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A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00883090
Recruitment Status : Completed
First Posted : April 17, 2009
Results First Posted : January 16, 2012
Last Update Posted : January 16, 2012
Sponsor:
Information provided by (Responsible Party):
CSL Behring

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label)
Condition Factor XIII Deficiency
Intervention Biological: FXIII Concentrate (Human)
Enrollment 15
Recruitment Details  
Pre-assignment Details One subject was not administered FXIII because of the Sponsor's decision. This subject was not included in the analyses.
Arm/Group Title FXIII
Hide Arm/Group Description All subjects treated with Factor XIII Concentrate (Human) (FXIII)
Period Title: Overall Study
Started 15 [1]
Completed 13
Not Completed 2
Reason Not Completed
Sponsor's decision             1
Withdrawal by Subject             1
[1]
Of the 15 participants started, 14 were administered FXIII
Arm/Group Title FXIII
Hide Arm/Group Description All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Overall Number of Baseline Participants 14
Hide Baseline Analysis Population Description
[Not Specified]
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 14 participants
24.0  (12.55)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 14 participants
Female
7
  50.0%
Male
7
  50.0%
1.Primary Outcome
Title Peak FXIII Concentration at Steady State
Hide Description [Not Specified]
Time Frame 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: Units/mL
0.9  (0.20)
2.Primary Outcome
Title Trough FXIII Concentration at Steady State
Hide Description [Not Specified]
Time Frame 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: Units/mL
0.05  (0.05)
3.Primary Outcome
Title Time to Peak Concentration
Hide Description [Not Specified]
Time Frame 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: hr
1.7  (1.44)
4.Primary Outcome
Title Incremental Recovery
Hide Description 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.
Time Frame 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: Units/mL/Units/kg
0.02  (0.01)
5.Primary Outcome
Title Terminal Half-life
Hide Description [Not Specified]
Time Frame 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: days
6.6  (2.29)
6.Primary Outcome
Title Area Under the Curve at Steady State
Hide Description [Not Specified]
Time Frame 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: Units*hr/mL
184.0  (65.78)
7.Primary Outcome
Title Clearance
Hide Description [Not Specified]
Time Frame 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: mL/hr/kg
0.25  (0.09)
8.Primary Outcome
Title Volume of Distribution at Steady State
Hide Description [Not Specified]
Time Frame 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: mL/kg
51.1  (12.61)
9.Primary Outcome
Title Mean Residence Time
Hide Description [Not Specified]
Time Frame 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Overall Number of Participants Analyzed 13
Mean (Standard Deviation)
Unit of Measure: days
10.0  (3.45)
10.Secondary Outcome
Title Adverse Events
Hide Description Number of participants with an adverse event
Time Frame 16 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population was the safety population. The safety population comprised all subjects who received a dose of Factor XIII.
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Overall Number of Participants Analyzed 14
Measure Type: Number
Unit of Measure: participants
8
11.Secondary Outcome
Title Laboratory Safety Parameters
Hide Description Number of participants with clinically significant laboratory safety parameter values. The laboratory safety parameters measured included serum chemistries, hematology and urinalysis.
Time Frame 16 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population was the safety population. The safety population comprised all subjects who received a dose of Factor XIII.
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Overall Number of Participants Analyzed 14
Measure Type: Number
Unit of Measure: participants
0
12.Secondary Outcome
Title Vital Signs
Hide Description 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.
Time Frame 16 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The analysis population was the safety population. The safety population comprised all subjects who received a dose of Factor XIII.
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Overall Number of Participants Analyzed 14
Measure Type: Number
Unit of Measure: participants
0
Time Frame 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.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title FXIII
Hide Arm/Group Description All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
All-Cause Mortality
FXIII
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
FXIII
Affected / at Risk (%) # Events
Total   0/14 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
FXIII
Affected / at Risk (%) # Events
Total   8/14 (57.14%)    
Infections and infestations   
Acute bronchitis  1  2/14 (14.29%)  2
Flu  1  1/14 (7.14%)  1
Infected sebaceous cyst  1  1/14 (7.14%)  1
Tinea corporis  1  1/14 (7.14%)  1
Urinary tract infection  1  1/14 (7.14%)  1
Injury, poisoning and procedural complications   
Ankle injury  1  1/14 (7.14%)  1
Bruising of arm  1  1/14 (7.14%)  1
Contusion of knee  1  1/14 (7.14%)  1
Contusion of toe  1  1/14 (7.14%)  1
Investigations   
Fibrin D dimer increased  1  1/14 (7.14%)  1
Prothrombin increased  1  1/14 (7.14%)  1
Thrombin-antithrombin III complex increased  1  1/14 (7.14%)  1
Metabolism and nutrition disorders   
Borderline diabetes  1  1/14 (7.14%)  1
Reproductive system and breast disorders   
Penile adhesion  1  1/14 (7.14%)  1
Skin and subcutaneous tissue disorders   
Ecchymosis  1  1/14 (7.14%)  1
Rash  1  1/14 (7.14%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (12.0)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Clinical Trial Disclosure Manager
Organization: CSL Behring
Phone: Use email contact
EMail: clinicaltrials@cslbehring.com
Layout table for additonal information
Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT00883090     History of Changes
Other Study ID Numbers: BI71023_2002
2009-010387-41 ( EudraCT Number )
1479 ( Other Identifier: CSL Behring )
First Submitted: April 16, 2009
First Posted: April 17, 2009
Results First Submitted: December 7, 2011
Results First Posted: January 16, 2012
Last Update Posted: January 16, 2012