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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Congenital Bleeding Disorder, Congenital FXIII Deficiency

Intervention / Treatment
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* Drug: catridecacog


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 6 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: A<br> | Drug: catridecacog<br>* Monthly administration of recombinant factor XIII as preventative treatment of bleeding episodes. Dose: 35 IU/kg body weight intravenous (into the vein)<br>* Other names: recombinant factor XIII;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse Events (AEs)(Serious and Non-serious) | An AE was defined as any unfavourable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Trial AEs (serious) included any event such as death, life-threatening experience, in-subject hospitalisation, significant disability/ congential anomaly experienced from the trial product. | All AEs were collected and reported from screening (week 0) for a minimum period of 52 weeks or until the end of trial visit. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Antibody and Inhibitor Development | All subjects who received rFXIII were monitored for anti-rFXIII antibodies and inhibitor development. Samples passed through 2 tiers of ELISA testing: an initial screen with a specific cut-off point (including  5% false positives) and a second confirmatory assay for samples yielding a result above the screening cut-off point. If samples were confirmed as antibody positive in the confirmation assay, an inhibitor assay was also carried out to detect functional inhibitors. Percentage of subjects with antibody and inhibitor development were reported. | From week 0 to week 52 |