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A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency

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ClinicalTrials.gov Identifier: NCT00885742
Recruitment Status : Completed
First Posted : April 22, 2009
Results First Posted : July 4, 2012
Last Update Posted : July 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 41
Recruitment Details  
Pre-assignment Details  
Arm/Group Title FXIII
Hide Arm/Group Description All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Period Title: Overall Study
Started 41
Completed 41
Not Completed 0
Arm/Group Title FXIII
Hide Arm/Group Description All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Overall Number of Baseline Participants 41
Hide Baseline Analysis Population Description
[Not Specified]
Age Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 41 participants
20.1  (11.20)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 41 participants
< 16 years 18
16 to < 65 years 23
≥ 65 years 0
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 41 participants
Female
16
  39.0%
Male
25
  61.0%
1.Primary Outcome
Title The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII‑Containing Product to Treat the Bleeding Event)
Hide Description The number of subjects requiring treatment with a Factor XIII-containing product to treat a spontaneous bleeding event.
Time Frame Up to week 52
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Overall Number of Participants Analyzed 41
Measure Type: Number
Unit of Measure: participants
0
2.Secondary Outcome
Title Association of the Incidence of Spontaneous Bleeding Events Requiring Treatment and FXIII Activity Trough Levels
Hide Description 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
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
3.Secondary 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 study treatment is related, or possibly related, and AEs with missing relationship.
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Population comprised all subjects who received a dose of FXIII Concentrate (Human) during the study.
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Overall Number of Participants Analyzed 41
Measure Type: Number
Unit of Measure: participants
Any treatment-emergent AEs 33
Any treatment-emergent and treatment-related AE 3
Any treatment-emergent SAE 4
4.Secondary Outcome
Title Peak FXIII Concentration at Steady State
Hide Description [Not Specified]
Time Frame At 12, 24, 36 and 48 weeks: at 30 and 60 minutes after the end of the infusion.
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Overall Number of Participants Analyzed 41
Mean (Standard Deviation)
Unit of Measure: Units/mL
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)
5.Secondary Outcome
Title Trough FXIII Concentration at Steady State
Hide Description [Not Specified]
Time Frame At 12, 24, 36 and 48 weeks: immediately before infusion.
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Overall Number of Participants Analyzed 41
Mean (Standard Deviation)
Unit of Measure: Units/mL
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)
6.Secondary Outcome
Title Time to Peak Concentration
Hide Description [Not Specified]
Time Frame At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Overall Number of Participants Analyzed 41
Mean (Standard Deviation)
Unit of Measure: Hour
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)
7.Secondary Outcome
Title Incremental Recovery
Hide Description 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.
Hide Outcome Measure Data
Hide Analysis Population Description
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).
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Overall Number of Participants Analyzed 41
Mean (Standard Deviation)
Unit of Measure: Units/mL/Units/kg
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)
8.Secondary Outcome
Title Achievement of Trough Factor XIII Levels of 5% or Higher.
Hide Description 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.
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title FXIII
Hide Arm/Group Description:
All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Overall Number of Participants Analyzed 41
Measure Type: Number
Unit of Measure: participants
Week 12 (n = 40) 40
Week 24 (n = 41) 41
Week 36 (n = 41) 41
Week 48 (n = 41) 40
Time Frame 12 months
Adverse Event Reporting Description 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.
 
Arm/Group Title FXIII
Hide Arm/Group Description All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
All-Cause Mortality
FXIII
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
FXIII
Affected / at Risk (%) # Events
Total   4/41 (9.76%)    
General disorders   
Chest pain with radiation to left arm  1  1/41 (2.44%)  1
Infections and infestations   
Appendicitis  1  1/41 (2.44%)  1
Urinary tract infection  1  1/41 (2.44%)  1
Injury, poisoning and procedural complications   
Hip injury  1  1/41 (2.44%)  1
Traumatic chest injury NOS  1  1/41 (2.44%)  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 5%
FXIII
Affected / at Risk (%) # Events
Total   27/41 (65.85%)    
Gastrointestinal disorders   
Vomiting  1  3/41 (7.32%)  6
General disorders   
Fever  1  5/41 (12.20%)  5
Infections and infestations   
Upper respiratory infection  1  10/41 (24.39%)  11
Injury, poisoning and procedural complications   
Abrasions  1  3/41 (7.32%)  5
Fall  1  4/41 (9.76%)  4
Bruising of thigh  1  3/41 (7.32%)  3
Investigations   
Thrombin-antithrombin III complex increased  1  3/41 (7.32%)  5
Musculoskeletal and connective tissue disorders   
Wrist pain  1  3/41 (7.32%)  3
Nervous system disorders   
Headache  1  3/41 (7.32%)  10
Respiratory, thoracic and mediastinal disorders   
Cough  1  5/41 (12.20%)  6
Nasal congestion  1  4/41 (9.76%)  4
Rhinorrhea  1  3/41 (7.32%)  4
Sore throat  1  3/41 (7.32%)  3
Skin and subcutaneous tissue disorders   
Acne  1  3/41 (7.32%)  3
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: NCT00885742     History of Changes
Other Study ID Numbers: BI71023_3001
1482 ( Other Identifier: CSL Behring )
2009-010722-19 ( EudraCT Number )
First Submitted: April 21, 2009
First Posted: April 22, 2009
Results First Submitted: April 30, 2012
Results First Posted: July 4, 2012
Last Update Posted: July 16, 2012