Safety and PK study of rFXIII in 1-6 year olds with FXIII deficiency

• Research type

  Research Study

• Full title

  A Phase 3b Trial Investigating the Pharmacokinetics and Safety Profile of a Single Intravenous Dose of rFXIII in Paediatric (1 to less than 6 Years Old) Subjects with Congenital FXIII A-subunit Deficiency

• IRAS ID

  58678

• Contact name

  Andrew Will

• Sponsor organisation

  Novo Nordisk Ltd

• Eudract number

  2009-016869-28

• ISRCTN Number

  N/A

• Clinicaltrials.gov Identifier

  N/A

• Research summary

  This study consists of a single intravenous (into the vein) dose of a blood clotting factor (recombinant Factor XIII (rFXIII)) in children (1 to < 6 years old) with congenital FXIII deficiency. This study will evaluate the safety and pharmacokinetics (how the drug is absorbed, distributed, metabolised and excreted by the body) of rFXIII in children. 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. Another phase 3 study is nearing completion in patients 6 years and above (1725). The study described in this application will generate valuable information to the safety profile of rFXIII in patients between 1 and <6 years of age. The follow-on safety extension trial, F13CD-3835, will enable patients to continue to receive the study drug until it is available as a marketed product and therefore make the participation in this study more desirable.

• REC name

  South Central - Oxford A Research Ethics Committee

• REC reference

  10/H0604/67

• Date of REC Opinion

  20 Sep 2010

• REC opinion

  Favourable Opinion