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| ID | Type | Description | Link |
|---|---|---|---|
| 1482 | Other Identifier | CSL Behring | |
| 2009-010722-19 | EudraCT Number |
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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| FXIII | Experimental | All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human). |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| FXIII Concentrate (Human) | Biological | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Up to week 52 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | 12 months |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Program Director, Clinical R&D | CSL Behring | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Study Site | Dothan | Alabama | 36305 | United States | ||
| Study Site |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 25377187 | Derived | Ashley C, Chang E, Davis J, Mangione A, Frame V, Nugent DJ. Efficacy and safety of prophylactic treatment with plasma-derived factor XIII concentrate (human) in patients with congenital factor XIII deficiency. Haemophilia. 2015 Jan;21(1):102-8. doi: 10.1111/hae.12524. Epub 2014 Nov 7. |
| Label | URL |
|---|---|
| Factor XIII Study BI71023\_2002 (NCT00883090) | View source |
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| ID | Title | Description |
|---|---|---|
| FG000 | FXIII | All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human). |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| 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. |
| 12 months |
| Peak FXIII Concentration at Steady State | At 12, 24, 36 and 48 weeks: at 30 and 60 minutes after the end of the infusion. |
| Trough FXIII Concentration at Steady State | At 12, 24, 36 and 48 weeks: immediately before infusion. |
| Time to Peak Concentration | 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. | 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. | At 12, 24, 36 and 48 weeks: immediately before infusion. |
| Oakland |
| California |
| 94610 |
| United States |
| Study Site | Orange | California | 92868 | United States |
| Study Site | San Francisco | California | 94115 | United States |
| Study Site | Stockton | California | 95204 | United States |
| Study Site | Hartford | Connecticut | 06106 | United States |
| Study Site | Fort Meyers | Florida | 33908 | United States |
| Study | Miami | Florida | 33136 | United States |
| Study Site | Boise | Idaho | 83712 | United States |
| Study Site | South Bend | Indiana | 46601 | United States |
| Study Site | Boston | Massachusetts | 02115 | United States |
| Study Site | Saint Paul | Minnesota | 55102 | United States |
| Study Site | Kansas City | Missouri | 64108 | United States |
| Study Site | Las Vegas | Nevada | 89015 | United States |
| Study Site | Lebanon | New Hampshire | 03756 | United States |
| Study Site | Newark | New Jersey | 07102 | United States |
| Study Site | Albany | New York | 12208 | United States |
| Study Site | New York | New York | 10021 | United States |
| Study Site | Chapel Hill | North Carolina | 27599 | United States |
| Study Site | Hershey | Pennsylvania | 17033 | United States |
| Study Site | Dallas | Texas | 75390 | United States |
| Study Site | Milwaukee | Wisconsin | 53233 | United States |
| Study Site | Santa Cruz de Tenerife | 38009 | Spain |
| Factor XIII Study BI71023\_3002 (NCT00945906) | View source |
| COMPLETED |
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| NOT COMPLETED |
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| ID | Title | Description |
|---|---|---|
| BG000 | FXIII | All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human). |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | |||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age Continuous | Mean | Standard Deviation | years |
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| Age, Customized | Number | participants |
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| Sex: Female, Male | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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. | The Efficacy Population comprised all subjects who received a dose of FXIII Concentrate (Human) during the study and included those who were assessed for efficacy at Baseline and had at least 1 follow-up FXIII activity trough level. | Posted | Number | participants | Up to week 52 |
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| Secondary | 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. | The analysis population comprised those subjects with spontaneous bleeding events requiring treatment with a FXIII-containing product. Note: no subjects had spontaneous bleeding events requiring treatment with a FXIII-containing product, so no subjects were analyzed. | Posted | Number | participants | 12 months |
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| Secondary | 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. | The Safety Population comprised all subjects who received a dose of FXIII Concentrate (Human) during the study. | Posted | Number | participants | 12 months |
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| Secondary | Peak FXIII Concentration at Steady State | The Pharmacokinetic (PK) Population comprised all subjects who received a dose of FXIII Concentrate (Human) during the study and included those who completed the study (defined as having sufficient bioanalytical assessments to calculate reliable estimates of the PK parameters). | Posted | Mean | Standard Deviation | Units/mL | At 12, 24, 36 and 48 weeks: at 30 and 60 minutes after the end of the infusion. |
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| Secondary | Trough FXIII Concentration at Steady State | The Pharmacokinetic (PK) Population comprised all subjects who received a dose of FXIII Concentrate (Human) during the study and included those who completed the study (defined as having sufficient bioanalytical assessments to calculate reliable estimates of the PK parameters). | Posted | Mean | Standard Deviation | Units/mL | At 12, 24, 36 and 48 weeks: immediately before infusion. |
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| Secondary | Time to Peak Concentration | The Pharmacokinetic (PK) Population comprised all subjects who received a dose of FXIII Concentrate (Human) during the study and included those who completed the study (defined as having sufficient bioanalytical assessments to calculate reliable estimates of the PK parameters). | Posted | Mean | Standard Deviation | Hour | At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion. |
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| Secondary | 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. | The Pharmacokinetic (PK) Population comprised all subjects who received a dose of FXIII Concentrate (Human) during the study and included those who completed the study (defined as having sufficient bioanalytical assessments to calculate reliable estimates of the PK parameters). | Posted | Mean | Standard Deviation | Units/mL/Units/kg | At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion. |
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| Secondary | 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. | The Efficacy Population comprised all subjects who received a dose of FXIII Concentrate (Human) during the study and included those who were assessed for efficacy at Baseline and had at least 1 follow-up FXIII activity trough level. | Posted | Number | participants | At 12, 24, 36 and 48 weeks: immediately before infusion. |
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12 months
The Safety Population comprised all subjects who received a dose of FXIII Concentrate (Human) during the study. Data presented for other, non-serious AEs are for treatment-emergent AEs.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | FXIII | All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human). | 4 | 41 | 27 | 41 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Appendicitis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
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| Urinary tract infection | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
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| Hip injury | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
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| Traumatic chest injury NOS | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
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| Chest pain with radiation to left arm | General disorders | MedDRA (12.0) | Systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Vomiting | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Fever | General disorders | MedDRA (12.0) | Systematic Assessment |
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| Abrasions | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
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| Fall | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
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| Bruising of thigh | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
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| Thrombin-antithrombin III complex increased | Investigations | MedDRA (12.0) | Systematic Assessment |
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| Wrist pain | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA (12.0) | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Rhinorrhea | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Sore throat | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Acne | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
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| Upper respiratory infection | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
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The investigator must provide a copy of any results communication to the sponsor for review at least 30 days prior to public release. The sponsor may request any changes necessary to prevent forfeiture of patent rights to data not in the public domain. For a multi-center study, the investigator must wait (i) at least 1 year after the study is completed at all sites or (ii) until notified by the sponsor that no multi-center publication is planned, before seeking publication review.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Trial Disclosure Manager | CSL Behring | Use email contact | clinicaltrials@cslbehring.com |
| ID | Term |
|---|---|
| D005177 | Factor XIII Deficiency |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ≥ 65 years |
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| Title | Denominators | Categories | ||||
|---|---|---|---|---|---|---|
| Week 12 (n = 40) |
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| Week 24 (n = 40) |
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| Week 36 (n = 41) |
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| Week 48 (n = 40) |
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| Title | Denominators | Categories | ||||
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| Week 12 (n = 40) |
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| Week 24 (n = 41) |
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| Week 36 (n = 41) |
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| Week 48 (n = 41) |
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