998HB303 Previously Untreated Patients With Severe Haemophilia B
Research type
Research Study
Full title
An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc; BIIB029) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia B
IRAS ID
150097
Contact name
Raina Liesner
Contact email
Sponsor organisation
Biogen Idec Research Ltd
Eudract number
2013-003629-27
Research summary
Haemophilia B is a genetic bleeding disorder that occurs predominantly in males resulting in a deficiency of coagulation factor IX (FIX), an enzyme involved in blood clotting. There is no currently available cure for haemophilia B. Current treatment focuses on factor replacement therapy. Investigational medicine, Recombinant coagulation factor IX Fc fusion protein (rFIXFc; BIIB029) is being developed as a treatment for haemophilia B. rFIXFc 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, while maintaining the activity profile of FIX.
The main purpose of the study is to determine the safety and effectiveness of rFIXFc in previously untreated male subjects with haemophilia B. About 60 males younger than 18 years are expected to take part in this study around the world (majority of the patients recruited will be infants and young children). It is expected that the majority of participants will take part in this study for at least 3 years.
This is an open-label study which means that each patient and the study staff will know what dose of rFIXFc the participant is being treated with. After the first dose of rFIXFc, the Investigator 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 developes inhibitors to rFIXFc 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/0602
Date of REC Opinion
8 Jul 2014
REC opinion
Further Information Favourable Opinion