Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII 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: NCT00945906
Recruitment Status : Completed
First Posted : July 24, 2009
Results First Posted : October 12, 2012
Last Update Posted : October 12, 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) (FXIII)
Enrollment 61
Recruitment Details  
Pre-assignment Details  
Arm/Group Title FXIII
Hide Arm/Group Description Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%.
Period Title: Overall Study
Started 61
Completed 54
Not Completed 7
Reason Not Completed
Withdrawal by Subject             1
Did not want to return for last visit             2
Unable to return for last visit             2
Moved to another country             2
Arm/Group Title FXIII
Hide Arm/Group Description Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%.
Overall Number of Baseline Participants 61
Hide Baseline Analysis Population Description
[Not Specified]
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 61 participants
18.5  (12.48)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 61 participants
< 16 years 29
16 to < 65 years 32
>=65 years 0
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 61 participants
Female
26
  42.6%
Male
35
  57.4%
1.Primary Outcome
Title Adverse Events
Hide Description 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 treatment is related, or possibly related and AEs with missing relationship.
Time Frame After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study. Treatment related AEs are events whose relationship to study treatment is related, or possibly related, in the opinion of the investigator. AEs with missing relationship are considered related to treatment.
Arm/Group Title FXIII
Hide Arm/Group Description:
Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%.
Overall Number of Participants Analyzed 61
Measure Type: Number
Unit of Measure: participants
Any treatment-emergent AE 42
Treatment-emergent and related AE 2
Serious AE 2
2.Secondary Outcome
Title Hematology and Chemistry Testing
Hide Description Number of participants with treatment-emergent clinically significant hematology and/or chemistry laboratory parameter values.
Time Frame After the first infusion and at the end-of-study (or withdrawal) visit.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
Arm/Group Title FXIII
Hide Arm/Group Description:
Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%.
Overall Number of Participants Analyzed 61
Measure Type: Number
Unit of Measure: participants
Clinically significant hematology test result 1
Clinically significant chemistry test result 1
3.Secondary Outcome
Title FXIII Antibody Testing
Hide Description Number of participants with serum Factor XIII antibodies.
Time Frame Before the first infusion, then every 48 weeks, at the end-of-study (or withdrawal) visit and after a bleeding episode requiring treatment with a Factor XIII -containing product.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
Arm/Group Title FXIII
Hide Arm/Group Description:
Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%.
Overall Number of Participants Analyzed 61
Measure Type: Number
Unit of Measure: participants
Subjects with Factor XIII antibodies 1
Subjects without Factor XIII antibodies 60
4.Secondary Outcome
Title FXIII Concentration
Hide Description Trough Factor XIII concentration.
Time Frame Before the first infusion, at 24 and 48 weeks after the first infusion, and at the end-of-study (or withdrawal) visit.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
Arm/Group Title FXIII
Hide Arm/Group Description:
Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%.
Overall Number of Participants Analyzed 61
Mean (Standard Deviation)
Unit of Measure: Units/mL
Baseline (n = 35) 0.0987  (0.03695)
Week 4 (n = 28) 0.1177  (0.03484)
Week 8 (n = 8) 0.1238  (0.02973)
Week 12 (n = 8) 0.1075  (0.03196)
Week 16 (n = 4) 0.1025  (0.04573)
Week 20 (n = 5) 0.1160  (0.03578)
Week 24 (n = 41) 0.1341  (0.03346)
Week 28 (n = 3) 0.1667  (0.06658)
Week 32 (n = 2) 0.0950  (0.02121)
Week 36 (n = 3) 0.1083  (0.07489)
Week 40 (n = 3) 0.0933  (0.04509)
Week 44 (n = 1) 0.0500  (0)
Week 48 (n = 13) 0.1246  (0.02961)
Week 72 (n = 2) 0.1400  (0.01414)
5.Secondary Outcome
Title Number of Subjects With at Least One Bleeding Episode
Hide Description Number of subjects with at least one bleeding episode at any time after the first infusion in the study, and the number of subjects with at least one bleeding episode requiring Factor XIII treatment.
Time Frame After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
Arm/Group Title FXIII
Hide Arm/Group Description:
Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%.
Overall Number of Participants Analyzed 61
Measure Type: Number
Unit of Measure: participants
At least one bleeding episode (after treatment) 10
At least one bleeding episode requiring treatment 1
6.Secondary Outcome
Title Number of Bleeding Episodes
Hide Description Number of bleeding episodes at any time after the first infusion in the study.
Time Frame After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
Arm/Group Title FXIII
Hide Arm/Group Description:
Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%.
Overall Number of Participants Analyzed 61
Measure Type: Number
Unit of Measure: Episodes
14
Time Frame After the first infusion until study completion. Study completion is up to 2 years or until Factor XIII Concentrate (Human) is commercially available in the USA.
Adverse Event Reporting Description The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
 
Arm/Group Title FXIII
Hide Arm/Group Description Subjects were administered FXIII Concentrate (Human) by intravenous (IV) infusion approximately every 28 days to maintain a trough FXIII level of approximately 5 to 20%.
All-Cause Mortality
FXIII
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
FXIII
Affected / at Risk (%) # Events
Total   2/61 (3.28%)    
Blood and lymphatic system disorders   
Factor XIII inhibition  1  1/61 (1.64%)  1
General disorders   
Fever  1  1/61 (1.64%)  1
Infections and infestations   
Pelvic inflammatory disease  1  1/61 (1.64%)  1
Pyelonephritis  1  1/61 (1.64%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (12.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 4%
FXIII
Affected / at Risk (%) # Events
Total   19/61 (31.15%)    
Gastrointestinal disorders   
Vomiting  1  3/61 (4.92%)  4
General disorders   
Fever  1  6/61 (9.84%)  7
Infections and infestations   
Upper respiratory infection  1  4/61 (6.56%)  5
Injury, poisoning and procedural complications   
Bruising  1  4/61 (6.56%)  5
Nervous system disorders   
Headache  1  4/61 (6.56%)  4
Respiratory, thoracic and mediastinal disorders   
Cough  1  5/61 (8.20%)  5
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.
CSL agreements and restrictions on publishing may vary with individual investigators; however, CSL will not prohibit any investigator from publishing. CSL supports the publication of results from all centers of a multi-center trial and generally requires that reports based on single-site data not precede the primary publication of the entire clinical trial.
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: NCT00945906     History of Changes
Other Study ID Numbers: BI71023_3002
1488 ( Other Identifier: CSL Behring )
First Submitted: July 23, 2009
First Posted: July 24, 2009
Results First Submitted: September 12, 2012
Results First Posted: October 12, 2012
Last Update Posted: October 12, 2012