rFIXFc in Patients with Haemophilia B
Research type
Research Study
Full title
B-LONG: An Open-label, Multicenter Evaluation of the Safety, Pharmacokinetics, and Efficacy of Recombinant, Long-acting Coagulation Factor IX Fc fusion (rFIXFc) in the Prevention and Treatment of Bleeding in Previously Treated Subjects With Severe Hemophilia B
IRAS ID
44728
Contact name
K John Pasi
Sponsor organisation
Biogen Idec Inc
Eudract number
2009-014295-21
Clinicaltrials.gov Identifier
Research summary
Haemophilia B is a bleeding disorder caused by a deficiency in Factor IX (FIX, a protein in the blood that is needed for clotting). This deficiency results in bleeding into joints, soft tissue and muscles. Depending on the severity of the bleed it can be life-threatening if not treated appropriately. There is no cure for haemophilia B so treatment focuses on the injection of products containing FIX. The goal of treatment is to raise the level of FIX in the blood to stop bleeding (on-demand treatment) or prevent bleeding (prophylaxis treatment). Recombinant Factor IX Fc fusion protein (rFIXFc) has been engineered to potentially be longer-acting than current treatments which would mean injections have to be given less frequently. rFIXFc has been administered to 14 patients and was generally well-tolerated. The purpose of this study is to investigate the safety, pharmacokinetics (the amount of drug in blood over time), and efficacy (how long it works and how long it circulates) of rFIXFc in previously treated participants with severe hemophilia B. This is an open label, non-randomised study with 4 arms: Arm 1 investigates a low dose of rFIXFc for prophylaxis treatment Arm 2 investigates a high dose of rFIXFc for prophylaxis treatment Arm 3 investigates on-demand treatment Arm 4 investigates treatment for patients undergoing surgery Males with severe haemophilia B aged 12 and older and weighing at least 40 kg may be eligible to participate in the study. All patients will be followed for any clinically notable changes from baseline in physical examination, vital signs, lab values, and incidence of adverse events, including inhibitor development. The number of breakthrough bleeding episodes will also be monitored. Patients will be in the study for approximately 35-61 weeks. This study is sponsored by Biogen Idec Inc. Approximately 75 patients will participate in this study worldwide.
REC name
London - Central Research Ethics Committee
REC reference
10/H0718/30
Date of REC Opinion
5 Jul 2010
REC opinion
Further Information Favourable Opinion