GENA-03

• Research type

  Research Study

• Full title

  PROSPECTIVE CLINICAL STUDY IN CHILDREN WITH SEVERE HAEMOPHILIA A TO INVESTIGATE CLINICAL EFFICACY, IMMUNOGENICITY, PHARMACOKINETICS, AND SAFETY OF HUMAN-CL RHFVIII

• IRAS ID

  55807

• Contact name

  Raina Liesner

• Sponsor organisation

  Octapharma AG

• Eudract number

  2010-018644-14

• ISRCTN Number

  Not Known

• Research summary

  Twenty years after the introduction of recombinant( genetically engineered) FactorVIII (FVIII) derived from hamster cell lin's, Octapharma have developed a new recombinant FVIII which uses a human cell line therefore reducing exposure to animal proteins. This submission is for a Prospective, non-controlled, open-labelled, multi-national, multicentre Crossover PK phase III study in 60 children with haemophilia A (FVIII:C <1%).(50 [2 x 25] evaluable subjects are needed)to compensate drop outs allocated to one of two age cohorts of each-30 PTPs (previously treated patients aged 2-5 years, and-30 PTPs aged 6-12 years in approximately 15-20 European'study centersthe investigation of pharmacokinetic properties of Human-cl rhFVIII in up to 15 subjects (13 evaluable) of each age cohort is also part of the study followed by an open 6 months prophylactic ( preventing, rather than treating)treatment period for at least 50 EDs (exposure days).A follow up study lasting until product launch in the individual countries is planned.Octapharma can promise but cannot guarantee that such a follow-up study can be initiated.

• REC name

  London - Riverside Research Ethics Committee

• REC reference

  10/H0706/48

• Date of REC Opinion

  15 Dec 2010

• REC opinion

  Further Information Favourable Opinion