4311(explorer7) Concizumab prophylaxis in patients with inhibitors
Research type
Research Study
Full title
Efficacy and Safety of Concizumab prophylaxis in patients with haemophilia A or B with inhibitors
IRAS ID
269017
Contact name
Michela Nielsen
Contact email
Sponsor organisation
Novo Nordisk A/S
Eudract number
2018-004889-34
Duration of Study in the UK
3 years, 4 months, 21 days
Research summary
Haemophilia is a bleeding disorder that some people are born with. There are two types: haemophilia A (most common) and haemophilia B (rare). People with Haemophilia A have too little clotting factor VIII in the blood, those with haemophilia B have too little clotting factor IX in the blood. Many people with haemophilia A or B use something called replacement factor therapy. For some people, these products stop working because they have developed resistance to the therapy. This is called an inhibitor. Treatment of patients with inhibitors is therefore problematic. Products used to treat haemophilia with inhibitors are called bypassing agents and are usually given as injections into the vein.
In this study we will look at how well the study drug, concizumab, taken as an under the skin injection, works to prevent bleeds in people who have developed an inhibitor towards either factor VIII or factor IX. Concizumab aspires to address the high unmet medical need in haemophilia patients with inhibitors by offering a treatment injected under the skin to prevent bleeds. Preventing bleeds is also known as prophylaxis.
Patients currently treated only when they have a bleed (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 prophylaxis will be allocated to receive concizumab prophylaxis from the start of the study.
Patients entered into the study will be seen after 1 week, then every 4 weeks for the first 24 weeks. After this, patients will be seen every 8 weeks. Over the entire study there will be 29 visits over approximately 3 years.
Patients will be recruited at specialist Haemophilia centres.REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
19/NW/0490
Date of REC Opinion
30 Sep 2019
REC opinion
Further Information Favourable Opinion