A Phase 3 interventional study of FXIII Concentrate (Human) in Factor XIII Deficiency, sponsored by CSL Behring. Completed at 23 sites in 2 countries. Per ClinicalTrials.gov, last updated 2012-07-16.
Sponsored by CSL Behring · Phase 3 and Interventional
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.
CSL Behring is the lead sponsor of 142 studies on the registry; 18 are open to participants now.
Of its 24 completed or terminated interventional studies of FDA-regulated products, 17 (71%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
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.
Also known as: Fibrogammin-P
The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII-Containing Product to Treat the Bleeding Event)
The number of subjects requiring treatment with a Factor XIII-containing product to treat a spontaneous bleeding event.
Time frame: Up to week 52
Association of the Incidence of Spontaneous Bleeding Events Requiring Treatment and FXIII Activity Trough Levels
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.
Time frame: 12 months
Adverse Events
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.
Time frame: 12 months
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
Incremental recovery (U/mL/U/kg) is defined as maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of (U/kg) infusion.
Time frame: At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.
Achievement of Trough Factor XIII Levels of 5% or Higher.
Number of subjects with Factor XIII level ≥ 5% before infusion at Week 12, Week 24, Week 36 and Week 48.
Time frame: At 12, 24, 36 and 48 weeks: immediately before infusion.
| Milestone | FXIII |
|---|---|
| Started | 41 |
| Completed | 41 |
| Not completed | 0 |
The number of subjects requiring treatment with a Factor XIII-containing product to treat a spontaneous bleeding event.
| participants | FXIII |
|---|---|
| The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII-Containing Product to Treat the Bleeding Event) | 0 |
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.
No measurements were reported for this outcome.
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.
| participants | FXIII |
|---|---|
| Any treatment-emergent AEs | 33 |
| Any treatment-emergent and treatment-related AE | 3 |
| Any treatment-emergent SAE | 4 |
| Units/mL | FXIII |
|---|---|
| Week 12 (n = 40) | 0.968 ± 0.2292 |
| Week 24 (n = 40) | 1.045 ± 0.3825 |
| Week 36 (n = 41) | 0.962 ± 0.2306 |
| Week 48 (n = 40) | 0.983 ± 0.2633 |
| Units/mL | FXIII |
|---|---|
| Week 12 (n = 40) | 0.132 ± 0.0305 |
| Week 24 (n = 41) | 0.136 ± 0.0362 |
| Week 36 (n = 41) | 0.130 ± 0.0254 |
| Week 48 (n = 41) | 0.150 ± 0.1138 |
| Hour | FXIII |
|---|---|
| Week 12 (n = 40) | 0.632 ± 0.2296 |
| Week 24 (n = 40) | 0.615 ± 0.1978 |
| Week 36 (n = 39) | 0.623 ± 0.2350 |
| Week 48 (n = 40) | 0.636 ± 0.2328 |
Incremental recovery (U/mL/U/kg) is defined as maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of (U/kg) infusion.
| Units/mL/Units/kg | FXIII |
|---|---|
| Week 12 (n = 39) | 0.021 ± 0.0059 |
| Week 24 (n = 38) | 0.023 ± 0.0104 |
| Week 36 (n = 39) | 0.021 ± 0.0056 |
| Week 48 (n = 38) | 0.020 ± 0.0056 |
Number of subjects with Factor XIII level ≥ 5% before infusion at Week 12, Week 24, Week 36 and Week 48.
| participants | FXIII |
|---|---|
| Week 12 (n = 40) | 40 |
| Week 24 (n = 41) | 41 |
| Week 36 (n = 41) | 41 |
| Week 48 (n = 41) | 40 |
Collected over 12 months. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| FXIII | — | 4/41 (9.8%) | 27/41 (65.9%) |
| Event | FXIII |
|---|---|
| AppendicitisInfections and infestations | 1/41 |
| Urinary tract infectionInfections and infestations | 1/41 |
| Hip injuryInjury, poisoning and procedural complications | 1/41 |
| Traumatic chest injury NOSInjury, poisoning and procedural complications | 1/41 |
| Chest pain with radiation to left armGeneral disorders | 1/41 |
| Event | FXIII |
|---|---|
| Upper respiratory infectionInfections and infestations | 10/41 |
| FeverGeneral disorders | 5/41 |
| CoughRespiratory, thoracic and mediastinal disorders | 5/41 |
| FallInjury, poisoning and procedural complications | 4/41 |
| Nasal congestionRespiratory, thoracic and mediastinal disorders | 4/41 |
| VomitingGastrointestinal disorders | 3/41 |
| AbrasionsInjury, poisoning and procedural complications | 3/41 |
| Bruising of thighInjury, poisoning and procedural complications | 3/41 |
| Thrombin-antithrombin III complex increasedInvestigations | 3/41 |
| Wrist painMusculoskeletal and connective tissue disorders | 3/41 |
| Age Continuous(years) | FXIII |
|---|---|
| Mean | 20.1 ± 11.20 |
| Age, Customized(participants) | FXIII |
|---|---|
| < 16 years | 18 |
| 16 to < 65 years | 23 |
| ≥ 65 years | 0 |
| Sex: Female, Male(Participants) | FXIII |
|---|---|
| Female | 16 |
| Male | 25 |
This study is completed, as verified in Jun 2012. You cannot join it, but the record below documents what was studied.
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Factor XIII Deficiency
CSL Behring