997HA306 - Previously Untreated Patients With Severe Haemophilia A
Research type
Research Study
Full title
An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc; BIIB031) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia A
IRAS ID
156097
Contact name
Raina Liesner
Contact email
Sponsor organisation
Biogen Idec Research Ltd
Eudract number
2013-005512-10
Research summary
Haemophilia A is a genetic bleeding disorder that occurs predominantly in males resulting in a deficiency of coagulation factor VIII (FVIII) activity, a protein essential for blood clotting. Most patients are diagnosed when they are under 2 years old. There is no cure for haemophilia A, so treatment focuses on replacement of FVIII with the intravenous (IV) administration of FVIII-containing coagulation products to promote clotting. Investigational medicine, Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc; BIIB031) is being developed as a treatment for Haemophilia A. rFVIIIFc has been designed to have a longer circulating half life (time taken for the concentration of a drug in the plasma to fall by half the initial level) aiming to provide individuals with prolonged protection and prevention of bleeding with less frequent dosing.
The main purpose of this study is to investigate the safety and efficacy (effectiveness) of rFVIIIFc in previously untreated patients (PUPs). About 125 males younger than 18 years with severe haemophilia A are expected to take part in this study around the world. It is expected that most patients enrolled into this study will be under 2 years old at enrolment. It is expected that the majority of patients will take part in this study for at least 3 years.
All patients will receive rFVIIIFc. After the first dose of rFVIIIFc the study doctor may choose to begin prophylaxis (preventive) treatment OR may choose to treat the participant only when a bleeding episode occurs (episodic treatment) before beginning prophylaxis treatment. The duration of episodic treatment is at the Investigator’s discretion, in accordance with local standard of care.
Surgery is allowed during the study. If a participant develops inhibitors to rFVIIIFc the Investigator will have the option to treat the participant under a immune tolerance induction (ITI) regimen until the participant's body learns not to react to it.REC name
London - Central Research Ethics Committee
REC reference
14/LO/1195
Date of REC Opinion
7 Aug 2014
REC opinion
Favourable Opinion