XTEND-ed
Research type
Research Study
Full title
A Phase 3 open-label, multicenter study of the long-term safety and efficacy of intravenous recombinant coagulation factor VIII Fc-von willebrand factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN; BIVV001) in previously treated patients with severe hemophilia A
IRAS ID
294749
Contact name
Mary Mathias
Contact email
Sponsor organisation
Bioverativ Therapeutics Inc.
Eudract number
2020-002215-22
Clinicaltrials.gov Identifier
Duration of Study in the UK
4 years, 2 months, 1 days
Research summary
: Haemophilia A is a hereditary X-linked bleeding disorder that occurs almost only in males and is characterised by the lack of a blood factor called Factor VIII. Severe Haemophilia A is defined by FVIII levels in the blood less than 1%; the lower the amount the more severe the haemophilia. Individuals with severe haemophilia experience frequent bleeding episodes into major joints, soft tissue and muscle either following a minor bump or spontaneously. The disease can be life threatening or lead to long-term complications. It also has significant effects on physical and mental well-being, quality of life and financial implications for the patient and their family.
In the UK most current therapies are to prevent bleeds (prophylactic). This treatment improves long term outcomes by preventing joint bleeding, joint movement reduction and chronic pain. Prophylactic treatment can cause treatment burden as the treatment has to be given 2-3 times a week, by injection and increases risk of infection. These are often a reason why some patients chose to be treated only when an episode of bleeding (episode treatment) occurs however the likelihood of more bleeds in episode treatment is higher than in patients receiving prophylactic treatment.
BIV001 is a new class of blood clotting Factor VIII used in treatment that is hoped to prevent and control bleeding episodes, maintaining higher activity levels in the blood than currently available treatment and is only required to be administered once a week. This new class of drug prevents and controls bleeding episodes for longer periods of time and potentially reducing the burden of frequent injections and in turn may improve the QoL for these patients.
Most patients will have been receiving BIVV001 in 2 other studies (EFC16295 paediatrics) and EFC16293 (adolescents and adults) and will roll over to this study to continue taking BIVV001.REC name
North West - Liverpool Central Research Ethics Committee
REC reference
21/NW/0119
Date of REC Opinion
28 May 2021
REC opinion
Further Information Favourable Opinion