OBI-1-302 Congenital Hemophila A Study

• Research type

  Research Study

• Full title

  Efficacy and Safety of B-Domain Deleted Recombinant Porcine Factor VIII (OBI-1) in the Treatment of Patients with Congenital Hemophilia A With Factor VIII Inhibitors

• IRAS ID

  81403

• Contact name

  Ri Liesner

• Sponsor organisation

  Inspiration Biopharmaceuticals, Inc.

• Eudract number

  2011-001899-18

• ISRCTN Number

  n/a

• Research summary

  This study is testing whether the study drug (OBI-1) is effective and safe for the treatment of serious bleeding episodes in people with congenital hemophilia with factor VIII inhibitors, an inherited blood clotting disorder in which one of the required proteins, factor VIII, is absent or not functioning. Treatment is with replacement human factor VIII. Some patients develop antibodies to human factor VIII and no longer respond to the replacement therapy. OBI-1 is a blood clotting protein made from porcine factor VIII. This factor VIII works similarly to human factor VIII, but the antibodies that block human factor VIII function will be less likely to stop OBI-1 from functioning. This study is being conducted in patients with congenital hemophilia A with factor VIII inhibitors experiencing a serious bleeding episode have a history of or are currently experiencing a suboptimal response to treatment with bypass agents. Subjects will be treated in a hospital setting and the study drug will be given by infusion. Dosing is at the discretion of the investigator, and the amount and frequency of the dose given will be determined by the extent of the subject??s bleeding, their response to treatment, their factor VIII blood levels and their inhibitor titers. Unless the subject elects to withdraw consent or the treatment proves not to be effective for that subject, the treatment will continue until the bleeding is controlled and the bleed site is adequately healed. The subject??s response to the therapy will be regularly assessed by the investigator. Clinical evaluation of the bleed site and regular monitoring of blood factor VIII levels will contribute to the evaluation of the response. The primary measure of the treatment??s efficacy will be made 24 hours after the initial dose, by the investigator, using a well defined 3-point scale.

• REC name

  Yorkshire & The Humber - Leeds East Research Ethics Committee

• REC reference

  11/YH/0336

• Date of REC Opinion

  25 Oct 2011

• REC opinion

  Further Information Favourable Opinion