Phase 3
Completed N=63
Safety of Monthly Recombinant Factor XIII Replacement Therapy in Subjects With Congenital Factor XIII Deficiency: An Extension to Trial F13CD-1725
Congenital Bleeding Disorder · Congenital FXIII Deficiency
Source: ClinicalTrials.gov NCT00978380 ↗
Enrolled (actual)
63
Serious AEs
14.1%
Results posted
Dec 2016
Primary outcomePrimary: Adverse Events (AEs)(Serious and Non-serious) — 920; 19; 901 Events
◆ Published Evidence
Established
25citations · ~2 / year
Pharmacokinetics of recombinant factor XIII at steady state in patients with congenital factor XIII A-subunit deficiency.
Summary
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.
Linked Publications (3)
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Pharmacokinetics of recombinant factor XIII at steady state in patients with congenital factor XIII A-subunit deficiency.
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Pharmacokinetic characterization of recombinant factor XIII (FXIII)-A2 across age groups in patients with FXIII A-subunit congenital deficiency.
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Reciprocal stabilization of coagulation factor XIII-A and -B subunits is a determinant of plasma FXIII concentration.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Adverse Events (AEs)(Serious and Non-serious) |
920; 19; 901 | — |
| SECONDARY Antibody and Inhibitor Development |
— | — |
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT00978380) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.