Safety of a Single Intravenous Dose of Recombinant Factor XIII in Children With Congenital FXIII A-subunit Deficiency

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

This trial is conducted in Europe and United States of America (USA). The aim of this clinical trial is to investigate the pharmacokinetics (at which rate the substance is distributed and eliminated from the body) and the safety profile of catridecacog (recombinant factor XIII (rFXIII)) in children with congenital FXIII A-subunit deficiency. Young children (1 to less than 6 years old) with congenital FXIII deficiency are evaluated.

Inclusion Criteria:

• Signed Informed Consent by subject's parents or subject's legally acceptable representative before any trial related activities. Trial related activities are any procedures that would not have been performed during the normal management of the subject
• Age 1 to less than 6 years old at the time of enrolment
• Congenital FXIII subunit-A deficiency previously documented by genotyping or evaluated by genotyping through blood sampling at screening visit
• Body weight at least 10 kg

Exclusion Criteria:

• Known antibodies to FXIII
• Hereditary or acquired coagulation disorder other than FXIII A-subunit congenital deficiency
• Platelet count (thrombocytes) of less than 50 × 10^9/L (at screening visit)
• Previous history of autoimmune disorder involving autoantibodies e.g., systemic lupus erythematosus
• Previous history of arterial or venous thromboembolic events e.g., cerebrovascular accident or deep vein thrombosis
• Known or suspected allergy to trial product or related products
• Any surgical procedure in the 30 days prior to enrolment and any planned surgery during the trial period
• Any disease or condition which, judged by the Investigator, could imply a potential hazard to the subject or interfere with the trial participation or trial outcome including renal and/or liver dysfunction