4307 (explorer8) efficacy of concizumab in HA/HB without inhibitors
Research type
Research Study
Full title
Efficacy and Safety of Concizumab prophylaxis in patients with haemophilia A or B without inhibitors
IRAS ID
269096
Contact name
Michela Nielsen
Contact email
Sponsor organisation
Novo Nordisk A/S
Eudract number
2018-004891-36
Duration of Study in the UK
3 years, 3 months, 27 days
Research summary
Haemophilia is a bleeding disorder that some people are born with. The most common type of haemophilia is called haemophilia A. This means the person has too little clotting factor VIII in the blood. A less common type is called haemophilia B. This means the person has too little clotting factor IX in the blood. Many people with haemophilia A or B use something called replacement factor therapy. Usually the products to treat haemophilia are given as injections into the vein.
In this study we will look at how well a new drug, concizumab works to prevent bleeds in people with haemophilia A or B (without inhibitors). Preventing bleeds is also known as prophylaxis. Concizumab is given as an injection under the skin.
Patients currently treated only when they have a bleed (known as on-demand) will be randomised to either concizumab prophylaxis or no prophylaxis (continuing on their current on-demand treatment regimen) for 24 weeks. After this all patients will receive concizumab prophylaxis for a further 136 weeks.
Patients currently treated with regular factor prophylaxis will be assigned into the non-randomised treatment arms and receive concizumab prophylaxis from the start of the study.
Patients entered into the study will be seen after 1 week and then every 4 weeks during the main part of the study (24 weeks). After this, patients will be seen every 8 weeks. Over the entire study there will be 29 visits and patients will be in the study for just over 3 years.
Patients will be recruited at specialist Haemophilia centres.REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
19/NW/0476
Date of REC Opinion
30 Sep 2019
REC opinion
Further Information Favourable Opinion