40K Pegylated N8 in Non Bleeding Patients with Haemophilia A

• Research type

  Research Study

• Full title

  A Multi-National, Open-Label, Dose Escalation Trial, Evaluating Safety and Pharmacokinetics of Intravenous Doses of NNC 0129-0000-1003 in Patients with Haemophilia A

• IRAS ID

  58213

• Contact name

  Benny Sorensen

• Sponsor organisation

  Novo Nordisk A/S

• Eudract number

  2010-018520-68

• ISRCTN Number

  n/a

• Research summary

  The trial is a multi-national, open-label, dose escalation trial evaluating safety and pharmacokinetics (PK) of three i.v. doses of 40K PEG-N8 in non-bleeding patients with severe haemophilia A without inhibitors. Haemophilia A patients often produce antibodies against FVIII treatment (inhibitors) and these antibodies neutralise FVIII treatment making it ineffective. Haemophilia A is a recessive X-linked congenital (inherited) bleeding disorder affecting approximately 1 in 5,000 males. The trial is a first human dose trial. All patients will be given a single dose of their current FVIII product at the same dose level as 40K PEG-N8, however, the dose for previous FVIII will not exceed 75 IU/kg. The planned dose levels are 25 U/kg, 50 U/kg and 75 U/kg and each patient will only receive one dose. The trial is designed to provide information regarding the safety profile and pharmacokinetics (PK-measuring what happens to the drug in the body) of 40K PEG-N8 after a single intravenous (into a vein) administration to patients with Haemophilia A.40K PEG-N8 is produced by Novo Nordisk A/S in a laboratory using genetic recombinant methods and is produced without using any animal products (free of animal based serum). A large molecule of polyethylene glycol (PEG) is attached to the rFVIII molecules thereby creating a compound which is expected to have a longer effect than normal rFVIII by being present for longer in the blood. The aim is that this drug can be injected once a week rather than current rFVIII products which need to be injected 2-3 times per week. 40K PEG-N8 has previously only been tested in animals. A maximum of 30 patients will be recruited into this trial with a view of having 18 patients treated with study drug and complete the trial. A patient will be in the trial for approximately 9 weeks.

• REC name

  South Central - Oxford B Research Ethics Committee

• REC reference

  10/H0605/50

• Date of REC Opinion

  16 Sep 2010

• REC opinion

  Further Information Favourable Opinion