First in Human study of BAX 855 in Haemophilia A patients

• Research type

  Research Study

• Full title

  A phase 1, prospective, open label, cross-over, dose-escalation study in previously treated patients with severe (FVIII less than 1 percent) hemophilia A

• IRAS ID

  85848

• Sponsor organisation

  Baxter Innovations GmbH

• Eudract number

  2011-002011-28

• Research summary

  This is a first in human study in which we are investigating the safety, tolerability, breakdown and elimination from the body of a new drug BAX 855 (PEGylated Recombinant Factor VIII) in comparison to with the licensed drug ADVATE (Recombinant Factor VIII) in patients with severe haemophilia A.People with haemophilia A do not produce enough factor VIII. Factor VIII is a protein necessary for blood to clot effectively. If left untreated this results in uncontrolled bleeding either spontaneously or following minor injury/trauma. The prevention of such bleeding episodes is of vital importance to avoid complications from abnormal bleeding.Injections of Factor VIII e.g. ADVATE are used to prevent and control bleeding in people with haemophilia A. As the duration of action of these medicines is not very long, patients need repeated administration every few days. BAX 855 is a PEGylated form of genetically-engineered (recombinant) Factor VIII. The Factor VIII is linked to a chemical called polyethylene glycol (PEG) 'pegylated'). Pegylated proteins are less susceptible to antibodies (part of body's defence system which stop Factor VIII products including ADVATE from working properly). In addition, pegylated proteins are not as likely to be neutralized (rendered inactive) by inhibitors in the body. It is therefore hoped that BAX 855 will stay in the body for longer and have a longer duration of activity than the medicine that's currently used. This would mean that people with haemophilia A would need injections less frequently.The study will comprise of 2 groups of 8 subjects. Each subject in group 1 will receive a single intravenous infusion of BAX855 and ADVATE separately. After the safety evaluation, the subjects in group 2 will receive the next planned higher dose of BAX855 and ADVATE. Each subject will participate in the study for 10 to 15 weeks.The study will take place at Quintiles Drug Research Unit at Guy's Hospital.

• REC name

  London - London Bridge Research Ethics Committee

• REC reference

  11/LO/1079

• Date of REC Opinion

  19 Sep 2011

• REC opinion

  Further Information Favourable Opinion