4310 - explorer™4 efficacy and safety of concizumab in inhibitor pts
Research type
Research Study
Full title
A Multi-Centre, Randomised, Open-Label, Controlled Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients with Inhibitors
IRAS ID
226708
Contact name
Kingsley Hampton
Contact email
Sponsor organisation
Novo Nordisk A/S
Eudract number
2016-000510-30
Duration of Study in the UK
2 years, 2 months, 8 days
Research summary
In the genetic disorders haemophilia A and B, patients are not able to produce adequate amounts of the blood clotting substance factors (F) VIII and IX, respectively, leading to frequent bleeds.
In severe forms of haemophilia current standard of care is prophylactic factor replacement. This usually requires a somewhat time-consuming and tedious procedure where the patient has to inject the missing blood clotting factor into his vein 2-3 times weekly. Furthermore, some patients develop so-called neutralising antibodies (‘inhibitors’) to the injected clotting factor, since their immune systems consider the injected factor as ‘foreign’. This severely impedes effective treatment options.
Concizumab is novel molecule that has been engineered to target a separate pathway in the blood clotting process. Early clinical trials conducted so far have demonstrated that concizumab is safe, as well as indicating efficacy in reducing the number of bleeds. However, further clinical trials are required in order to explore and assess the exact level of efficacy of concizumab on the number of bleeds patients experience.
In the explorer™4 trial, adult patients with severe forms of haemophilia A or B with inhibitors will be eligible for participation. Up to 28 participants from Europe, US and Asia will be randomly allocated to receive either concizumab or standard care (factor VIIa). In the concizumab group, each participant will inject himself on a daily basis (~0.2 mL) with concizumab in the skin in the abdominal area using a pen device with a tiny needle. The procedure is expected to offer better efficacy, compliance and satisfaction compared to current standard treatment. After 24 weeks the participants in the FVII group will also receive concizumab on a daily basis. Participants will be treated with concizumab for up to 76 weeks. During this treatment phase additional safety data and efficacy will be collected and analysed.
REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
17/NW/0374
Date of REC Opinion
21 Jun 2017
REC opinion
Favourable Opinion