Efficacy and Safety of Concizumab Prophylaxis in Patients With Haemophilia A or B Without Inhibitors

Who is this study for? Child to adult male patients with Hemophilia
What treatments are being studied? Concizumab
Status: Active_not_recruiting
Location: See all (108) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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.

Eligibility
Participation Requirements
Sex: Male
Minimum Age: 12
Healthy Volunteers: f
View:

• Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial.

• Male aged 12 years or older at the time of signing informed consent.

• Congenital severe haemophilia A (FVIII below 1%) or B (FIX equal to or below 2%).

Locations
United States
California
Children's Hospital Los Angeles - Endocrinology
Los Angeles
Center for Inherited Blood Disorders
Orange
Georgia
Center for Blood Disorders Augusta University
Augusta
Iowa
University of Iowa_Iowa City
Iowa City
Indiana
Indiana Hemophilia-Thromb Ctr
Indianapolis
Michigan
Children's Hospital of Michigan
Detroit
Michigan State University
Lansing
North Carolina
Novant Hlth Vasc Ins Charlotte
Charlotte
Pennsylvania
M.S. Hershey Medical Center
Hershey
Tennessee
Vanderbilt University Medical Center_Nashville_0
Nashville
Texas
University of Texas San Antonio
San Antonio
Wisconsin
Versiti, CCBD
Milwaukee
Other Locations
Algeria
Haematology and Blood Bank Department
Algiers
CHU Constantine BEN BADIS/ Hematology department
Constantine
Australia
The Alfred
Melbourne
Fiona Stanley Hospital - Haemophilia and Haemostasis Centre
Murdoch
Royal Children's Hospital
Parkville
Bosnia and Herzegovina
University Clinical Center of Republic Srpska (205)
Banja Luka
Bulgaria
UMHAT Tsaritsa Yoanna-ISUL EAD
Sofia
UMHAT Sveta Marina EAD Clinic of Pediatric Clinical Hemat
Varna
Canada
Hamltn Hth Sci/McMstr Child Hosp
Hamilton
Eastern Health Authority
St. John's
Croatia
KBC Zagreb, Rebro, Hemofilija centar
Zagreb
Denmark
Copenhagen Center for Heamatology
Copenhagen
Estonia
North Estonia Medical Centre Foundation
Tallinn
France
Centre Hospitalier Regional Et Universitaire de Brest-Hopital de La Cavale Blanche
Brest
CHU de Caen - Côte de Nacre
Caen
Hopital de Bicetre
Le Kremlin-bicêtre
Centre Hospitalier Universitaire de Nantes-Hopital Hotel-Dieu
Nantes
Hôpital Pontchaillou
Rennes
Centre Hospitalier Universitaire de Saint Etienne-Hopital Nord
Saint-priest-en-jarez
Germany
Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie
Bonn
Universitätsklinikum des Saarlandes - Hämostaseologie und Transfusionsmedizin
Homburg
Hungary
MH Eü. Központ -Orszagos Haemophilia Kozpont
Budapest
India
St. John's Medical college and Hospital
Bangalore
J K Lon Hospital
Jaipur
Sahyadri Speciality Hospital
Pune
Sahyadri Super Speciality Hospital
Pune
CMCV
Ranipet
Israel
Sheba MC The Israeli National Hemophilia Center
Tel Litwinsky
Italy
Dipartimento di Ematologia Univ. Firenze
Florence
Istituto di Medicina Int. A. Bianchi Bonomi Univ. Milano
Milan
Policlinico Umberto I Sezione Ematologia
Roma
A.O.U Città Salute Scienza Torino
Torino
Japan
Nagoya University Hospital_Blood Transfusion
Aichi
Hiroshima University Hospital, Hematology
Hiroshima
Hyogo prefectural kobe children's hospital
Hyōgo
Itoigawa sogo Hospital_Department of Pediatrics
Niigata
Osaka National Hospital
Osaka
Saitama Children's Med Centre_Hematology-Oncology
Saitama
Saitama Medical Univ. Hospital
Saitama
Shizuoka Children's Hospital
Shizuoka
Shizuoka Children's Hospital, Hematology-Oncology
Shizuoka
National Center for Child Health and Development_Hematology
Tokyo
Ogikubo Hospital_Pediatries & Blood
Tokyo
Lithuania
Children Oncohaematology department Children's Hospital,
Vilnius
Vilnius University hospital Santaros klinikos
Vilnius
Malaysia
Hospital Ampang
Ampang
Hospital Ampang
Ampang, Selangor
Mexico
Hospital Universitario Dr. José Eleuterio González
Monterrey
Poland
Szpital Uniwersytecki, Oddzial Kliniczny Hematologii
Krakow
Uniwersytecki Szpital Dzieciecy, Dzial Krwiolecznictwa
Lublin
Uniwersytecki Szpital Kliniczny nr 1 Klinika Hematoonkologii i Transplantacji Szpiku
Lublin
Uniwersytecki Szpital Kliniczny W Poznaniu
Poznan
Instytut Hematologii i Transfuzjologii
Warsaw
Uniwersytecki Szpital Kliniczny Im. Jana Mikulicza-Radeckiego We Wroclawiu
Wroclaw
Portugal
ULS São João, E.P.E.
Porto
Republic of Korea
Daejeon Eulji Medical Center, Eulji University
Daejeon
Daejeon Eulji University Hospital
Daejeon
Jeju National University Hospital
Jeju City
Jeju National University Hospital
Jeju-do
Severance Hospital, Yonsei University Health System
Seoul
Russian Federation
Children Regional Clinical Hospital
Krasnodar
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
Serbia
Clinical Centre of Serbia, Institute for Haematology
Belgrade
Institute for Mother and Child Health Care of Serbia
Belgrade
University Clinical Centre Kragujevac
Kragujevac
Clinical Centre of Vojvodina
Novi Sad
Slovakia
Nemocnica sv. Cyrila a Metoda, UNB,Klinika hemat. a transfuz
Bratislava
South Africa
Charlotte Maxeke Johannesburg Academic Hospital
Parktown, Johannesburg
Pietersburg Hospital
Polokwane
Spain
Hospital Universitario La Paz
Madrid
Hospital Regional Universitario de Málaga
Málaga
Hospital Univ. Central de Asturias
Oviedo
Hospital Virgen del Rocío
Seville
Hospital Universitario y Politécnico La Fe
Valencia
Sweden
Koagulationsmottagning
Malmo
Koagulationsmottagningen
Solna
Switzerland
Universitätsspital Zürich - Klinik für Medizinische Onkologie und Hämatologie
Zurich
Thailand
RAMA -Hemato-Med_Department of Haematology
Bangkok
Turkey
Acıbadem Adana Hastanesi-Hematoloji
Adana
Gazi Üniversitesi Hastanesi- Hematoloji
Ankara
Gazi University
Ankara
Hacettepe Üniversitesi Hastanesi- Endokrinoloji
Ankara
Hacettepe University Medical Faculty
Ankara
İstanbul Üniversitesi İstanbul Tıp Fakültesi Hastanesi- Onkoloji Enstitüsü
Capa-istanbul
Trakya Üniversitesi Tıp Fakültesi Hastanesi-Hematoloji
Edirne
Ege Üniversitesi Hastanesi- Hematoloji
Izmir
Ondokuz Mayis University Medical Faculty Ped. Haematology
Samsun
Ukraine
National specialized children's hospital 'OHMATDYT' - Haemostasis centre
Kyiv
Institute of blood pathology and transfusion medicine of NAMSU - General and haematol. surgery
Lviv
United Kingdom
Belfast City Hospital
Belfast
Royal Free Haemophilia Comprehensive Care Center
London
Royal Free Haemophilia Comprehensive Care Centre
London
Royal Hallamshire Hospital
Sheffield
Time Frame
Start Date: 2019-11-13
Completion Date: 2028-02-21
Participants
Target number of participants: 156
Treatments
Experimental: Arm 1: No prophylaxis (PPX)
Haemophilia A (HA) and haemophilia B (HB) patients, previously treated on-demand, will be randomised 1:2 to no prophylaxis versus concizumab prophylaxis. In the extension phase, this group will receive treatment with concizumab.
Experimental: Arm 2: Concizumab prophylaxis
HA and HB patients, previously treated on-demand, will be randomised 1:2 to no prophylaxis versus concizumab prophylaxis.
Experimental: Arm 3: Concizumab prophylaxis
The HA patients enrolled into the concizumab phase 2 trial NN7415-4255 (explorer 5) will be offered enrolment into this arm.
Experimental: Arm 4: Concizumab prophylaxis
Arm 4 will include patients previously on prophylaxis with factor products with a minimum of 24 weeks observation in NN7415-4322 (explorer 6) (at least 30 HA and 30 HB patients).~In addition, arm 4 will also include: 1) Patients who were randomised to arms 1 and 2 before the treatment pause. 2) HA patients who were in NN7415-4255 (explorer 5) at the time of the treatment pause, and who have now completed explorer 5. 3) On demand patients included after arms 1 and 2 are closed.
Related Therapeutic Areas
Sponsors
Leads: Novo Nordisk A/S

This content was sourced from clinicaltrials.gov