Human-cl rhFVIII in Severe Haemophilia A (GENA-08)

• Research type

  Research Study

• Full title

  Clinical Study to Investigate the Efficacy, Safety, and Immunogenicity of human-cl rhFVIII in Previously Treated Patients With Severe Haemophilia A

• IRAS ID

  27912

• Sponsor organisation

  Octapharma AG

• Eudract number

  2009-011055-43

• ISRCTN Number

  Not Known

• Research summary

  This is a multi-centre study taking place in the UK, Germany, Austria and Bulgaria. In total, 32 evaluable previously treated patients suffering from severe haemophilia A will be enrolled. Haemophilia A is an inherited disorder resulting from a deficiency of blood coagulation factor (FVIIII), which causes bleeding. This disorder can be effectively managed by infusing normal human plasma-derived or recombinant (r) FVIII concentrates. Until today, rFVIII has been produced exclusively using hamster cells. However, they may contain non-human contaminants or compounds capable of eliciting an immune response (epitopes). These may give rise to immune reactions when rFVIII is administered to humans and may render infused FVIII ineffective. Human-cl rhFVIII is derived from a genetically modified human cell-line, which ensures that compounds of non-human material capable of provoking an immune response are absent. The pharmacokinetic properties, safety and efficacy of this treatment have already been studied in a previous clinical study in 22 patients. The aim of the current study is to investigate the efficacy and safety of Human-cl rhFVIII and its capacity to cause an immune response when given every other day for 6 months for the prevention of bleeding. Human-cl rhFVIII will also be used to treat break-through bleeds and for prevention of bleeds during surgery. Following confirmation of eligibility to enter the study, each patient will visit the study centre 5 times and give blood samples to monitor routine safety laboratory parameters and to check whether the patient has developed an immune response (inhibitor) to FVIII. In addition, on 3 occasions during the study the amount of infused drug in the patient??s blood will be measured (in-vivo recovery), since a significantly reduced in-vivo recovery may hint that a FVIII inhibitor has developed.

• REC name

  London - Riverside Research Ethics Committee

• REC reference

  09/H0706/67

• Date of REC Opinion

  30 Oct 2009

• REC opinion

  Further Information Favourable Opinion