Safety extension study to 3760: Monthly rFXIII replacement therapy

• Research type

  Research Study

• Full title

  Safety and efficacy evaluation of Recombinant Factor XIII for Prevention of Bleeding in Paediatric Patients with FXIII A-subunit Congenital Deficiency.

• IRAS ID

  58399

• Contact name

  Andrew Will

• Sponsor organisation

  Novo Nordisk Ltd

• Eudract number

  2010-020192-23

• ISRCTN Number

  N/A

• Clinicaltrials.gov Identifier

  N/A

• Research summary

  This study is a safety extension trial to the planned phase 3b (F13CD-3760) paediatric pharmacokinetic and safety profile study. The participants are patients aged between 1 and 6 years with congenital factor XIII deficiency, a very rare genetic bleeding disorder caused by low levels of FXII activity in the blood. Congenital FXIII deficiency is usually identified early, as delayed bleeding from the umbilical stump or circumcision site in newborn infants. As such prophylaxis treatment with a FXIII containing product is started in infancy once diagnosed. People with FXIII deficiency have a lifelong tendency for abnormal bleeding, either spontaneously or after trivial injuries. In severe FXIII deficiency bleeds may occur in muscles or joints but can also be life-threatening and disabling bleeding into the brain or spinal cord. Treatment for FXIII deficiency involves monthly infusions of a concentrate of FXIII protein which helps restore blood clotting to normal preventing bleeding. The FXIII concentrate currently available as a marketed product is manufactured from human blood donations and involves sophisticated purification steps. However, clotting factor concentrates made from human plasma cannot absolutely eliminate the risk of contamination with certain viruses or prions (the agent which causes new variant cJD).A recombinant (made in the laboratory) clotting factor concentrate replacement therapy has been developed for FXIII deficiency and offers to provide a safer treatment ss there is no direct clinical benefit to participants in the safety study, entry into 3835 allows participants to continue to receive rFXIII until the drug is commercially available. This means that once participants switch from plasma derived to a recombinant product they do not have to switch back to the plasma product.

• REC name

  South Central - Oxford A Research Ethics Committee

• REC reference

  10/H0604/68

• Date of REC Opinion

  20 Sep 2010

• REC opinion

  Favourable Opinion