Clinical Research Directory
Browse clinical research sites, groups, and studies.
Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors
Sponsor: Novo Nordisk A/S
Summary
This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B without inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use. Participants who usually only take medicine to treat bleeds (on-demand) will be placed in one of two groups. In one group participants will get study medicine from the start of the study. In the other group participants will continue with their normal medicine and get study medicine after 6 months. Which treatment the participant gets is decided by chance. Participants who usually take medicine to prevent bleeds (prophylaxis treatment) or who are already being treated with concizumab (study medicine) will receive the study medicine from the start of the study. Participants will have to inject themselves with the study medicine 1 time every day under the skin. This can be done at home. The study doctor will hand out the medicine in the form of a pen-injector. The pen-injector will contain the study medicine. The study will last for up to 8 years. The length of time the participant will be in the study depends on when they agreed to take part and when the medicine is available for purchase in their country (or 31 December 2027 at the latest). The time between visits will be approximately 4 weeks for the first 6 to 12 months depending on the group participants are in, and approximately 8 weeks for the rest of the study. If the participant attends extra visits due to the prescription medicine not being available for purchase in their country, these will be 14 weeks apart. Participants will be asked to record information in an electronic diary during the study and may also be asked to wear an activity tracker.
Official title: Efficacy and Safety of Concizumab Prophylaxis in Patients With Haemophilia A or B Without Inhibitors
Key Details
Gender
MALE
Age Range
12 Years - Any
Study Type
INTERVENTIONAL
Enrollment
156
Start Date
2019-11-13
Completion Date
2028-02-21
Last Updated
2026-03-24
Healthy Volunteers
No
Interventions
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.
Locations (112)
Children's Hospital Los Angeles - Endocrinology
Los Angeles, California, United States
Center for Inherited Blood Disorders
Orange, California, United States
Center for Blood Disorders Augusta University
Augusta, Georgia, United States
Indiana Hemophilia-Thromb Ctr
Indianapolis, Indiana, United States
University of Iowa_Iowa City
Iowa City, Iowa, United States
Children's Hospital of Michigan
Detroit, Michigan, United States
Michigan State University
Lansing, Michigan, United States
Novant Hlth Vasc Ins Charlotte
Charlotte, North Carolina, United States
M.S. Hershey Medical Center
Hershey, Pennsylvania, United States
Vanderbilt University Medical Center_Nashville_0
Nashville, Tennessee, United States
University of Texas San Antonio
San Antonio, Texas, United States
Versiti, CCBD
Milwaukee, Wisconsin, United States
Haematology and Blood Bank Department
Algiers, Algeria
CHU Constantine BEN BADIS/ Hematology department
Constantine, Algeria
The Alfred
Melbourne, Victoria, Australia
Royal Children's Hospital
Parkville, Victoria, Australia
The Royal Children's Hospital
Parkville, Victoria, Australia
Fiona Stanley Hospital - Haemophilia and Haemostasis Centre
Murdoch, Western Australia, Australia
University Clinical Center of Republic Srpska (205)
Banja Luka, Bosnia and Herzegovina
UMHAT Tsaritsa Yoanna - ISUL EAD, Pediatric clinical hematology and oncology
Sofia, Bulgaria
UMHAT Sveta Marina EAD, Clinic of Pediatric Clinical Hematology and Oncology
Varna, Bulgaria
Eastern Health Authority
St. John's, Newfoundland and Labrador, Canada
Hamltn Hth Sci/McMstr Child Hosp
Hamilton, Ontario, Canada
KBC Zagreb_Hematology
Zagreb, Croatia
Copenhagen Center for Heamatology
Copenhagen, Denmark
North Estonia Medical Centre Foundation
Tallinn, Estonia
Centre Hospitalier Regional Et Universitaire de Brest-Hopital de La Cavale Blanche
Brest, France
CHU de Caen - Côte de Nacre
Caen, France
Hopital de Bicetre
Le Kremlin-Bicêtre, France
Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu
Nantes, France
Hôpital Pontchaillou
Rennes, France
Centre Hospitalier Universitaire de Saint Etienne-Hopital Nord
Saint-Priest-en-Jarez, France
Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie
Bonn, Germany
Universität des Saarlandes - Hämostaseologie und Transfusionsmedizin
Homburg, Germany
MH Eü. Központ -Orszagos Haemophilia Kozpont
Budapest, Hungary
St. John's Medical college and Hospital
Bangalore, Karnataka, India
Sahyadri Speciality Hospital
Pune, Maharashtra, India
Sahyadri Super Speciality Hospital
Pune, Maharashtra, India
J K Lon Hospital
Jaipur, Rajasthan, India
CMCV
Ranipet, Tamil Nadu, India
Sheba MC The Israeli National Hemophilia Center
Tel Litwinsky, Israel
Dipartimento di Ematologia Univ. Firenze
Florence, FI, Italy
Istituto di Medicina Int. A. Bianchi Bonomi Univ. Milano
Milan, MI, Italy
Policlinico Umberto I Sezione Ematologia
Roma, Italy
A.O.U Città Salute Scienza Torino
Torino, Italy
Nagoya University Hospital_Blood Transfusion
Aichi, Japan
Hiroshima University Hospital, Hematology
Hiroshima, Japan
Hiroshima University Hospital_Hematology
Hiroshima, Japan
Hyogo prefectural kobe children's hospital Dept. of Haem and Onclogy
Hyōgo, Japan
Hyogo prefectural kobe children's hospital
Hyōgo, Japan
Itoigawa sogo Hospital_Department of Pediatrics
Niigata, Japan
Osaka National Hospital_Dept. of Infectious diseases
Osaka, Japan
Saitama Children's Med Centre_Hematology-Oncology
Saitama, Japan
Saitama Medical Univ. Hospital_Dep of Int Med, Cent for Hemo
Saitama, Japan
Saitama Medical Univ. Hospital
Saitama, Japan
Shizuoka Children's Hospital, Hematology-Oncology
Shizuoka, Japan
Shizuoka Children's Hospital
Shizuoka, Japan
National Center for Child Health and Development_Hematology
Tokyo, Japan
Ogikubo Hospital_Pediatries & Blood
Tokyo, Japan
Children Oncohaematology department Children's Hospital,
Vilnius, Lithuania
Vilnius University hospital Santaros klinikos
Vilnius, Lithuania
Hospital Ampang
Ampang, Selangor, Malaysia
Hospital Ampang
Ampang, Selangor, Malaysia
Hospital Universitario Dr. José Eleuterio González
Monterrey, Nuevo León, Mexico
Uniwersytecki Szpital Kliniczny W Poznaniu
Poznan, Greater Poland Voivodeship, Poland
Szpital Uniwersytecki, Oddzial Kliniczny Hematologii
Krakow, Lesser Poland Voivodeship, Poland
Instytut Hematologii i Transfuzjologii
Warsaw, Masovian Voivodeship, Poland
Uniwersytecki Szpital Kliniczny nr 1 Klinika Hematoonkologii i Transplantacji Szpiku
Lublin, Poland
Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa
Lublin, Poland
Uniwersytecki Szpital Kliniczny Im. Jana Mikulicza-Radeckiego We Wroclawiu
Wroclaw, Poland
ULS São João, E.P.E.
Porto, Portugal
Children Regional Clinical Hospital
Krasnodar, Russia
Morozovskaya municipal children hospital
Moscow, Russia
National Medical Research institution of haemotology
Moscow, Russia
Republican Hospital n.a. V. A. Baranov
Petrozavodsk, Russia
City out-patient clinic 37, City Hemophilia Centre
Saint Petersburg, Russia
Clinical Centre of Serbia, Institute for Haematology
Belgrade, Serbia
Institute for Mother and Child Health Care of Serbia
Belgrade, Serbia
University Clinical Centre Kragujevac
Kragujevac, Serbia
Clinical Centre of Vojvodina
Novi Sad, Serbia
Nemocnica sv. Cyrila a Metoda, UNB,Klinika hemat. a transfuz
Bratislava, Slovakia
Charlotte Maxeke Johannesburg Academic Hospital
Parktown, Johannesburg, Gauteng, South Africa
Pietersburg Hospital
Polokwane, Limpopo, South Africa
Daejeon Eulji Medical Center, Eulji University
Daejeon, South Korea
Daejeon Eulji University Hospital
Daejeon, South Korea
Jeju National University Hospital
Jeju City, South Korea
Jeju National University Hospital
Jeju-do, South Korea
Severance Hospital, Yonsei University Health System
Seoul, South Korea
Hospital Universitario La Paz
Madrid, Spain
Hospital Regional Universitario de Málaga
Málaga, Spain
Hospital Univ. Central de Asturias
Oviedo, Spain
Hospital Virgen del Rocío
Seville, Spain
Hospital Universitario y Politécnico La Fe
Valencia, Spain
Koagulationsmottagning - SUS
Malmo, Sweden
Koagulationsmottagningen
Solna, Sweden
Universitätsspital Zürich - Klinik für Medizinische Onkologie und Hämatologie
Zurich, Switzerland
Ramathibodi Hospital_Department of Haematology
Bangkok, Thailand
Gazi University
Ankara, Beşevler/Ankara, Turkey (Türkiye)
Gazi Üniversitesi Hastanesi- Hematoloji
Ankara, Beşevler/Ankara, Turkey (Türkiye)
İstanbul Üniversitesi İstanbul Tıp Fakültesi Hastanesi- Onkoloji Enstitüsü
Capa-ISTANBUL, Capa-ISTANBUL, Turkey (Türkiye)
Acıbadem Adana Hastanesi-Hematoloji
Adana, Turkey (Türkiye)
Hacettepe University Medical Faculty
Ankara, Turkey (Türkiye)
Hacettepe Üniversitesi Hastanesi- Endokrinoloji
Ankara, Turkey (Türkiye)
Trakya Üniversitesi Tıp Fakültesi Hastanesi-Hematoloji
Edirne, Turkey (Türkiye)
Ege Üniversitesi Hastanesi- Hematoloji
Izmir, Turkey (Türkiye)
Ondokuz Mayıs Üniversitesi Hastanesi - Hematoloji
Samsun, Turkey (Türkiye)
National specialized children's hospital 'OHMATDYT' - Haemostasis centre
Kyiv, Ukraine
Institute of blood pathology and transfusion medicine of NAMSU - General and haematol. surgery
Lviv, Ukraine
Belfast City Hospital
Belfast, United Kingdom
Royal Free Haemophilia Comprehensive Care Center
London, United Kingdom
Royal Free Hospital - Haemophilia
London, United Kingdom
Royal Hallamshire Hospital
Sheffield, United Kingdom