4255 - explorer™5 efficacy & safety of concizumab in Haem A no inhib
Research type
Research Study
Full title
A Multi-Centre Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients with Severe Haemophilia A without Inhibitors
IRAS ID
226705
Contact name
Pratima Chowdary
Contact email
Sponsor organisation
Novo Nordisk A/S
Eudract number
2016-000614-29
Duration of Study in the UK
2 years, 0 months, 27 days
Research summary
In the genetic disorder haemophilia A, patients are not able to produce adequate amounts of the blood clotting substance factor VIII (FVIII). An impaired blood clotting process is associated with frequent bleeds, which can be painful and lead to disability in joints and muscles. One in every 5,000 male is affected by haemophilia A.\n\nIn severe forms of haemophilia A current standard of care is prophylactic treatment with genetically engineered forms of FVIII. This usually requires a somewhat time-consuming and tedious procedure where the patient has to inject FVIII into his vein 2-3 times weekly. Furthermore, around 30% of the patients develop so-called neutralising antibodies to the injected FVIII, since their immune systems consider the injected FVIII as ‘foreign’. This phenomenon severely impedes treatment options.\nConcizumab is novel molecule (antibody) 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 (so-called phase 2 trials) are required in order to explore and assess the exact level of efficacy of concizumab on the number of bleeds patients experience.\n\nIn the explorer™5 trial, adult patients with severe forms of haemophilia A will be eligible for participation. The trial will be enrolling 36 patients from Europe, US and Asia. Each patient 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 compliance and satisfaction compared to current standard of care with FVIII. The patients will be treated with concizumab for 76 weeks. During this treatment phase additional safety data and efficacy on number of bleeds will be collected and analysed.\n
REC name
London - Hampstead Research Ethics Committee
REC reference
17/LO/0997
Date of REC Opinion
20 Jun 2017
REC opinion
Favourable Opinion