NN7088-3885 Paediatric PTP Study in Haemophilia A

• Research type

  Research Study

• Full title

  NN7088-3885 (Pathfinder 5): A multinational, open-label, non-controlled trial on safety, efficacy and pharmacokinetics of NNC 0129-0000-1003 in previously treated paediatric patients with severe haemophilia A

• IRAS ID

  119278

• Contact name

  Jayanthi Alamelu

• Sponsor organisation

  Novo Nordisk Ltd

• Eudract number

  2012-001711-23

• ISRCTN Number

  N/A

• Clinicaltrials.gov Identifier

  N/A

• Research summary

  Development of inhibitory antibodies is considered the most serious complication of replacement therapy with any coagulation factor product. This trial is designed to minimise the risk of inhibitor development. The anticipated benefits of the study drug include reduced frequency and number of required injections compared with existing FVIII treatments due to the half-life of the study drug. Lack of compliance with a frequent injection schedule is one of the most commonly cited reasons for failure of prophylaxis with factor treatments. Frequent dosing is known to interrupt and restrict daily activities. Fewer injections will preserve the vein status and presumably lead to better compliance and less interruption of daily life and thereby improve the quality of life of the patients and their family. This study is looking for children aged under 12 years old who have severe haemophilia A and have previously been treated with FVIII products. The study drug being tested in this trial is a recombinant FVIII product with a view to fewer injections due to the half-life of the study drug. Children aged between 0-12 years old with severe haemophilia A who have had previous treatment with other FVIII products would be eligible. Eligibility criteria have been designed in order to exclude potentially fragile paediatric patients where the treatment with study drug may present a risk. Patients with a history of inhibitors as well as patients with present inhibitors are not eligible for the trials. The study is being carried out in Haemophilia Centres and Comprehensive Care Centres across England covering the North, Midlands and South over 6 centres. Participants will be treated twice weekly with fixed dose of study drug for 26 weeks. Participants continuing in the extension phase must remain on the twice weekly prophylaxis regimen until one year of treatment is completed.

• REC name

  North East - Newcastle & North Tyneside 1 Research Ethics Committee

• REC reference

  13/NE/0001

• Date of REC Opinion

  6 Feb 2013

• REC opinion

  Further Information Favourable Opinion