Trial Outcomes & Findings for A Study of the Use of Factor XIII Concentrate in Patients With Inherited FXIII Deficiency (NCT NCT00883090)

NCT ID: NCT00883090

Last Updated: 2012-01-16

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

15 participants

Primary outcome timeframe

12 weeks

Results posted on

2012-01-16

Participant Flow

One subject was not administered FXIII because of the Sponsor's decision. This subject was not included in the analyses.

Participant milestones

Participant milestones
Measure	FXIII All subjects treated with Factor XIII Concentrate (Human) (FXIII)
Overall Study STARTED	15
Overall Study COMPLETED	13
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	FXIII All subjects treated with Factor XIII Concentrate (Human) (FXIII)
Overall Study Sponsor's decision	1
Overall Study Withdrawal by Subject	1

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	FXIII n=14 Participants All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Age Continuous	24.0 years STANDARD_DEVIATION 12.55 • n=99 Participants
Sex: Female, Male Female	7 Participants n=99 Participants
Sex: Female, Male Male	7 Participants n=99 Participants

PRIMARY outcome

Timeframe: 12 weeks

Population: 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).

Outcome measures

Outcome measures
Measure	FXIII n=13 Participants All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Peak FXIII Concentration at Steady State	0.9 Units/mL Standard Deviation 0.20

PRIMARY outcome

Timeframe: 12 weeks

Population: 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).

Outcome measures

Outcome measures
Measure	FXIII n=13 Participants All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Trough FXIII Concentration at Steady State	0.05 Units/mL Standard Deviation 0.05

PRIMARY outcome

Timeframe: 12 weeks

Population: 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).

Outcome measures

Outcome measures
Measure	FXIII n=13 Participants All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Time to Peak Concentration	1.7 hr Standard Deviation 1.44

PRIMARY outcome

Timeframe: 12 weeks

Population: 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).

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.

Outcome measures

Outcome measures
Measure	FXIII n=13 Participants All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Incremental Recovery	0.02 Units/mL/Units/kg Standard Deviation 0.01

PRIMARY outcome

Timeframe: 12 weeks

Population: 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).

Outcome measures

Outcome measures
Measure	FXIII n=13 Participants All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Terminal Half-life	6.6 days Standard Deviation 2.29

PRIMARY outcome

Timeframe: 12 weeks

Population: 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).

Outcome measures

Outcome measures
Measure	FXIII n=13 Participants All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Area Under the Curve at Steady State	184.0 Units*hr/mL Standard Deviation 65.78

PRIMARY outcome

Timeframe: 12 weeks

Population: 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).

Outcome measures

Outcome measures
Measure	FXIII n=13 Participants All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Clearance	0.25 mL/hr/kg Standard Deviation 0.09

PRIMARY outcome

Timeframe: 12 weeks

Population: 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).

Outcome measures

Outcome measures
Measure	FXIII n=13 Participants All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Volume of Distribution at Steady State	51.1 mL/kg Standard Deviation 12.61

PRIMARY outcome

Timeframe: 12 weeks

Population: 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).

Outcome measures

Outcome measures
Measure	FXIII n=13 Participants All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Mean Residence Time	10.0 days Standard Deviation 3.45

SECONDARY outcome

Timeframe: 16 weeks

Population: The analysis population was the safety population. The safety population comprised all subjects who received a dose of Factor XIII.

Number of participants with an adverse event

Outcome measures

Outcome measures
Measure	FXIII n=14 Participants All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Adverse Events	8 participants

SECONDARY outcome

Timeframe: 16 weeks

Population: The analysis population was the safety population. The safety population comprised all subjects who received a dose of Factor XIII.

Number of participants with clinically significant laboratory safety parameter values. The laboratory safety parameters measured included serum chemistries, hematology and urinalysis.

Outcome measures

Outcome measures
Measure	FXIII n=14 Participants All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Laboratory Safety Parameters	0 participants

SECONDARY outcome

Timeframe: 16 weeks

Population: The analysis population was the safety population. The safety population comprised all subjects who received a dose of Factor XIII.

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.

Outcome measures

Outcome measures
Measure	FXIII n=14 Participants All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Vital Signs	0 participants

Adverse Events

FXIII

Serious events: 0 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	FXIII n=14 participants at risk All subjects treated with Factor FXIII Concentrate (Human) (FXIII)
Infections and infestations Acute bronchitis	14.3% 2/14 • Number of events 2 • 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.
Infections and infestations Flu	7.1% 1/14 • Number of events 1 • 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.
Infections and infestations Infected sebaceous cyst	7.1% 1/14 • Number of events 1 • 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.
Infections and infestations Tinea corporis	7.1% 1/14 • Number of events 1 • 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.
Infections and infestations Urinary tract infection	7.1% 1/14 • Number of events 1 • 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.
Injury, poisoning and procedural complications Ankle injury	7.1% 1/14 • Number of events 1 • 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.
Injury, poisoning and procedural complications Bruising of arm	7.1% 1/14 • Number of events 1 • 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.
Injury, poisoning and procedural complications Contusion of knee	7.1% 1/14 • Number of events 1 • 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.
Injury, poisoning and procedural complications Contusion of toe	7.1% 1/14 • Number of events 1 • 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.
Investigations Fibrin D dimer increased	7.1% 1/14 • Number of events 1 • 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.
Investigations Prothrombin increased	7.1% 1/14 • Number of events 1 • 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.
Investigations Thrombin-antithrombin III complex increased	7.1% 1/14 • Number of events 1 • 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.
Skin and subcutaneous tissue disorders Ecchymosis	7.1% 1/14 • Number of events 1 • 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.
Skin and subcutaneous tissue disorders Rash	7.1% 1/14 • Number of events 1 • 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.
Metabolism and nutrition disorders Borderline diabetes	7.1% 1/14 • Number of events 1 • 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.
Reproductive system and breast disorders Penile adhesion	7.1% 1/14 • Number of events 1 • 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.

Additional Information

Clinical Trial Disclosure Manager

CSL Behring

Phone: Use email contact

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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.
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