Trial Outcomes & Findings for An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency (NCT NCT00945906)
NCT ID: NCT00945906
Last Updated: 2012-10-12
Results Overview
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.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
61 participants
Primary outcome timeframe
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.
Results posted on
2012-10-12
Participant Flow
Participant milestones
| Measure |
FXIII
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 Study
STARTED
|
61
|
|
Overall Study
COMPLETED
|
54
|
|
Overall Study
NOT COMPLETED
|
7
|
Reasons for withdrawal
| Measure |
FXIII
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 Study
Withdrawal by Subject
|
1
|
|
Overall Study
Did not want to return for last visit
|
2
|
|
Overall Study
Unable to return for last visit
|
2
|
|
Overall Study
Moved to another country
|
2
|
Baseline Characteristics
An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Baseline characteristics by cohort
| Measure |
FXIII
n=61 Participants
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%.
|
|---|---|
|
Age Continuous
|
18.5 years
STANDARD_DEVIATION 12.48 • n=5 Participants
|
|
Age, Customized
< 16 years
|
29 participants
n=5 Participants
|
|
Age, Customized
16 to < 65 years
|
32 participants
n=5 Participants
|
|
Age, Customized
>=65 years
|
0 participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
26 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
35 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 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.Population: 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.
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.
Outcome measures
| Measure |
FXIII
n=61 Participants
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%.
|
|---|---|
|
Adverse Events
Any treatment-emergent AE
|
42 participants
|
|
Adverse Events
Treatment-emergent and related AE
|
2 participants
|
|
Adverse Events
Serious AE
|
2 participants
|
SECONDARY outcome
Timeframe: After the first infusion and at the end-of-study (or withdrawal) visit.Population: The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
Number of participants with treatment-emergent clinically significant hematology and/or chemistry laboratory parameter values.
Outcome measures
| Measure |
FXIII
n=61 Participants
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%.
|
|---|---|
|
Hematology and Chemistry Testing
Clinically significant hematology test result
|
1 participants
|
|
Hematology and Chemistry Testing
Clinically significant chemistry test result
|
1 participants
|
SECONDARY outcome
Timeframe: 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.Population: The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
Number of participants with serum Factor XIII antibodies.
Outcome measures
| Measure |
FXIII
n=61 Participants
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%.
|
|---|---|
|
FXIII Antibody Testing
Subjects with Factor XIII antibodies
|
1 participants
|
|
FXIII Antibody Testing
Subjects without Factor XIII antibodies
|
60 participants
|
SECONDARY outcome
Timeframe: Before the first infusion, at 24 and 48 weeks after the first infusion, and at the end-of-study (or withdrawal) visit.Population: The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
Trough Factor XIII concentration.
Outcome measures
| Measure |
FXIII
n=61 Participants
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%.
|
|---|---|
|
FXIII Concentration
Week 48 (n = 13)
|
0.1246 Units/mL
Standard Deviation 0.02961
|
|
FXIII Concentration
Week 72 (n = 2)
|
0.1400 Units/mL
Standard Deviation 0.01414
|
|
FXIII Concentration
Baseline (n = 35)
|
0.0987 Units/mL
Standard Deviation 0.03695
|
|
FXIII Concentration
Week 4 (n = 28)
|
0.1177 Units/mL
Standard Deviation 0.03484
|
|
FXIII Concentration
Week 8 (n = 8)
|
0.1238 Units/mL
Standard Deviation 0.02973
|
|
FXIII Concentration
Week 12 (n = 8)
|
0.1075 Units/mL
Standard Deviation 0.03196
|
|
FXIII Concentration
Week 16 (n = 4)
|
0.1025 Units/mL
Standard Deviation 0.04573
|
|
FXIII Concentration
Week 20 (n = 5)
|
0.1160 Units/mL
Standard Deviation 0.03578
|
|
FXIII Concentration
Week 24 (n = 41)
|
0.1341 Units/mL
Standard Deviation 0.03346
|
|
FXIII Concentration
Week 28 (n = 3)
|
0.1667 Units/mL
Standard Deviation 0.06658
|
|
FXIII Concentration
Week 32 (n = 2)
|
0.0950 Units/mL
Standard Deviation 0.02121
|
|
FXIII Concentration
Week 36 (n = 3)
|
0.1083 Units/mL
Standard Deviation 0.07489
|
|
FXIII Concentration
Week 40 (n = 3)
|
0.0933 Units/mL
Standard Deviation 0.04509
|
|
FXIII Concentration
Week 44 (n = 1)
|
0.0500 Units/mL
Standard Deviation 0
|
SECONDARY outcome
Timeframe: 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.Population: The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
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.
Outcome measures
| Measure |
FXIII
n=61 Participants
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%.
|
|---|---|
|
Number of Subjects With at Least One Bleeding Episode
At least one bleeding episode (after treatment)
|
10 participants
|
|
Number of Subjects With at Least One Bleeding Episode
At least one bleeding episode requiring treatment
|
1 participants
|
SECONDARY outcome
Timeframe: 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.Population: The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
Number of bleeding episodes at any time after the first infusion in the study.
Outcome measures
| Measure |
FXIII
n=61 Participants
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%.
|
|---|---|
|
Number of Bleeding Episodes
|
14 Episodes
|
Adverse Events
FXIII
Serious events: 2 serious events
Other events: 19 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
FXIII
n=61 participants at risk
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%.
|
|---|---|
|
Blood and lymphatic system disorders
Factor XIII inhibition
|
1.6%
1/61 • Number of events 1 • 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.
The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
|
|
Infections and infestations
Pelvic inflammatory disease
|
1.6%
1/61 • Number of events 1 • 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.
The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
|
|
Infections and infestations
Pyelonephritis
|
1.6%
1/61 • Number of events 1 • 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.
The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
|
|
General disorders
Fever
|
1.6%
1/61 • Number of events 1 • 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.
The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
|
Other adverse events
| Measure |
FXIII
n=61 participants at risk
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%.
|
|---|---|
|
Infections and infestations
Upper respiratory infection
|
6.6%
4/61 • Number of events 5 • 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.
The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
8.2%
5/61 • Number of events 5 • 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.
The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
|
|
General disorders
Fever
|
9.8%
6/61 • Number of events 7 • 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.
The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
|
|
Injury, poisoning and procedural complications
Bruising
|
6.6%
4/61 • Number of events 5 • 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.
The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
|
|
Gastrointestinal disorders
Vomiting
|
4.9%
3/61 • Number of events 4 • 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.
The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
|
|
Nervous system disorders
Headache
|
6.6%
4/61 • Number of events 4 • 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.
The Safety Population consisted of all subjects who received a dose of Factor XIII Concentrate (Human) during the study.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee 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.
- Publication restrictions are in place
Restriction type: OTHER