Treatment with Human-cl rhFVIII in Patients with Severe Haemophilia A

• Research type

  Research Study

• Full title

  Immunogenicity, Efficacy and Safety of Treatment with Human-cl rhFVIII in Previously Untreated Patients with Severe Haemophilia A

• IRAS ID

  117313

• Contact name

  Raina J Liesner

• Sponsor organisation

  Octapharma AG

• Eudract number

  2012-002554-23

• ISRCTN Number

  ISRCTN

• Research summary

  This study is designed as a prospective, multicentre, multinational, (approximately 40 centres worldwide) open label, non-controlled phase III study in 100 male patients without any previous treatment with factor VIII (FVIII) or other blood products containing FVIII who are suffering from severe haemophilia A. For each patient the exposure and the effect (efficacy) of Human-cl rhFVIII in the prevention and treatment of bleeds, the frequency of break through bleeds in the case of prophylactic treatment and the tolerability and safety of Human-cl rhFVIII will be thoroughly assessed.Screening Visit, Follow up Visits, Three Monthly Visit and Completion Visit will be performed. In the course of the follow up visits (i.e. every 3-4 exposure days (ED) (s) until ED 20, then every 10-12 ED until ED 100 scheduled to be performed after the Screening Visit. For patients that develop FVIII inhibitors which are a patient immune system response to the exposure to FVIII, Immune Tolerance Induction treatment (ITI) will commence. Levels of FVIII inhibitors will be assessed for each patient.A patient completes the study by reaching 100 EDs or after a maximum of 5 years. Patients who receive ITI treatment, the maximum length of ITI treatment will be 36 months, after which the patient will be withdrawn from the study. For patients undergoing surgical interventions during the study treatment details will be documented before during and after surgery respectively.The study is planned to start in the 4th quarter of 2012 and continues until the 4th quarter of 2018.

• REC name

  London - Central Research Ethics Committee

• REC reference

  12/LO/1881

• Date of REC Opinion

  19 Feb 2013

• REC opinion

  Further Information Favourable Opinion