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

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