Continuation Study of Prophylactic BAX 855 in PTP with Haemophilia A
Research type
Research Study
Full title
A Phase 3b Continuation study of the Safety and Efficacy of PEGylated Recombinant Factor VIII (PEG-rFVIII; BAX 855) in Prophylaxis of Bleeding in Previously Treated Patients with Severe Hemophilia A
IRAS ID
138498
Contact name
Pratima Chowdary
Contact email
Sponsor organisation
Baxter Innovations GmbH
Eudract number
2013-002236-24
Clinicaltrials.gov Identifier
Research summary
This is a Continuation study of the Safety and Efficacy of Factor VIII(BAX 855) in Prophylaxis of Bleeding in Previously Treated Patients with Severe Hemophilia 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 removed from the circulation more slowly and so work for longer than their unpegylated counterparts. PEGylated proteins may be less susceptible to antibodies (part of the body’s defence system which stops Factor VIII products, including ADVATE, from working properly).In addition, PEGylated proteins might be less likely to be neutralised (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 is currently used. This would mean that people with haemophilia would need injections less frequently and may bleed less frequently when using the product regularly to prevent bleeding (prophylaxis).The subject participation period is estimated to beapproximately 6 to 36 months.All subjects will continue on study until a minimum of 100 Exposure days per subject has been achieved across all BAX 855 studies in which each subject participated. Following 100 ED,subjects will be given the option to continue until the study is terminated.REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
14/NW/0001
Date of REC Opinion
11 Feb 2014
REC opinion
Further Information Favourable Opinion