Safety of Monthly Recombinant Factor XIII Replacement Therapy in Subjects With Congenital Factor XIII Deficiency: An Extension to Trial F13CD-1725

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

This trial is conducted in Asia, Europe and North America. The aim of the trial is to investigate the safety of monthly replacement therapy of recombinant factor XIII in patients with congenital FXIII deficiency. The trial continues until the product is commercially available, but an interim assessment will take place when all subjects have completed 52 weeks in the trial.

Inclusion Criteria:

• For subjects who participated in F13CD-1725:
• Previous participation (means up to and inclusive Visit 16, (End of Trial)) in F13CD-1725
• For all other subjects:
• Diagnosis of congenital FXIII A-subunit deficiency (confirmed by genotyping at screening visit or documented results from previously performed genotyping)
• Body weight at least 20 kg

Exclusion Criteria:

• Known neutralizing antibodies (inhibitors) towards FXIII
• Any known congenital or acquired coagulation disorder other than congenital FXIII deficiency
• Platelet count (thrombocytes) of less than 50 × 109/L. For subjects who participated in F13CD-1725 platelet count from visit 15 in F13CD-1725 must be used for evaluation.
• Females of childbearing potential who are pregnant, breastfeeding or are not using adequate contraceptive methods