Trial Outcomes & Findings for Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency (NCT NCT01253811)
NCT ID: NCT01253811
Last Updated: 2016-06-24
Results Overview
An adverse event was described as any untoward medical occurrence in a subject or clinical investigation subject administered a pharmaceutical product, and which does not necessarily have a causal relationship with this treatment. Treatment emergent adverse events (serious and non-serious), defined as adverse events occurring from first trial product administration to the end of the subject's participation in the trial.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
6 participants
Primary outcome timeframe
Week 0 to end of trial visit (week 173) for a minimum period of 52 weeks.
Results posted on
2016-06-24
Participant Flow
The trial was conducted at 5 sites in 3 countries as follows: Israel (IS): 1 site; United Kingdom (UK): 2 sites; and United States (US): 2 sites.
Subjects who completed the F13CD-3760 (NCT01230021) trial were eligible to get enrolled in this trial.
Participant milestones
| Measure |
rFXIII 35 IU/kg
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
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|---|---|
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Overall Study
STARTED
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6
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Overall Study
Exposed
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6
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Overall Study
COMPLETED
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5
|
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Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
rFXIII 35 IU/kg
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
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|---|---|
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Overall Study
Withdrawal criteria
|
1
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Baseline Characteristics
Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency
Baseline characteristics by cohort
| Measure |
rFXIII 35 IU/kg
n=6 Participants
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
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|---|---|
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Age, Continuous
|
3.0 years
STANDARD_DEVIATION 1.3 • n=5 Participants
|
|
Sex: Female, Male
Female
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3 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
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0 Participants
n=5 Participants
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|
Race (NIH/OMB)
American Indian or Alaska Native
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0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
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3 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
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1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
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2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
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PRIMARY outcome
Timeframe: Week 0 to end of trial visit (week 173) for a minimum period of 52 weeks.Population: The safety analysis set included all subjects who received at least one dose of the trial product.
An adverse event was described as any untoward medical occurrence in a subject or clinical investigation subject administered a pharmaceutical product, and which does not necessarily have a causal relationship with this treatment. Treatment emergent adverse events (serious and non-serious), defined as adverse events occurring from first trial product administration to the end of the subject's participation in the trial.
Outcome measures
| Measure |
rFXIII 35 IU/kg
n=6 Participants
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
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|---|---|
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Number of Treatment Emergent (Serious and Non-serious) Adverse Events
All adverse events
|
100 number of events
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Number of Treatment Emergent (Serious and Non-serious) Adverse Events
Serious adverse events
|
2 number of events
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Number of Treatment Emergent (Serious and Non-serious) Adverse Events
Non-serious adverse events
|
98 number of events
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SECONDARY outcome
Timeframe: Week 0 to end of trial visit (week 173).Population: The SAS included all subjects who received at least one dose of the trial product. There were no antibodies against rFXIII detected in any patient during the trial.
All subjects who received rFXIII were monitored for the frequency of development of anti-rFXIII antibodies. Samples passed through 2 tiers of ELISA testing: an initial screen with a specific cut-off point (including \~5% false positives) and a second confirmatory assay for samples yielding a result above the screening cut-off point. If samples were confirmed as antibody positive in the confirmation assay, an inhibitor assay was also carried out to detect functional inhibitors.
Outcome measures
| Measure |
rFXIII 35 IU/kg
n=6 Participants
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
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|---|---|
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Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors.
|
0 percentage of subjects
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SECONDARY outcome
Timeframe: Every 6th month, from week 24 to end of trial visit (week 173).Population: The safety analysis set included all subjects who received at least one dose of the trial product.
Clinical laboratory assessments for creatinine at week 24 to end of trial visit.
Outcome measures
| Measure |
rFXIII 35 IU/kg
n=6 Participants
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
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|---|---|
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Clinical Laboratory Assessments: Biochemistry: Creatinine
Week 24 (pre-dose), N=5
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30.20 mmol/L
Standard Deviation 8.23
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Clinical Laboratory Assessments: Biochemistry: Creatinine
Week 48 (pre-dose), N=5
|
28.00 mmol/L
Standard Deviation 6.16
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Clinical Laboratory Assessments: Biochemistry: Creatinine
Week 72 (pre-dose), N=6
|
28.67 mmol/L
Standard Deviation 5.13
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Clinical Laboratory Assessments: Biochemistry: Creatinine
Week 96 (pre-dose), N=5
|
37.00 mmol/L
Standard Deviation 4.74
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Clinical Laboratory Assessments: Biochemistry: Creatinine
Week 120 (pre-dose), N=4
|
31.50 mmol/L
Standard Deviation 5.92
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Clinical Laboratory Assessments: Biochemistry: Creatinine
Week 144 (pre-dose), N=3
|
31.00 mmol/L
Standard Deviation 6.00
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Clinical Laboratory Assessments: Biochemistry: Creatinine
Week 168 (pre-dose), N=1
|
55.00 mmol/L
Standard Deviation 0.00
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Clinical Laboratory Assessments: Biochemistry: Creatinine
End of trial (week 173) pre-dose, N= 6
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35.17 mmol/L
Standard Deviation 9.79
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SECONDARY outcome
Timeframe: Every 6th month, week 24 to end of trial visit (week 173).Population: The safety analysis set included all subjects who received at least one dose of the trial product.
Clinical laboratory assessments for urea at week 24 to end ot trial visit.
Outcome measures
| Measure |
rFXIII 35 IU/kg
n=6 Participants
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
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|---|---|
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Clinical Laboratory Assessments: Biochemistry: Urea
Week 24 (pre-dose), N=5
|
5.50 mmol/L
Standard Deviation 1.63
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Clinical Laboratory Assessments: Biochemistry: Urea
Week 48 (pre-dose), N=5
|
4.00 mmol/L
Standard Deviation 1.22
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Clinical Laboratory Assessments: Biochemistry: Urea
Week 72 (pre-dose), N=6
|
3.99 mmol/L
Standard Deviation 0.69
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Clinical Laboratory Assessments: Biochemistry: Urea
Week 96 (pre-dose), N=5
|
4.50 mmol/L
Standard Deviation 1.14
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Clinical Laboratory Assessments: Biochemistry: Urea
Week 120 (pre-dose), N=4
|
3.48 mmol/L
Standard Deviation 0.73
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Clinical Laboratory Assessments: Biochemistry: Urea
Week 144 (pre-dose), N=3
|
4.52 mmol/L
Standard Deviation 1.35
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Clinical Laboratory Assessments: Biochemistry: Urea
Week 168 (pre-dose), N=1
|
5.00 mmol/L
Standard Deviation 0.00
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Clinical Laboratory Assessments: Biochemistry: Urea
End of trial (week 173) pre-dose, N= 6
|
4.28 mmol/L
Standard Deviation 0.84
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SECONDARY outcome
Timeframe: Every 6th month, from week 24 to end of trial visit (week 173).Population: The safety analysis set included all subjects who received at least one dose of the trial product.
Clinical laboratory assessments for ALAT at week 24 to end of trial visit.
Outcome measures
| Measure |
rFXIII 35 IU/kg
n=6 Participants
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
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|---|---|
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Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT)
Week 24 (pre-dose), N=4
|
17.75 IU/L
Standard Deviation 5.85
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|
Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT)
Week 48 (pre-dose), N=5
|
16.20 IU/L
Standard Deviation 2.39
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Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT)
Week 72 (pre-dose), N=5
|
16.40 IU/L
Standard Deviation 4.93
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Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT)
Week 96 (pre-dose), N=4
|
12.50 IU/L
Standard Deviation 3.70
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Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT)
Week 120 (pre-dose), N=3
|
21.33 IU/L
Standard Deviation 12.10
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Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT)
Week 144 (pre-dose), N=2
|
17.00 IU/L
Standard Deviation 1.41
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Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT)
Week 168 (pre-dose), N=1
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14.00 IU/L
Standard Deviation 0.00
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Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT)
End of trial (week 173) pre-dose, N=5
|
16.00 IU/L
Standard Deviation 3.94
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SECONDARY outcome
Timeframe: Every 6th month, from week 24 to end of trial visit (week 173).Population: The safety analysis set included all subjects who received at least one dose of the trial product.
Clinical laboratory assessments for ASAT at week 24 to end of trial visit.
Outcome measures
| Measure |
rFXIII 35 IU/kg
n=6 Participants
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
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|---|---|
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Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT)
Week 24 (pre-dose), N=4
|
30.75 IU/L
Standard Deviation 5.68
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Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT)
Week 48 (pre-dose), N=5
|
38.20 IU/L
Standard Deviation 10.99
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Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT)
Week 72 (pre-dose), N=5
|
31.20 IU/L
Standard Deviation 8.11
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|
Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT)
Week 96 (pre-dose), N=4
|
26.25 IU/L
Standard Deviation 1.71
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Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT)
Week 120 (pre-dose), N=3
|
27.00 IU/L
Standard Deviation 3.00
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Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT)
Week 144(pre-dose), N=2
|
26.00 IU/L
Standard Deviation 1.41
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Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT)
Week 168 (pre-dose), N=1
|
25.00 IU/L
Standard Deviation 0.00
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Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT)
End of trial (week 173) pre-dose, N=5
|
28.00 IU/L
Standard Deviation 3.61
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SECONDARY outcome
Timeframe: Every 6th month, from week 0 to end of trial visit (week 173).Population: The safety analysis set included all subjects who received at least one dose of the trial product.
Clinical values for haemoglobin collected from week 0 to end of trial visit.
Outcome measures
| Measure |
rFXIII 35 IU/kg
n=6 Participants
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
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|---|---|
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Clinical Laboratory Assessments: Haematology: Haemoglobin
Week 0 (pre-dose), N=6
|
7.366 mmol/L
Standard Deviation 0.25
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Clinical Laboratory Assessments: Haematology: Haemoglobin
Week 24 (pre-dose), N=4
|
7.624 mmol/L
Standard Deviation 0.19
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Clinical Laboratory Assessments: Haematology: Haemoglobin
Week 48 (pre-dose), N=3
|
7.138 mmol/L
Standard Deviation 0.31
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Clinical Laboratory Assessments: Haematology: Haemoglobin
Week 72 (pre-dose), N=3
|
7.262 mmol/L
Standard Deviation 0.65
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Clinical Laboratory Assessments: Haematology: Haemoglobin
Week 96 (pre-dose), N=3
|
7.635 mmol/L
Standard Deviation 0.27
|
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Clinical Laboratory Assessments: Haematology: Haemoglobin
Week 120 (pre-dose), N=3
|
7.821 mmol/L
Standard Deviation 0.38
|
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Clinical Laboratory Assessments: Haematology: Haemoglobin
Week 144 (pre-dose), N=3
|
7.717 mmol/L
Standard Deviation 0.70
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Clinical Laboratory Assessments: Haematology: Haemoglobin
Week 168 (pre-dose), N=2
|
7.759 mmol/L
Standard Deviation 0.53
|
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Clinical Laboratory Assessments: Haematology: Haemoglobin
End of trial (week 173) pre-dose, N=6
|
8.048 mmol/L
Standard Deviation 0.45
|
SECONDARY outcome
Timeframe: Every 6th month, from week 0 to end of trial visit (week 173).Population: The safety analysis set included all subjects who received at least one dose of the trial product.
Clinical laboratory values for leucocytes collected from week 0 to end of trial visit.
Outcome measures
| Measure |
rFXIII 35 IU/kg
n=6 Participants
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
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|---|---|
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Clinical Laboratory Assessments: Haematology: Leucocytes
Week 0 (pre-dose), N=6
|
8.367 10^9 cells/L
Standard Deviation 2.20
|
|
Clinical Laboratory Assessments: Haematology: Leucocytes
Week 24 (pre-dose), N=4
|
7.373 10^9 cells/L
Standard Deviation 1.88
|
|
Clinical Laboratory Assessments: Haematology: Leucocytes
Week 48 (pre-dose), N=3
|
5.387 10^9 cells/L
Standard Deviation 1.06
|
|
Clinical Laboratory Assessments: Haematology: Leucocytes
Week 72 (pre-dose), N=4
|
5.863 10^9 cells/L
Standard Deviation 1.96
|
|
Clinical Laboratory Assessments: Haematology: Leucocytes
Week 96 (pre-dose), N=4
|
6.643 10^9 cells/L
Standard Deviation 1.89
|
|
Clinical Laboratory Assessments: Haematology: Leucocytes
Week 120(pre-dose), N=3
|
5.027 10^9 cells/L
Standard Deviation 2.12
|
|
Clinical Laboratory Assessments: Haematology: Leucocytes
Week 144(pre-dose), N=3
|
4.850 10^9 cells/L
Standard Deviation 3.00
|
|
Clinical Laboratory Assessments: Haematology: Leucocytes
Week 168(pre-dose), N=2
|
4.275 10^9 cells/L
Standard Deviation 2.65
|
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Clinical Laboratory Assessments: Haematology: Leucocytes
End of trial (week 173) pre-dose, N=6
|
7.605 10^9 cells/L
Standard Deviation 1.25
|
SECONDARY outcome
Timeframe: Every 6th month, from week 0 to end of trial visit (week 173).Population: The safety analysis set included all subjects who received at least one dose of the trial product.
Clinical laboratory values for thrombocytes collected from week 0 to end of trial visit.
Outcome measures
| Measure |
rFXIII 35 IU/kg
n=6 Participants
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
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|---|---|
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Clinical Laboratory Assessments: Haematology: Thrombocytes
Week 0 (pre-dose), N=6
|
316.3 10^9 cells/L
Standard Deviation 50.75
|
|
Clinical Laboratory Assessments: Haematology: Thrombocytes
Week 24 (pre-dose), N=4
|
296.0 10^9 cells/L
Standard Deviation 54.17
|
|
Clinical Laboratory Assessments: Haematology: Thrombocytes
Week 48 (pre-dose), N=3
|
277.0 10^9 cells/L
Standard Deviation 93.72
|
|
Clinical Laboratory Assessments: Haematology: Thrombocytes
Week 72 (pre-dose), N=4
|
283.3 10^9 cells/L
Standard Deviation 41.63
|
|
Clinical Laboratory Assessments: Haematology: Thrombocytes
Week 96 (pre-dose), N=4
|
332.3 10^9 cells/L
Standard Deviation 107.3
|
|
Clinical Laboratory Assessments: Haematology: Thrombocytes
Week 120 (pre-dose), N=3
|
269.7 10^9 cells/L
Standard Deviation 102.2
|
|
Clinical Laboratory Assessments: Haematology: Thrombocytes
Week 144 (pre-dose), N=3
|
324.3 10^9 cells/L
Standard Deviation 44.64
|
|
Clinical Laboratory Assessments: Haematology: Thrombocytes
Week 168 (pre-dose), N=2
|
307.0 10^9 cells/L
Standard Deviation 15.56
|
|
Clinical Laboratory Assessments: Haematology: Thrombocytes
End of trial (week 173) pre-dose, N=6
|
321.3 10^9 cells/L
Standard Deviation 60.45
|
SECONDARY outcome
Timeframe: Every 6th month, from week 0 to end of trial visit (week 173).Population: The safety analysis set included all subjects who received at least one dose of the trial product.
Clinical laboratory values for erythrocytes collected from week 0 to end of trial visit.
Outcome measures
| Measure |
rFXIII 35 IU/kg
n=6 Participants
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
|
|---|---|
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Clinical Laboratory Assessments: Haematology: Erythrocytes
Week 0 (pre-dose), N=6
|
4.863 10^12 cells/L
Standard Deviation 0.43
|
|
Clinical Laboratory Assessments: Haematology: Erythrocytes
Week 24 (pre-dose), N=4
|
4.745 10^12 cells/L
Standard Deviation 0.18
|
|
Clinical Laboratory Assessments: Haematology: Erythrocytes
Week 48 (pre-dose), N=3
|
4.587 10^12 cells/L
Standard Deviation 0.12
|
|
Clinical Laboratory Assessments: Haematology: Erythrocytes
Week 72 (pre-dose), N=4
|
4.505 10^12 cells/L
Standard Deviation 0.22
|
|
Clinical Laboratory Assessments: Haematology: Erythrocytes
Week 96 (pre-dose), N=4
|
4.628 10^12 cells/L
Standard Deviation 0.26
|
|
Clinical Laboratory Assessments: Haematology: Erythrocytes
Week 120 (pre-dose), N=3
|
4.247 10^12 cells/L
Standard Deviation 1.19
|
|
Clinical Laboratory Assessments: Haematology: Erythrocytes
Week 144 (pre-dose), N=3
|
4.627 10^12 cells/L
Standard Deviation 0.11
|
|
Clinical Laboratory Assessments: Haematology: Erythrocytes
Week 168 (pre-dose), N=2
|
4.875 10^12 cells/L
Standard Deviation 0.11
|
|
Clinical Laboratory Assessments: Haematology: Erythrocytes
End of trial (week 173), N=6
|
4.662 10^12 cells/L
Standard Deviation 0.21
|
SECONDARY outcome
Timeframe: Every 6th month, from week 0 to end of trial visit (week 173).Population: The safety analysis set included all subjects who received at least one dose of the trial product.
Clinical laboratory values for haematocrit collected from week 0 to end of trial visit.
Outcome measures
| Measure |
rFXIII 35 IU/kg
n=6 Participants
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
|
|---|---|
|
Clinical Laboratory Assessments: Haematology: Haematocrit
Week 0 (pre-dose), N=6
|
35.07 percentage of red blood cells
Standard Deviation 0.99
|
|
Clinical Laboratory Assessments: Haematology: Haematocrit
Week 24 (pre-dose), N=4
|
36.05 percentage of red blood cells
Standard Deviation 1.90
|
|
Clinical Laboratory Assessments: Haematology: Haematocrit
Week 48 (pre-dose), N=3
|
32.83 percentage of red blood cells
Standard Deviation 1.76
|
|
Clinical Laboratory Assessments: Haematology: Haematocrit
Week 72 (pre-dose), N=4
|
34.80 percentage of red blood cells
Standard Deviation 2.49
|
|
Clinical Laboratory Assessments: Haematology: Haematocrit
Week 96 (pre-dose), N=4
|
36.85 percentage of red blood cells
Standard Deviation 1.07
|
|
Clinical Laboratory Assessments: Haematology: Haematocrit
Week 120 (pre-dose), N=3
|
32.83 percentage of red blood cells
Standard Deviation 8.61
|
|
Clinical Laboratory Assessments: Haematology: Haematocrit
Week 144 (pre-dose), N=3
|
36.6 percentage of red blood cells
Standard Deviation 3.44
|
|
Clinical Laboratory Assessments: Haematology: Haematocrit
Week 168 (pre-dose), N=2
|
36.40 percentage of red blood cells
Standard Deviation 2.26
|
|
Clinical Laboratory Assessments: Haematology: Haematocrit
End of trial (week 173), N=6
|
37.45 percentage of red blood cells
Standard Deviation 3.22
|
SECONDARY outcome
Timeframe: Week 0 to end of trial visit (week 173).Population: The safety analysis set included all subjects who received at least one dose of the trial product. Two abnormal physical examination findings related to skin and musculo-skeletal system were assessed as clinically significant by the investigator during the trial.
Number of subjects in percentage with changes in values of physical examinations from week 0 to end of trial visit were collected.
Outcome measures
| Measure |
rFXIII 35 IU/kg
n=6 Participants
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
|
|---|---|
|
Physical Examinations
Skin
|
3 percentage of subjects
|
|
Physical Examinations
Musculo-skeletal system
|
1 percentage of subjects
|
SECONDARY outcome
Timeframe: Week 0 to end of trial visit (week 173).Population: The safety analysis set included all subjects who received at least one dose of the trial product.
Values collected for systolic BP from week 0 to end of trial visit.
Outcome measures
| Measure |
rFXIII 35 IU/kg
n=6 Participants
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
|
|---|---|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 0, N=6
|
107.0 mmHg
Standard Deviation 13.7
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 4, N=6
|
101.0 mmHg
Standard Deviation 8.4
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 8, N=6
|
99.0 mmHg
Standard Deviation 9.4
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 12, N=6
|
106.3 mmHg
Standard Deviation 9.4
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 16, N=6
|
106.0 mmHg
Standard Deviation 4.1
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 20, N=6
|
100.7 mmHg
Standard Deviation 10.0
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 24, N=6
|
112.0 mmHg
Standard Deviation 12.6
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 28, N=6
|
101.3 mmHg
Standard Deviation 11.4
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 32, N=6
|
103.7 mmHg
Standard Deviation 20.6
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 36, N=6
|
102.3 mmHg
Standard Deviation 11.9
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 40, N=6
|
99.7 mmHg
Standard Deviation 8.3
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 44, N=6
|
99.7 mmHg
Standard Deviation 13.0
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 48, N=6
|
103.5 mmHg
Standard Deviation 19.4
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 60, N=5
|
105.4 mmHg
Standard Deviation 7.8
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 72, N=6
|
101.7 mmHg
Standard Deviation 7.3
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 84, N=6
|
100.7 mmHg
Standard Deviation 10.7
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 96, N=5
|
101.2 mmHg
Standard Deviation 4.8
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 108, N=5
|
109.6 mmHg
Standard Deviation 10.9
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 120, N=4
|
107.0 mmHg
Standard Deviation 7.0
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 132, N=4
|
99.3 mmHg
Standard Deviation 6.2
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 144, N=3
|
101.7 mmHg
Standard Deviation 7.8
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 156, N=3
|
110.7 mmHg
Standard Deviation 7.0
|
|
Vital Signs: Systolic BP (Blood Pressure)
Week 168, N=2
|
107.0 mmHg
Standard Deviation 7.1
|
|
Vital Signs: Systolic BP (Blood Pressure)
End of trial (week 173), N=6
|
96.5 mmHg
Standard Deviation 4.8
|
SECONDARY outcome
Timeframe: Week 0 to end of trial visit (week 173).Population: The safety analysis set one dose of the trial product.
Values collected for diastolic BP from week 0 to end of trial visit.
Outcome measures
| Measure |
rFXIII 35 IU/kg
n=6 Participants
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
|
|---|---|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 0, N=6
|
64.0 mmHg
Standard Deviation 7.6
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 4, N=6
|
62.7 mmHg
Standard Deviation 11.4
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 8, N=6
|
58.3 mmHg
Standard Deviation 19.1
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 12, N=6
|
57.5 mmHg
Standard Deviation 7.9
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 16, N=6
|
52.0 mmHg
Standard Deviation 3.3
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 20, N=6
|
65.2 mmHg
Standard Deviation 6.5
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 24, N=6
|
64.0 mmHg
Standard Deviation 4.4
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 28, N=6
|
58.3 mmHg
Standard Deviation 10.4
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 32, N=6
|
61.7 mmHg
Standard Deviation 16.4
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 36, N=6
|
66.2 mmHg
Standard Deviation 11.1
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 40, N=6
|
62.5 mmHg
Standard Deviation 6.4
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 44, N=6
|
60.3 mmHg
Standard Deviation 7.3
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 48, N=6
|
64.7 mmHg
Standard Deviation 10.9
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 60, N=5
|
69.4 mmHg
Standard Deviation 10.9
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 72, N=6
|
58.2 mmHg
Standard Deviation 4.6
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 84, N=6
|
57.5 mmHg
Standard Deviation 8.1
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 96, N=5
|
62.0 mmHg
Standard Deviation 6.3
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 108, N=5
|
57.6 mmHg
Standard Deviation 10.4
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 120, N=4
|
62.0 mmHg
Standard Deviation 3.4
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 132, N=4
|
55.8 mmHg
Standard Deviation 8.4
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 144, N=3
|
52.0 mmHg
Standard Deviation 9.2
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 156, N=3
|
57.7 mmHg
Standard Deviation 6.7
|
|
Vital Signs: Diastolic BP (Blood Pressure)
Week 168, N=2
|
53.5 mmHg
Standard Deviation 2.1
|
|
Vital Signs: Diastolic BP (Blood Pressure)
End of trial (week 173), N=6
|
56.2 mmHg
Standard Deviation 5.5
|
SECONDARY outcome
Timeframe: Week 0 to end of trial visit (week 173).Population: The safety analysis included all subjects who received at least one dose of the trial product.
Values collected for pulse from week 0 to end of trial visit.
Outcome measures
| Measure |
rFXIII 35 IU/kg
n=6 Participants
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
|
|---|---|
|
Vital Signs: Pulse
Week 168, N=2
|
92.0 beats/minute
Standard Deviation 9.9
|
|
Vital Signs: Pulse
End of trial (week 173), N=6
|
94.0 beats/minute
Standard Deviation 16.8
|
|
Vital Signs: Pulse
Week 0, N=6
|
113.2 beats/minute
Standard Deviation 10.6
|
|
Vital Signs: Pulse
Week 4, N=6
|
97.7 beats/minute
Standard Deviation 16.9
|
|
Vital Signs: Pulse
Week 8, N=6
|
106.2 beats/minute
Standard Deviation 4.6
|
|
Vital Signs: Pulse
Week 12, N=6
|
104.5 beats/minute
Standard Deviation 7.3
|
|
Vital Signs: Pulse
Week 16, N=6
|
109.8 beats/minute
Standard Deviation 9.8
|
|
Vital Signs: Pulse
Week 20, N=6
|
105.3 beats/minute
Standard Deviation 19.7
|
|
Vital Signs: Pulse
Week 24, N=6
|
103.7 beats/minute
Standard Deviation 6.4
|
|
Vital Signs: Pulse
Week 28, N=6
|
102.2 beats/minute
Standard Deviation 9.3
|
|
Vital Signs: Pulse
Week 32, N=6
|
108.3 beats/minute
Standard Deviation 20.5
|
|
Vital Signs: Pulse
Week 36, N=6
|
95.5 beats/minute
Standard Deviation 22.2
|
|
Vital Signs: Pulse
Week 40, N=6
|
93.2 beats/minute
Standard Deviation 19.5
|
|
Vital Signs: Pulse
Week 44, N=6
|
95.5 beats/minute
Standard Deviation 23.2
|
|
Vital Signs: Pulse
Week 48, N=6
|
100.5 beats/minute
Standard Deviation 7.9
|
|
Vital Signs: Pulse
Week 60, N=5
|
109.0 beats/minute
Standard Deviation 15.1
|
|
Vital Signs: Pulse
Week 72, N=6
|
100.8 beats/minute
Standard Deviation 11.4
|
|
Vital Signs: Pulse
Week 84, N=6
|
99.3 beats/minute
Standard Deviation 7.9
|
|
Vital Signs: Pulse
Week 96, N=4
|
96.5 beats/minute
Standard Deviation 16.2
|
|
Vital Signs: Pulse
Week 108, N=5
|
98.4 beats/minute
Standard Deviation 18.0
|
|
Vital Signs: Pulse
Week 120, N=4
|
85.0 beats/minute
Standard Deviation 16.4
|
|
Vital Signs: Pulse
Week 132, N=4
|
86.3 beats/minute
Standard Deviation 16.7
|
|
Vital Signs: Pulse
Week 144, N=3
|
88.0 beats/minute
Standard Deviation 11.1
|
|
Vital Signs: Pulse
Week 156, N=3
|
100.7 beats/minute
Standard Deviation 11.0
|
SECONDARY outcome
Timeframe: Weeks 0 to end of trial visit (week 173).Population: There were no bleeding episodes requiring treatment with a haemostatic agent (rFXIII) during the trial, which included subjects from full analysis set who received at least one dose of the trial product.
The rate (number per subject year) of all spontaneous, traumatic and intracranial bleeding episodes requiring treatment with FXIII-containing products during the rFXIII treatment period was assessed for treatment period.
Outcome measures
Outcome data not reported
Adverse Events
rFXIII 35 IU/kg
Serious events: 1 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
rFXIII 35 IU/kg
n=6 participants at risk
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
|
|---|---|
|
Injury, poisoning and procedural complications
Head injury
|
16.7%
1/6 • Number of events 2 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
Other adverse events
| Measure |
rFXIII 35 IU/kg
n=6 participants at risk
Subjects received 35 IU/kg recombinant factor XIII (rFXIII) slow intravenous (i.v.) administered every 4 weeks (28 ± 2 days) as preventive treatment of bleeding episodes. Each dose was reconstituted, diluted with saline and injection was given at a rate not exceeding 1-2 mL min-1. This dose was identical to the dose administered in the trial F13CD-3760. The correct dosing was calculated based on subject's body weight. Subjects received rFXIII either at the clinic during the first year or as home treatment after one year, and only if allowed, according to local regulations.
|
|---|---|
|
Blood and lymphatic system disorders
Lymphopenia
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Gastrointestinal disorders
Abdominal pain upper
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Gastrointestinal disorders
Diarrhoea
|
50.0%
3/6 • Number of events 5 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Gastrointestinal disorders
Nausea
|
33.3%
2/6 • Number of events 2 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Gastrointestinal disorders
Tooth loss
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Gastrointestinal disorders
Vomiting
|
33.3%
2/6 • Number of events 4 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
General disorders
Fatigue
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
General disorders
Infusion site extravasation
|
33.3%
2/6 • Number of events 2 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
General disorders
Infusion site rash
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
General disorders
Pain
|
33.3%
2/6 • Number of events 2 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
General disorders
Pyrexia
|
66.7%
4/6 • Number of events 6 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Infections and infestations
Acute tonsillitis
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Infections and infestations
Gastroenteritis
|
33.3%
2/6 • Number of events 3 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Infections and infestations
Gastroenteritis viral
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Infections and infestations
Influenza
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Infections and infestations
Nasopharyngitis
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Infections and infestations
Otitis media acute
|
16.7%
1/6 • Number of events 2 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Infections and infestations
Pharyngitis streptococcal
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Infections and infestations
Upper respiratory tract infection
|
50.0%
3/6 • Number of events 7 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Infections and infestations
Varicella
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Infections and infestations
Viral infection
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Injury, poisoning and procedural complications
Contusion
|
50.0%
3/6 • Number of events 3 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Injury, poisoning and procedural complications
Fall
|
50.0%
3/6 • Number of events 7 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Injury, poisoning and procedural complications
Incorrect dose administered
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Injury, poisoning and procedural complications
Joint injury
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Injury, poisoning and procedural complications
Laceration
|
16.7%
1/6 • Number of events 2 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Injury, poisoning and procedural complications
Limb injury
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
33.3%
2/6 • Number of events 2 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Injury, poisoning and procedural complications
Thermal burn
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Injury, poisoning and procedural complications
Traumatic haematoma
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Injury, poisoning and procedural complications
Traumatic haemorrhage
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Investigations
Bacterial test positive
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Metabolism and nutrition disorders
Decreased appetite
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Nervous system disorders
Headache
|
50.0%
3/6 • Number of events 3 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
33.3%
2/6 • Number of events 3 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
16.7%
1/6 • Number of events 2 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory disorder
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
33.3%
2/6 • Number of events 7 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Respiratory, thoracic and mediastinal disorders
Sneezing
|
33.3%
2/6 • Number of events 3 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Respiratory, thoracic and mediastinal disorders
Snoring
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
16.7%
1/6 • Number of events 1 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Skin and subcutaneous tissue disorders
Rash
|
16.7%
1/6 • Number of events 3 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
|
Vascular disorders
Haematoma
|
33.3%
2/6 • Number of events 2 • All adverse events were collected and reported from screening (visit 1) and until the end of trial visit for a minimum period of 52 weeks.
The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Novo Nordisk maintains the right to be informed of any Investigator plans for publication and to review any scientific paper, presentation, communication or other information concerning the investigation described in this protocol. Any such communication must be submitted in writing to the Novo Nordisk trial manager prior to submission for comments. Comments will be given within four weeks from receipt of the planned communication.
- Publication restrictions are in place
Restriction type: OTHER