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| ID | Type | Description | Link |
|---|---|---|---|
| 2009-010387-41 | EudraCT Number | ||
| 1479 | Other Identifier | CSL Behring |
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Congenital deficiency of Factor XIII is an extremely rare hereditary disorder associated with potentially life-threatening bleeding. This study will evaluate the safety and recommended (best) amount or level of Factor XIII in a patient's blood. Factor XIII Concentrate (Human) is given to people whose blood is lacking Factor XIII. Factor XIII Concentrate (Human) works by assisting your blood in the usual clotting process, thereby preventing bleeding.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| FXIII | Experimental | All subjects treated with Factor XIII Concentrate (Human) (FXIII) |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| FXIII Concentrate (Human) | Biological | Subjects will receive approximately 40 U/kg of FXIII every 28 days for 3 doses administered as a bolus intravenous (IV) injection at approximately 250 U/minute. |
| Measure | Description | Time Frame |
|---|---|---|
| Peak FXIII Concentration at Steady State | 12 weeks | |
| Trough FXIII Concentration at Steady State | 12 weeks | |
| Time to Peak Concentration | 12 weeks | |
| Incremental Recovery | Incremental recovery (U/mL/U/kg) is defined as the maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of FXIII (U/kg) administered. | 12 weeks |
| Terminal Half-life | 12 weeks | |
| Area Under the Curve at Steady State | 12 weeks | |
| Clearance | 12 weeks | |
| Volume of Distribution at Steady State | 12 weeks | |
| Mean Residence Time | 12 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Adverse Events | Number of participants with an adverse event | 16 weeks |
| Laboratory Safety Parameters | Number of participants with clinically significant laboratory safety parameter values. The laboratory safety parameters measured included serum chemistries, hematology and urinalysis. |
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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 |
|---|---|---|---|
| 25458735 | Derived | Nugent DJ, Ashley C, Garcia-Talavera J, Lo LC, Mehdi AS, Mangione A. Pharmacokinetics and safety of plasma-derived factor XIII concentrate (human) in patients with congenital factor XIII deficiency. Haemophilia. 2015 Jan;21(1):95-101. doi: 10.1111/hae.12505. Epub 2014 Dec 2. |
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One subject was not administered FXIII because of the Sponsor's decision. This subject was not included in the analyses.
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| ID | Title | Description |
|---|---|---|
| FG000 | FXIII | All subjects treated with Factor XIII Concentrate (Human) (FXIII) |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | FXIII | All subjects treated with Factor FXIII Concentrate (Human) (FXIII) |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age Continuous | Mean |
| 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 | Peak FXIII Concentration at Steady State | The analysis population was the pharmacokinetic (PK) population. The PK population comprised all subjects in the safety population who completed the study (defined as having sufficient bioanalytical assessments to calculate reliable estimates of the PK parameters specified). | Posted | Mean | Standard Deviation | Units/mL | 12 weeks |
|
|
The time frame for adverse event (AE) reporting was up to 16 weeks and comprised the time from giving written informed consent (during screening) to 28 days after the last administration of study treatment.
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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 treated with Factor FXIII Concentrate (Human) (FXIII) |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Acute bronchitis | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Clinical Trial Disclosure Manager | CSL Behring | Use email contact | clinicaltrials@cslbehring.com |
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| 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 |
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|
| 16 weeks |
| Vital Signs | Number of participants with clinically significant vital signs. The vital signs measured included blood pressure, pulse rate and temperature. Clinically significant changes in vital signs were to be reported as adverse events. | 16 weeks |
| Orange |
| California |
| 92868 |
| United States |
| Study Site | San Francisco | California | 94115 | United States |
| Study Site | Stockton | California | 95204 | United States |
| Study Site | Boston | Massachusetts | 02115 | United States |
| Study Site | Santa Cruz de Tenerife | Spain |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
|
| Primary | Trough FXIII Concentration at Steady State | The analysis population was the PK population. The PK population comprised all subjects in the safety population who completed the study (defined as having sufficient bioanalytical assessments to calculate reliable estimates of the PK parameters specified). | Posted | Mean | Standard Deviation | Units/mL | 12 weeks |
|
|
|
| Primary | Time to Peak Concentration | The analysis population was the PK population. The PK population comprised all subjects in the safety population who completed the study (defined as having sufficient bioanalytical assessments to calculate reliable estimates of the PK parameters specified). | Posted | Mean | Standard Deviation | hr | 12 weeks |
|
|
|
| Primary | Incremental Recovery | Incremental recovery (U/mL/U/kg) is defined as the maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of FXIII (U/kg) administered. | The analysis population was the PK population. The PK population comprised all subjects in the safety population who completed the study (defined as having sufficient bioanalytical assessments to calculate reliable estimates of the PK parameters specified). | Posted | Mean | Standard Deviation | Units/mL/Units/kg | 12 weeks |
|
|
|
| Primary | Terminal Half-life | The analysis population was the PK population. The PK population comprised all subjects in the safety population who completed the study (defined as having sufficient bioanalytical assessments to calculate reliable estimates of the PK parameters specified). | Posted | Mean | Standard Deviation | days | 12 weeks |
|
|
|
| Primary | Area Under the Curve at Steady State | The analysis population was the PK population. The PK population comprised all subjects in the safety population who completed the study (defined as having sufficient bioanalytical assessments to calculate reliable estimates of the PK parameters specified). | Posted | Mean | Standard Deviation | Units*hr/mL | 12 weeks |
|
|
|
| Primary | Clearance | The analysis population was the PK population. The PK population comprised all subjects in the safety population who completed the study (defined as having sufficient bioanalytical assessments to calculate reliable estimates of the PK parameters specified). | Posted | Mean | Standard Deviation | mL/hr/kg | 12 weeks |
|
|
|
| Primary | Volume of Distribution at Steady State | The analysis population was the PK population. The PK population comprised all subjects in the safety population who completed the study (defined as having sufficient bioanalytical assessments to calculate reliable estimates of the PK parameters specified). | Posted | Mean | Standard Deviation | mL/kg | 12 weeks |
|
|
|
| Primary | Mean Residence Time | The analysis population was the PK population. The PK population comprised all subjects in the safety population who completed the study (defined as having sufficient bioanalytical assessments to calculate reliable estimates of the PK parameters specified). | Posted | Mean | Standard Deviation | days | 12 weeks |
|
|
|
| Secondary | Adverse Events | Number of participants with an adverse event | The analysis population was the safety population. The safety population comprised all subjects who received a dose of Factor XIII. | Posted | Number | participants | 16 weeks |
|
|
|
| Secondary | Laboratory Safety Parameters | Number of participants with clinically significant laboratory safety parameter values. The laboratory safety parameters measured included serum chemistries, hematology and urinalysis. | The analysis population was the safety population. The safety population comprised all subjects who received a dose of Factor XIII. | Posted | Number | participants | 16 weeks |
|
|
|
| Secondary | Vital Signs | Number of participants with clinically significant vital signs. The vital signs measured included blood pressure, pulse rate and temperature. Clinically significant changes in vital signs were to be reported as adverse events. | The analysis population was the safety population. The safety population comprised all subjects who received a dose of Factor XIII. | Posted | Number | participants | 16 weeks |
|
|
|
| 0 |
| 14 |
| 8 |
| 14 |
| Flu | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
|
| Infected sebaceous cyst | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
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| Tinea corporis | 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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| Ankle injury | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
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| Bruising of arm | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
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| Contusion of knee | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
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| Contusion of toe | Injury, poisoning and procedural complications | MedDRA (12.0) | Systematic Assessment |
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| Fibrin D dimer increased | Investigations | MedDRA (12.0) | Systematic Assessment |
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| Prothrombin increased | Investigations | MedDRA (12.0) | Systematic Assessment |
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| Thrombin-antithrombin III complex increased | Investigations | MedDRA (12.0) | Systematic Assessment |
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| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
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| Borderline diabetes | Metabolism and nutrition disorders | MedDRA (12.0) | Systematic Assessment |
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| Penile adhesion | Reproductive system and breast disorders | 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.
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |