40K Pegylated Recombinant FIX Non Bleeding Patients with Haemophilia B

• Research type

  Research Study

• Full title

  A Multi-Centre, Multi-National, Open-Label, Dose Escalation Trial Evaluating Safety and Pharmacokinetics of Ascending Intravenous Doses of 40K Pegylated Recombinant FIX in Non-Bleeding Patients with Haemophilia B

• IRAS ID

  20824

• Contact name

  Edward G D Tuddenham

• Eudract number

  2009-011085-28

• ISRCTN Number

  Not submitted

• Research summary

  This study is a multi-centre, multinational, open-label, dose escalation trial evaluating safety and Pharmacokinetics (PK) of three ascending i.v. doses of 40K PEG-rFIX (N9-GP) in non-bleeding patients with haemophilia B. The trial is the first human dose trial with N9-GP. The doses are 25 U/kg, 50 U/kg and 100 U/kg. Five patients will be allocated to each of the three dose levels. This trial is designed to provide information regarding the safety profile and the pharmacokinetics (PK - measuring what happens to the drug in the body) of N9-GP after a single intravenous (into a vein) administration to patients with haemophilia BN9-GP is produced by Novo Nordisk A/S in a laboratory using genetic recombinant methods and is produced without use of any animal products (free of animal based serum). A large molecule of polyethylene glycol (PEG) is attached to the rFIX molecules thereby creating a compound which is expected to have a longer effect than normal rFIX (BeneFIX©) or plasma derived FIX products by being present for longer time in the blood. N9-GP has previously only been tested in animals.Haemophilia B is a recessive X-linked congenital (inherited) bleeding disorder, caused by mutations (changes) in the coagulation (clotting) factor nine (FIX) gene located in the distal part on the long arm of the X-chromosome. Approximately 1 in 25,000 males have haemophilia B.A total of 20 patients were screened. Two patients were screening failures (i.e. 18 patients were enrolled) and two patients were withdrawn before dosing with N9-GP resulting in 16 patients exposed to N9-GP. The total duration of the trial per patient was approximately 8 weeks.The trial was conducted at 14 sites in 6 countries (1 site in France, 4 sites in Germany, 3 sites in Japan, 1 site in Spain, 3 sites in UK, and 2 sites in US). The trial was conducted in EU countries (UK, France, Germany, and Spain), Japan and USA.

• REC name

  London - Hampstead Research Ethics Committee

• REC reference

  09/H0720/84

• Date of REC Opinion

  3 Jun 2009

• REC opinion

  Favourable Opinion