Long Acting Factor VII Dose Finding PK Study in Haemophilia A and B

• Research type

  Research Study

• Full title

  An Exploratory Multi-centre, Multi-national, Randomised, Double Blinded, Parallel Arm Trial Evaluating Safety, Pharmacokinetics and Dose-Finding of Prophylactic Administration of Long Acting rFVIIa (LA-rFVIIa) in Haemophilia A or B Patients with Inhibitors

• IRAS ID

  26497

• Contact name

  Paul Giangrande

• Sponsor organisation

  Novo Nordisk A/S

• Eudract number

  2008-006424-54

• ISRCTN Number

  n/a

• Research summary

  Haemophilia is a sex-linked hereditary blood defect that occurs almost exclusively in males and is characterised by delayed clotting of the blood and consequent difficulty in controlling haemorrhage even after minor injuries. Haemophilia A patients lack clotting factor VIII and haemophilia B patients lack factor IX. Treatment is intravenous injections of either FVIII or FIX. In many patients the body produces antibodies (or inhibitors) against FVIII or FIX making treatment ineffective. This means patients need to use FVII which by-passes the need for FVIII or FIX and results in successful clotting of the blood. Early treatment of bleeding events is important to prevent long term damage to joints over time which can lead to disability and impairment of quality of life. By using a preventative or prophylactic regimen it is anticipated that at the correct dose bleeding episodes should be minimised. The drug in this study is a long acting form of FVII giving injections every 2nd day to reduce the number of bleeds. This study consists of 3 phases - observation where participants use rFVIIa to treat bleeds that occur; treatment phase at one of 3 different doses of study drug every 2nd day with the aim of preventing bleeds. If bleeds occur they will be treated with rFVIIa; and a follow-up phase of 4-8 weeks using only rFVIIa to treat bleeds. Twenty four haemophilia A or B patients without inhibitors will be screened to have 18 participants complete the trial (6 participants per dose level). Study duration will be approximately 7-11 months. The study will be carried out at haemophilia centres across the UK where participants are well known to the sites and currently under the care of the investigators who will be taking part in this trial.

• REC name

  South Central - Oxford B Research Ethics Committee

• REC reference

  09/H0605/108

• Date of REC Opinion

  14 Jan 2010

• REC opinion

  Further Information Favourable Opinion