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Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia With 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 with 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 get 1 injection with the study medicine every day under the skin. This participants will have to do themselves and 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 about seven years. The length of time the participants will be in the study depends on when they agreed to take part or when the medicine is available for purchase in their country (31 December 2026 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. Participants will be asked to record information into 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 With Inhibitors
Key Details
Gender
MALE
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
134
Start Date
2019-10-21
Completion Date
2027-02-21
Last Updated
2026-03-24
Healthy Volunteers
No
Interventions
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).
Locations (99)
Center for Inherited Blood Dis
Orange, California, United States
Children's Healthcare Atlanta
Atlanta, Georgia, United States
Indiana Hemophilia-Thromb Ctr
Indianapolis, Indiana, United States
Washington University School of Medicine_St. Louis
St Louis, Missouri, United States
St. Jude Affiliate Clinic at Novant Health Hemby Children's
Charlotte, North Carolina, United States
TriStar Medical Group Children's Specialist
Nashville, Tennessee, United States
University of Texas San Antonio
San Antonio, Texas, United States
Haematology and Blood Bank Department
Algiers, Algeria
CHU Constantine BEN BADIS/ Hematology department
Constantine, Algeria
The Alfred
Melbourne, Victoria, Australia
The Royal Children's Hospital
Parkville, Victoria, Australia
Fiona Stanley Hospital - Haemophilia and Haemostasis Centre
Murdoch, Western Australia, Australia
Klin. Abt. f. Hämatologie und Hämostaseologie, AKH Wien
Vienna, Austria
UMHAT Tsaritsa Yoanna - ISUL EAD, Pediatric clinical hematology and oncology
Sofia, Bulgaria
Hamltn Hth Sci/McMstr Child Hosp
Hamilton, Ontario, Canada
KBC Zagreb, Rebro, Hemofilija centar
Zagreb, Croatia
KBC Zagreb_Hematology
Zagreb, Croatia
KBC Zagreb, Zavod za pedijatrijsku hematologiju
Zagreb, Croatia
Ustav Hematologie a krevni tranfuze
Prague, Czechia
Fakultni Nemocnice Motol A Homolka
Prague, Czechia
Skejby Blodsygdomme, blødercentret
Aarhus N, Denmark
Hospices Civils de Lyon- Hopital Louis Pradel
Bron, France
Centre Hospitalier de Clermont-Ferrand-Hopital Estaing
Clermont-Ferrand, France
Ap-Hp-Hopital de Bicetre
Le Kremlin-Bicêtre, France
Centre Hospitalier Universitaire de Rennes - Hopital Pontchaillou
Rennes, France
St. John's Medical college and Hospital
Bangalore, Karnataka, India
Sahyadri Speciality Hospital
Pune, Maharashtra, India
Sahyadri Super Speciality Hospital
Pune, Maharashtra, India
All India Institute of Medical Sciences_New Dehli
New Dehli, New Delhi, India
CMCV
Ranipet, Tamil Nadu, India
CMCV
Ranipet, Tamil Nadu, India
All India Institute of Medical Sciences_New Dehli
New Delhi, India
Dipartimento di Ematologia Univ. Firenze
Florence, FI, Italy
Istituto Oncologico Veneto - Oncoematologia IOV
Castelfranco Veneto, Italy
Oncoematologia IOV
Castelfranco Veneto, Italy
Istituto di Medicina Int. A. Bianchi Bonomi Univ. Milano
Milan, Italy
Azienda OU "S.Maria della Misericordia"
Udine, Italy
Azienda Ospedaliera Universitaria Integrata Verona - Ospedale Donna Bambino Borgo Trento - U.O.C. Oncoematologia Pediatrica
Verona, Italy
Ospedale Donna Bambino U.O.C. Oncoematologia Pediatrica
Verona, Italy
Nagoya University Hospital_Blood Transfusion
Aichi, Japan
Kagoshima City Hospital_Pediatrics
Kagoshima, Japan
Kagoshima City Hospital
Kagoshima, Japan
St. Marianna University School of Medicine Hospital_Pediatrics
Kanagawa, Japan
Mie University Hospital_Dept.of Blood Trans.Med & Cell
Mie, Japan
Nara Medical University Hospital_Pediatrics
Nara, Japan
Saitama Medical Univ. Hospital_Dep of Int Med, Cent for Hemo
Saitama, Japan
Ogikubo Hospital_Pediatries & Blood
Tokyo, Japan
Hospital Pulau Pinang_Georgetown, Penang
George Town, Pulau Pinang, Malaysia
Hospital Queen Elizabeth 1
Kota Kinabalu, Sabah, Malaysia
Hospital Ampang
Ampang, Selangor, Malaysia
Hospital Ampang
Ampang, Selangor, Malaysia
Hospital Universitario Dr. José Eleuterio González
Monterrey, Nuevo León, Mexico
Rikshospitalet - avdeling for blodsykdommer
Oslo, Norway
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
SPSK nr 1 Klinika Hematoonkologii i Transplantacji Szpiku
Lublin, Poland
Uniwersytecki Szpital Kliniczny nr 1 Klinika Hematoonkologii i Transplantacji Szpiku
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
Institute for Health Care of Children and Adolescents
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
Haematology Clinic
Durban, KwaZulu-Natal, South Africa
Pietersburg Hospital
Polokwane, Limpopo, South Africa
Daejeon Eulji Medical Center, Eulji University
Daejeon, South Korea
Severance Hospital, Yonsei University Health System
Seoul, South Korea
Hospital Vall d'Hebron
Barcelona, Spain
Hospital Universitario La Paz
Madrid, Spain
Hospital Regional Universitario de Málaga
Málaga, Spain
Hospital Universitario Regional de Málaga
Málaga, Spain
Hospital Central de Asturias
Oviedo, Spain
Hospital Univ. Central de Asturias
Oviedo, Spain
Hospital Virgen del Rocío
Seville, Spain
Koagulationsmottagning - SUS
Malmo, Sweden
Koagulationsmottagningen - KS
Solna, Sweden
Sunpasitthiprasong Hospital_Pediatrics Department
Ubon Ratchathani, Mueang Distirct,, Thailand
Ramathibodi Hospital_Department of Haematology
Bangkok, Thailand
Maharaj Nakorn Chiang Mai Hospital _Pediatric Hematology and Oncology
Chiang Mai, Thailand
İstanbul Üniversitesi İstanbul Tıp Fakültesi Hastanesi- Onkoloji Enstitüsü
Capa-ISTANBUL, Capa-ISTANBUL, Turkey (Türkiye)
Akdeniz Üniversitesi Hastanesi- Hematoloji
Antalya, Konyaaltı/ Antalya, Turkey (Türkiye)
Acıbadem Adana Hastanesi-Hematoloji
Adana, Turkey (Türkiye)
Çukurova Üniversitesi Tıp Fakültesi Balcalı Hastanesi- Hematoloji
Adana, 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
Queen Elizabeth Hospital, Birmingham - Haemophilia
Birmingham, United Kingdom
Great Ormond Street Hospital for Children
London, United Kingdom
Manchester Royal Infirmary_Manchester
Manchester, United Kingdom
Queen's Medical Centre - Haemophilia Comprehensive Care Centre
Nottingham, United Kingdom
Royal Hallamshire Hospital
Sheffield, United Kingdom