4616(explorer10) concizumab prophylaxis in children below 12 years with haemophilia
Research type
Research Study
Full title
Open-label study investigating efficacy, safety and pharmacokinetics of concizumab prophylaxis in children below 12 years with haemophilia A or B with or without inhibitors
IRAS ID
1004198
Contact name
Clinical Transparency
Contact email
Sponsor organisation
Novo Nordisk A/S
Eudract number
2020-000504-11
Clinicaltrials.gov Identifier
Research summary
Haemophilia is a bleeding disorder that some people are born with. The most common type of haemophilia is called haemophilia A which means the person has too little clotting factor VIII in the blood. A less common type is called haemophilia B which 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.
For some people, these products stop working because they have developed resistance to the therapy. This is called an inhibitor. Products used to treat haemophilia with inhibitors are called bypassing agents.
Products for the treatment of haemophilia A or B, with or without inhibitors are usually given as injections into the vein.
There will be two ways to enter this study (also known as ‘arms’). Arm 1 will look at how well a new drug, concizumab works to prevent bleeds, in children below 12 years with haemophilia A or B, with or without inhibitors. Arm 2 will look at the safety of concizumab in patients transferring from a compassionate use trial, these patients can be any age. The compassionate use trial is not currently being run in the UK and therefore we will only be enrolling patients into Arm 1 in the UK.Preventing bleeds is also known as prophylaxis. Concizumab is given as an injection under the skin.
All patients will be given concizumab prophylaxis for 104 weeks, which will be split into a ‘main part’ and an ‘extension part’. There will be no change in treatment between these two parts.
Patients in the study will be seen after 1 week, with a visit 3 weeks later, all subsequent visits will occur every 4 weeks during the main part of the study and then patients will be seen every 8 weeks. Over the entire study there will be 20 visits and patients will be in the study for just over 2 years.
Patients will be recruited at specialist Haemophilia centres.REC name
London - Brent Research Ethics Committee
REC reference
21/LO/0829
Date of REC Opinion
6 Jan 2022
REC opinion
Further Information Favourable Opinion