A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Congenital deficiency of factor XIII (FXIII) is an extremely rare inherited disorder associated with potentially life-threatening bleeding. Factor XIII Concentrate is given to patients whose blood is lacking factor XIII. Factor XIII Concentrate works by assisting blood in the usual clotting process, thereby preventing bleeding.
In this study, patients will be treated with FXIII Concentrate (Human) and followed closely to determine that they receive the dose that will best minimize the chance of bruising and bleeding.
|Study Design:||Intervention Model: Single Group Assignment
Masking: Open Label
|Official Title:||A Prospective, Multicenter, Open-label, Phase 3b Study of Human Plasma-Derived Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency|
- The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII‑Containing Product to Treat the Bleeding Event) [ Time Frame: Up to week 52 ]The number of subjects requiring treatment with a Factor XIII-containing product to treat a spontaneous bleeding event.
- Association of the Incidence of Spontaneous Bleeding Events Requiring Treatment and FXIII Activity Trough Levels [ Time Frame: 12 months ]P-value determined from Generalized Estimating Equation (GEE) model parameter estimates with bleeding as the response variable and FXIII activity trough level as the explanatory variable.
- Adverse Events [ Time Frame: 12 months ]Number of subjects with any treatment-emergent adverse event (AE), treatment-related AE or serious AE (SAE). Treatment related AEs are defined as AEs whose relationship to study treatment is related, or possibly related, and AEs with missing relationship.
- Peak FXIII Concentration at Steady State [ Time Frame: At 12, 24, 36 and 48 weeks: at 30 and 60 minutes after the end of the infusion. ]
- Trough FXIII Concentration at Steady State [ Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion. ]
- Time to Peak Concentration [ Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion. ]
- Incremental Recovery [ Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion. ]Incremental recovery (U/mL/U/kg) is defined as maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of (U/kg) infusion.
- Achievement of Trough Factor XIII Levels of 5% or Higher. [ Time Frame: At 12, 24, 36 and 48 weeks: immediately before infusion. ]Number of subjects with Factor XIII level ≥ 5% before infusion at Week 12, Week 24, Week 36 and Week 48.
|Study Start Date:||August 2009|
|Study Completion Date:||April 2011|
|Primary Completion Date:||April 2011 (Final data collection date for primary outcome measure)|
All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Biological: FXIII Concentrate (Human)
Doses will be guided by the individual subject's most recent FXIII activity levels, with the objective of dosing every 28 days to maintain a trough FXIII activity level of approximately 5 to 20%.
Subjects enrolled in this study who did not complete the pharmacokinetic study (Factor XIII Study BI71023_2002 [NCT00883090]) will initially receive FXIII Concentrate (Human) at a dose of 40 U/kg by intravenous (IV) infusion.
Other Name: Fibrogammin-P
Please refer to this study by its ClinicalTrials.gov identifier: NCT00885742
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|Study Director:||Program Director, Clinical R&D||CSL Behring|