Evaluation of Recombinant Factor XIII for Prevention of Bleeding in Patients With FXIII Inherited Deficiency

A Multi-Centre, Open-Label, Single-Arm and Multiple Dosing Trial on Efficacy and Safety of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Subjects With Congenital Factor XIII Deficiency

The trial is conducted in Europe, North America and Asia. The aim of this trial is to evaluate catridecacog (recombinant factor XIII (rFXIII)) treatment in patients with inherited FXIII deficiency. It is expected that recombinant FXIII can be used for the prevention of bleeding episodes.

Inclusion Criteria:

• Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit)
• Treatment with regular FXIII replacement therapy initiated at least 6 months prior to screening and one of the following : a documented history of at least one 1 treatment-requiring bleeding episode prior to initiation of regular replacement therapy or a documented family history of FXIII congenital deficiency (only for subjects on regular replacement therapy prior to screening)
• Documented history of at least two 2 bleeding episodes requiring treatment with FXIII containing blood products within the last 12 months prior to screening (only for subjects receiving on-demand treatment prior to screening)

Exclusion Criteria:

• Known neutralizing antibodies (inhibitors) towards FXIII
• Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
• Documented history of at least 2 treatment-requiring bleeding episodes per year during previous regular replacement therapy with FXIII containing blood products (fresh frozen plasma (FFP), plasma-derived FXIII (pd FXIII) and cryoprecipitate)
• Known or suspected allergy to trial product(s) or related products
• Planned major surgery during the trial period. Catheter, ports and dental extractions do not count as surgeries and will not exclude the subject
• Renal insufficiency defined as current dialysis therapy
• Any history of confirmed venous or arterial thrombo-embolic events