F13CD-3720 Extension study to F13CD-1725 (Congential FXIII deficiency)

• Research type

  Research Study

• Full title

  A Multi-Centre, Open-Label, Single-Arm, and Multiple Dosing Trial on Safety of Monthly Replacement Therapy with Recombinant Factor XIII (rFXIII) in Subjects with Congenital Factor XIII Deficiency. (Safety Extension Trial to F13CD-1725)

• IRAS ID

  21442

• Contact name

  Andrew Mumford

• Eudract number

  2008-007883-41

• ISRCTN Number

  n/a

• Clinicaltrials.gov Identifier

  n/a

• Research summary

  This study is a safety extension trial to the phase 3a (F13CD-1725) trial of rFXIII. The participants are patients with Congenital factor XIII deficiency, a very rare genetic bleeding disorder caused by low levels of FXIII activity in the blood. FXIII has an important role in the blood clotting process. People with congenital FXIII deficiency have a lifelong tendency for abnormal bleeding, either spontaneously or after trivial injuries. In patients with severe FXIII deficiency bleeds may occur in muscles or joints but there can also be life-threatening and disabling bleeding into the brain or the spinal cord. Treatment for Congenital FXIII deficiency has been available for about 10 years and usually involves monthly infusions of a concentrate of the FXIII protein which helps restore blood clotting to normal and prevent bleeding. The FXIII concentrate currently available as a marketed product is manufactured from human blood donations. The manufacturing process is involves sophisticated purification steps. However, as is the case for all other clotting factor concentrates made from human plasma, we cannot absolutely eliminate the risk of contamination with certain viruses or prions (the agent which causes new variant cJD). A bio-engineered synthetic (recombinant) clotting factor concentrate (rFXIII) replacement therapy has now been developed for Congenital FXIII deficiency that offers to provide much safer treatment. The phase 3a trial is completed. The safety extension trial described in this application will allow the participants to continue receiving rFXIII until it is available as a marketed product. The data generated will contribute valuable information to the safety profile of rFXIII.

• REC name

  West Midlands - Coventry & Warwickshire Research Ethics Committee

• REC reference

  09/H1211/91

• Date of REC Opinion

  4 Nov 2009

• REC opinion

  Favourable Opinion