Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency

A Multi-Centre, Multinational, Open-Label, Single-Arm and Multiple Dosing Trial on Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency. Safety Extension Trial to F13CD-3760

This trial will be conducted in Asia, Europe and the United States of America (USA).

The aim of this clinical trial is to investigate long-term safety of rFXIII when administered for prevention of bleeding episodes in children aged between 1 and 6 years with congenital FXIII A-subunit deficiency. This trial is an extension to trial F13CD-3760 (mentor™4, NCT01230021). If applicable the trial will be extended up to maximum 3 years dependent on when recombinant factor XIII will be commercially available in subject's respective country for use in children of 1-6 years of age.

Inclusion Criteria:

• Completed participation in trial F13CD-3760 (NCT01230021)

Exclusion Criteria:

• Known or suspected hypersensitivity to trial product or related products
• Known history of development of inhibitors against FXIII (factor XIII)
• Hereditary or acquired coagulation disorder other than FXIII congenital deficiency
• Platelet count (thrombocytes) less than 50X10e9 / L
• 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
• Any disease or condition which, judged by the trial physician, could imply a potential hazard to the subject, interfere with the trial participation or trial outcome including renal and/or liver dysfunction