Trial Outcomes & Findings for A Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency (NCT NCT00885742)

NCT ID: NCT00885742

Last Updated: 2012-07-16

Results Overview

The number of subjects requiring treatment with a Factor XIII-containing product to treat a spontaneous bleeding event.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

41 participants

Primary outcome timeframe

Up to week 52

Results posted on

2012-07-16

Participant Flow

Participant milestones

Participant milestones
Measure	FXIII All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Overall Study STARTED	41
Overall Study COMPLETED	41
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	FXIII n=41 Participants All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Age Continuous	20.1 years STANDARD_DEVIATION 11.20 • n=5 Participants
Age, Customized < 16 years	18 participants n=5 Participants
Age, Customized 16 to < 65 years	23 participants n=5 Participants
Age, Customized ≥ 65 years	0 participants n=5 Participants
Sex: Female, Male Female	16 Participants n=5 Participants
Sex: Female, Male Male	25 Participants n=5 Participants

PRIMARY outcome

Timeframe: Up to week 52

Population: 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.

The number of subjects requiring treatment with a Factor XIII-containing product to treat a spontaneous bleeding event.

Outcome measures

Outcome measures
Measure	FXIII n=41 Participants All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
The Incidence of Spontaneous Bleeding Events Requiring Treatment (Treatment is Defined as Administration of a FXIII-Containing Product to Treat the Bleeding Event)	0 participants

SECONDARY outcome

Timeframe: 12 months

Population: 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.

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.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: 12 months

Population: The Safety Population comprised all subjects who received a dose of FXIII Concentrate (Human) during the study.

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.

Outcome measures

Outcome measures
Measure	FXIII n=41 Participants All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Adverse Events Any treatment-emergent AEs	33 participants
Adverse Events Any treatment-emergent and treatment-related AE	3 participants
Adverse Events Any treatment-emergent SAE	4 participants

SECONDARY outcome

Timeframe: At 12, 24, 36 and 48 weeks: at 30 and 60 minutes after the end of the infusion.

Population: 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).

Outcome measures

Outcome measures
Measure	FXIII n=41 Participants All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Peak FXIII Concentration at Steady State Week 24 (n = 40)	1.045 Units/mL Standard Deviation 0.3825
Peak FXIII Concentration at Steady State Week 36 (n = 41)	0.962 Units/mL Standard Deviation 0.2306
Peak FXIII Concentration at Steady State Week 12 (n = 40)	0.968 Units/mL Standard Deviation 0.2292
Peak FXIII Concentration at Steady State Week 48 (n = 40)	0.983 Units/mL Standard Deviation 0.2633

SECONDARY outcome

Timeframe: At 12, 24, 36 and 48 weeks: immediately before infusion.

Outcome measures

Outcome measures
Measure	FXIII n=41 Participants All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Trough FXIII Concentration at Steady State Week 12 (n = 40)	0.132 Units/mL Standard Deviation 0.0305
Trough FXIII Concentration at Steady State Week 24 (n = 41)	0.136 Units/mL Standard Deviation 0.0362
Trough FXIII Concentration at Steady State Week 36 (n = 41)	0.130 Units/mL Standard Deviation 0.0254
Trough FXIII Concentration at Steady State Week 48 (n = 41)	0.150 Units/mL Standard Deviation 0.1138

SECONDARY outcome

Timeframe: At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.

Outcome measures

Outcome measures
Measure	FXIII n=41 Participants All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Time to Peak Concentration Week 12 (n = 40)	0.632 Hour Standard Deviation 0.2296
Time to Peak Concentration Week 24 (n = 40)	0.615 Hour Standard Deviation 0.1978
Time to Peak Concentration Week 36 (n = 39)	0.623 Hour Standard Deviation 0.2350
Time to Peak Concentration Week 48 (n = 40)	0.636 Hour Standard Deviation 0.2328

SECONDARY outcome

Timeframe: At 12, 24, 36 and 48 weeks: immediately before infusion, then at 30 and 60 minutes after the end of the infusion.

Incremental recovery (U/mL/U/kg) is defined as maximum (peak) FXIII activity (U/mL) obtained after infusion, per dose of (U/kg) infusion.

Outcome measures

Outcome measures
Measure	FXIII n=41 Participants All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Incremental Recovery Week 48 (n = 38)	0.020 Units/mL/Units/kg Standard Deviation 0.0056
Incremental Recovery Week 12 (n = 39)	0.021 Units/mL/Units/kg Standard Deviation 0.0059
Incremental Recovery Week 24 (n = 38)	0.023 Units/mL/Units/kg Standard Deviation 0.0104
Incremental Recovery Week 36 (n = 39)	0.021 Units/mL/Units/kg Standard Deviation 0.0056

SECONDARY outcome

Timeframe: At 12, 24, 36 and 48 weeks: immediately before infusion.

Number of subjects with Factor XIII level ≥ 5% before infusion at Week 12, Week 24, Week 36 and Week 48.

Outcome measures

Outcome measures
Measure	FXIII n=41 Participants All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Achievement of Trough Factor XIII Levels of 5% or Higher. Week 12 (n = 40)	40 participants
Achievement of Trough Factor XIII Levels of 5% or Higher. Week 24 (n = 41)	41 participants
Achievement of Trough Factor XIII Levels of 5% or Higher. Week 36 (n = 41)	41 participants
Achievement of Trough Factor XIII Levels of 5% or Higher. Week 48 (n = 41)	40 participants

Adverse Events

FXIII

Serious events: 4 serious events

Other events: 27 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	FXIII n=41 participants at risk All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Infections and infestations Appendicitis	2.4% 1/41 • Number of events 1 • 12 months 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.
Infections and infestations Urinary tract infection	2.4% 1/41 • Number of events 1 • 12 months 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.
Injury, poisoning and procedural complications Hip injury	2.4% 1/41 • Number of events 1 • 12 months 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.
Injury, poisoning and procedural complications Traumatic chest injury NOS	2.4% 1/41 • Number of events 1 • 12 months 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.
General disorders Chest pain with radiation to left arm	2.4% 1/41 • Number of events 1 • 12 months 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.

Other adverse events

Other adverse events
Measure	FXIII n=41 participants at risk All subjects who received a dose of Factor XIII (FXIII) Concentrate (Human).
Gastrointestinal disorders Vomiting	7.3% 3/41 • Number of events 6 • 12 months 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.
General disorders Fever	12.2% 5/41 • Number of events 5 • 12 months 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.
Injury, poisoning and procedural complications Abrasions	7.3% 3/41 • Number of events 5 • 12 months 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.
Injury, poisoning and procedural complications Fall	9.8% 4/41 • Number of events 4 • 12 months 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.
Injury, poisoning and procedural complications Bruising of thigh	7.3% 3/41 • Number of events 3 • 12 months 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.
Investigations Thrombin-antithrombin III complex increased	7.3% 3/41 • Number of events 5 • 12 months 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.
Musculoskeletal and connective tissue disorders Wrist pain	7.3% 3/41 • Number of events 3 • 12 months 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.
Nervous system disorders Headache	7.3% 3/41 • Number of events 10 • 12 months 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.
Respiratory, thoracic and mediastinal disorders Cough	12.2% 5/41 • Number of events 6 • 12 months 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.
Respiratory, thoracic and mediastinal disorders Nasal congestion	9.8% 4/41 • Number of events 4 • 12 months 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.
Respiratory, thoracic and mediastinal disorders Rhinorrhea	7.3% 3/41 • Number of events 4 • 12 months 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.
Respiratory, thoracic and mediastinal disorders Sore throat	7.3% 3/41 • Number of events 3 • 12 months 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.
Skin and subcutaneous tissue disorders Acne	7.3% 3/41 • Number of events 3 • 12 months 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.
Infections and infestations Upper respiratory infection	24.4% 10/41 • Number of events 11 • 12 months 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.

Additional Information

Clinical Trial Disclosure Manager

CSL Behring

Phone: Use email contact

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee 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.
Publication restrictions are in place

Restriction type: OTHER