sofia
Charlotte Maxeke Johannesburg Academic Hospital, Parktown, Johannesburg
The Alfred, Melbourne
Haematology Clinic, Durban
Fiona Stanley Hospital - Haemophilia and Haemostasis Centre, Murdoch
Skejby Blodsygdomme, blødercentret, Aarhus N
KBC Zagreb, Zavod za pedijatrijsku hematologiju, Zagreb
RAMA -Hemato-Med_Department of Haematology, Bangkok
Hospital Pulau Pinang_Georgetown, Penang, George Town
Ustav Hematologie a krevni tranfuze, Prague
Haematology and Blood Bank Department, Algiers
Istituto di Medicina Int. A. Bianchi Bonomi Univ. Milano, Milan
CHU Constantine BEN BADIS/ Hematology department, Constantine
Hospital Universitario La Paz, Madrid
St. Jude Affiliate Clinic at Novant Health Hemby Children's, Charlotte
Hospital Regional Universitario de Málaga, Málaga
Hospital Universitario Regional de Málaga, Málaga
Children's Healthcare Atlanta, Atlanta
Istituto Oncologico Veneto - Oncoematologia IOV, Castelfranco Veneto
Oncoematologia IOV, Castelfranco Veneto
Hospital Central de Asturias, Oviedo
Hospital Univ. Central de Asturias, Oviedo
"Azienda OU S.Maria della Misericordia", Udine
University Clinical Centre Kragujevac, Kragujevac
Sunpasitthiprasong Hospital, Ubon Ratchathani
Istanbul University Oncology Institute, Capa-ISTANBUL
Centre Hospitalier Universitaire de Rennes - Hopital Pontchaillou, Rennes
Daejeon Eulji Medical Center, Eulji University, Daejeon
Azienda Ospedaliera Universitaria Integrata Verona - Ospedale Donna Bambino Borgo Trento - U.O.C. Oncoematologia Pediatrica, Verona
Ospedale Donna Bambino U.O.C. Oncoematologia Pediatrica, Verona
TriStar Medical Group Children's Specialist, Nashville
Hospital Virgen del Rocío, Seville
Indiana Hemophilia-Thromb Ctr, Indianapolis
Dipartimento di Ematologia Univ. Firenze, Florence
Maharaj Nakorn Chiang Mai Hospital -Pediatric Hematology and Oncology, Chiang Mai
Centre Hospitalier de Clermont-Ferrand-Hopital Estaing, Clermont-Ferrand
Washington University School of Medicine_St. Louis, St Louis
Hospital Universitario Dr. José Eleuterio González, Monterrey
Hospital Ampang, Ampang
Hospital Ampang, Ampang, Selangor
Hospices Civils de Lyon- Hopital Louis Pradel-1, Bron
University of Texas San Antonio, San Antonio
Institute of blood pathology and transfusion medicine of NAMSU - General and haematol. surgery, Lviv
Hospital Queen Elizabeth 1, Kota Kinabalu
Center for Inherited Blood Dis, Orange
Ap-Hp-Hopital de Bicetre-1, Le Kremlin-Bicêtre
All India Institute of Medical Sciences_New Dehli, New Dehli
All India Institute of Medical Sciences_New Dehli, New Delhi
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
Children Regional Clinical Hospital, Krasnodar
Sahyadri Speciality Hospital, Pune
Sahyadri Super Speciality Hospital, Pune
St. John's Medical college and Hospital, Bangalore
CMCV, Ranipet
KBC Zagreb, Rebro, Hemofilija centar, Zagreb
Fakultni nemocnice v Motole, Prague
CMCV, Ranipet
Nagoya University Hospital_Blood Transfusion, Aichi
Kagoshima City Hospital, Kagoshima
St. Marianna University School of Medicine Hospital_Pediatrics, Kanagawa
Nara Medical University Hospital_Pediatrics, Nara
Saitama Medical Univ. Hospital, Saitama
Ogikubo Hospital_Pediatries & Blood, Tokyo
Rikshospitalet - avdeling for blodsykdommer, Oslo
Szpital Uniwersytecki, Oddzial Kliniczny Hematologii, Krakow
Instytut Hematologii i Transfuzjologii, Warsaw
SPSK nr 1 Klinika Hematoonkologii i Transplantacji Szpiku, Lublin
Uniwersytecki Szpital Kliniczny nr 1 Klinika Hematoonkologii i Transplantacji Szpiku, Lublin
Uniwersytecki Szpital Kliniczny Im. Jana Mikulicza-Radeckiego We Wroclawiu, Wroclaw
ULS São João, E.P.E., Porto
Nemocnica sv. Cyrila a Metoda, UNB,Klinika hemat. a transfuz, Bratislava
Pietersburg Hospital, Polokwane
Severance Hospital, Yonsei University Health System, Seoul
Hospital Vall d'Hebron, Barcelona
Koagulationsmottagningen, Solna
Akdeniz Universitesi, Antalya
Acibadem Adana Hastanesi, Adana
Cukurova Universitesi, Adana
National specialized children's hospital 'OHMATDYT' - Haemostasis centre, Kyiv
West Midlands Adult Comprehensive Care Haemophilia, Birmingham
Great Ormond Street Hospital for Children, London
Royal Hallamshire Hospital, Sheffield
Lead Sponsor
Novo Nordisk A/S
INDUSTRY