8HA02PED-VIII Fc Fusion Protein Paediatric Subjects with Haemophilia A

• Research type

  Research Study

• Full title

  An Open-Label, Multicenter Evaluation of Safety, Pharmacokinetics, and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein, BIIB031, in the Prevention and Treatment of Bleeding Episodes in Pediatric Subjects with Hemophilia A

• IRAS ID

  94006

• Contact name

  Raina Liesner

• Sponsor organisation

  Biogen Idec Research Ltd

• Eudract number

  2011-003073-28

• Clinicaltrials.gov Identifier

  NCT01458106

• Research summary

  50 male patients aged 12 and under receive treatment with recombinant human coagulation factor VIII fusion protein (rFVIIIFc) twice a week to determine the safety of the study drug rFVIIIFc in patients with Haemophilia A. All patients will take part for about 6 to 9 months. This study will also look at the effectiveness of the study drug rFVIIIFc for the prevention and treatment of bleeding episodes. The Pharmacokinetic (Pk) group will have 24 subjects and the non-PK group will have 26 subjects. The PK groups will evaluate the pre-study of FVIII and rFVIIIFc, then the patients will begin twice weekly prophylactic treatment with rFVIIIFc. The subjects with no PK study will proceed directly to twice weekly prophylactic treatment. The study will consist of Screening, PK assessment (PK group only), treatment, and follow-up for the duration of 30 weeks for treatment and follow-up periods. Subjects in PK group, the duration of time needed for screening and PK assessments is approximately 12 weeks. Subjects in the non-PK group, the duration of time needed for screening is approximately 8 weeks.

• REC name

  London - Westminster Research Ethics Committee

• REC reference

  12/LO/0362

• Date of REC Opinion

  29 May 2012

• REC opinion

  Further Information Favourable Opinion