Study of Recombinant Factor IX Product, IB1001, in Previously Treated
Research type
Research Study
Full title
Study of Inspiration’s Recombinant Factor IX Product, IB1001, in Previously Treated Pediatric Subjects with Hemophilia B
IRAS ID
73021
Contact name
Ri Liesner
Sponsor organisation
Inspiration Biopharmaceuticals, Inc.
Eudract number
2010-024190-39
Clinicaltrials.gov Identifier
Research summary
Study of IB1001 in paediatric patients with Hemophilia BHaemophilia B is an inherited disorder in which the sufferer lacks the blood clotting factor IX, which results in recurrent spontaneous bleeding episodes. Treatment for haemophilia B involves administering factor IX replacement therapy on an as-needed basis or as a preventative measure (prophylaxis). Despite the fact that factor IX has been marketed for more than ten years, there is still a lack of adequate information to guide treatment of children. Moreover, products that are more cost effective have fewer cases of inhibitor development, and fewer numbers of breakthrough bleeding episodes are desired. The purpose of this study is to obtain data that can be used to evaluate treatment of severe haemophilia B in children aged 0-12 years who have been treated previously with other factor IX products. The study will evaluate what will happen to the product in the body (pharmacokinetics), its safety and its efficacy. Data from another ongoing study with IB1001 support the expectation that IB1001 should be safe and effective in the treatment and prevention of bleeding in subjects with severe haemophilia B.The patients will undergo an initial pharmacokinetics evaluation which consists of 3 visits over 4 days. Patients will then enter the prophylaxis part of the study, where they will receive IB1001 twice weekly, or the on-demand treatment part, where they will receive IB1001 at the time of a bleeding episode. IB1001 is administered via intravenous injection. Treatment will last approximately 6 months, and patients will be able to continue on treatment if the product is not licensed at that time.The study is funded by Inspiration Biopharmaceuticals. It will recruit in the UK, Poland, Mexico and Romania, as well as Brazil, India and Turkey. It has been opened in the USA.
REC name
Yorkshire & The Humber - Leeds East Research Ethics Committee
REC reference
11/YH/0145
Date of REC Opinion
20 Jun 2011
REC opinion
Further Information Favourable Opinion