PK, Safety and Efficacy of IB1001 in patients with severe hemophilia B

• Research type

  Research Study

• Full title

  Pharmacokinetics, Safety and Efficacy of Recombinant Factor IX Product, IB1001, in Patients with Severe Hemophilia B

• IRAS ID

  139429

• Contact name

  Charles Hay

• Contact email

  charles.hay@cmft.nhs.uk

• Sponsor organisation

  Cangene Europe Limited

• Eudract number

  2013-003742-16

• ISRCTN Number

  N/A

• Research summary

  Haemophilia B is an inherited disorder in which the sufferer lacks the blood clotting factor IX, which results in recurrent spontaneous bleeding episodes. The disorder is associated with the X chromosome; males are therefore far more likely to inherit the disorder than females. Treatment for severe 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 investigational drug product IB1001 is a coagulation factor IX intended for intravenous administration. IB1001 is manufactured from genetically engineered Chinese Hamster Ovary (CHO) cells and is under investigation for treatment and prophylaxis of bleeding in haemophilia B patients.

  Following the immune response to CHO cell proteins observed in some subjects treated in another study with IB1001, a manufacturing change has been implemented. The purpose of this study is to evaluate the "polished" product IB1001, and in particular what will happen to it in the body (pharmacokinetics), its safety and its efficacy, in children above 12 years and in adults with severe haemophilia B who have been treated previously with other factor IX products.

  The expected duration of subject study participation is 12 – 15 months. The study is funded by Cangene Europe Limited, UK. It will recruit in the UK, as well as in the USA and in other jurisdictions yet to be identified.

• REC name

  North East - York Research Ethics Committee

• REC reference

  14/NE/0028

• Date of REC Opinion

  29 Apr 2014

• REC opinion

  Further Information Favourable Opinion