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Safety and Efficacy of Monthly Replacement Therapy With Recombinant Factor XIII (rFXIII) in Paediatric Subjects With Congenital Factor XIII A-subunit Deficiency (mentor™5)

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ClinicalTrials.gov Identifier: NCT01253811
Recruitment Status : Completed
First Posted : December 3, 2010
Results First Posted : June 24, 2016
Last Update Posted : June 24, 2016
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Congenital Bleeding Disorder
Congenital FXIII Deficiency
Intervention Drug: catridecacog
Enrollment 6
Recruitment Details 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.
Pre-assignment Details Subjects who completed the F13CD-3760 (NCT01230021) trial were eligible to get enrolled in this trial.
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description 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.
Period Title: Overall Study
Started 6
Exposed 6
Completed 5
Not Completed 1
Reason Not Completed
Withdrawal criteria             1
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 6
Hide Baseline Analysis Population Description
The safety analysis set (SAS) included all patients exposed to trial drug (rFXIII).
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 6 participants
3.0  (1.3)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
Female
3
  50.0%
Male
3
  50.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
6
 100.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
American Indian or Alaska Native
0
   0.0%
Asian
3
  50.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
1
  16.7%
White
2
  33.3%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
1.Primary Outcome
Title Number of Treatment Emergent (Serious and Non-serious) Adverse Events
Hide Description 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.
Time Frame Week 0 to end of trial visit (week 173) for a minimum period of 52 weeks.
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set included all subjects who received at least one dose of the trial product.
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: number of events
All adverse events 100
Serious adverse events 2
Non-serious adverse events 98
2.Secondary Outcome
Title Percentage of Subjects With Development of Anti-rFXIII Antibodies, Including Inhibitors.
Hide Description 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.
Time Frame Week 0 to end of trial visit (week 173).
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: percentage of subjects
0
3.Secondary Outcome
Title Clinical Laboratory Assessments: Biochemistry: Creatinine
Hide Description Clinical laboratory assessments for creatinine at week 24 to end of trial visit.
Time Frame Every 6th month, from week 24 to end of trial visit (week 173).
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set included all subjects who received at least one dose of the trial product.
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mmol/L
Week 24 (pre-dose), N=5 30.20  (8.23)
Week 48 (pre-dose), N=5 28.00  (6.16)
Week 72 (pre-dose), N=6 28.67  (5.13)
Week 96 (pre-dose), N=5 37.00  (4.74)
Week 120 (pre-dose), N=4 31.50  (5.92)
Week 144 (pre-dose), N=3 31.00  (6.00)
Week 168 (pre-dose), N=1 55.00  (0.00)
End of trial (week 173) pre-dose, N= 6 35.17  (9.79)
4.Secondary Outcome
Title Clinical Laboratory Assessments: Biochemistry: Urea
Hide Description Clinical laboratory assessments for urea at week 24 to end ot trial visit.
Time Frame Every 6th month, week 24 to end of trial visit (week 173).
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set included all subjects who received at least one dose of the trial product.
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mmol/L
Week 24 (pre-dose), N=5 5.50  (1.63)
Week 48 (pre-dose), N=5 4.00  (1.22)
Week 72 (pre-dose), N=6 3.99  (0.69)
Week 96 (pre-dose), N=5 4.50  (1.14)
Week 120 (pre-dose), N=4 3.48  (0.73)
Week 144 (pre-dose), N=3 4.52  (1.35)
Week 168 (pre-dose), N=1 5.00  (0.00)
End of trial (week 173) pre-dose, N= 6 4.28  (0.84)
5.Secondary Outcome
Title Clinical Laboratory Assessments: Biochemistry: Alanine Aminotransferase (ALAT)
Hide Description Clinical laboratory assessments for ALAT at week 24 to end of trial visit.
Time Frame Every 6th month, from week 24 to end of trial visit (week 173).
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set included all subjects who received at least one dose of the trial product.
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: IU/L
Week 24 (pre-dose), N=4 17.75  (5.85)
Week 48 (pre-dose), N=5 16.20  (2.39)
Week 72 (pre-dose), N=5 16.40  (4.93)
Week 96 (pre-dose), N=4 12.50  (3.70)
Week 120 (pre-dose), N=3 21.33  (12.10)
Week 144 (pre-dose), N=2 17.00  (1.41)
Week 168 (pre-dose), N=1 14.00  (0.00)
End of trial (week 173) pre-dose, N=5 16.00  (3.94)
6.Secondary Outcome
Title Clinical Laboratory Assessments: Biochemistry: Aspartate Aminotransferase (ASAT)
Hide Description Clinical laboratory assessments for ASAT at week 24 to end of trial visit.
Time Frame Every 6th month, from week 24 to end of trial visit (week 173).
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set included all subjects who received at least one dose of the trial product.
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: IU/L
Week 24 (pre-dose), N=4 30.75  (5.68)
Week 48 (pre-dose), N=5 38.20  (10.99)
Week 72 (pre-dose), N=5 31.20  (8.11)
Week 96 (pre-dose), N=4 26.25  (1.71)
Week 120 (pre-dose), N=3 27.00  (3.00)
Week 144(pre-dose), N=2 26.00  (1.41)
Week 168 (pre-dose), N=1 25.00  (0.00)
End of trial (week 173) pre-dose, N=5 28.00  (3.61)
7.Secondary Outcome
Title Clinical Laboratory Assessments: Haematology: Haemoglobin
Hide Description Clinical values for haemoglobin collected from week 0 to end of trial visit.
Time Frame Every 6th month, from week 0 to end of trial visit (week 173).
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set included all subjects who received at least one dose of the trial product.
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mmol/L
Week 0 (pre-dose), N=6 7.366  (0.25)
Week 24 (pre-dose), N=4 7.624  (0.19)
Week 48 (pre-dose), N=3 7.138  (0.31)
Week 72 (pre-dose), N=3 7.262  (0.65)
Week 96 (pre-dose), N=3 7.635  (0.27)
Week 120 (pre-dose), N=3 7.821  (0.38)
Week 144 (pre-dose), N=3 7.717  (0.70)
Week 168 (pre-dose), N=2 7.759  (0.53)
End of trial (week 173) pre-dose, N=6 8.048  (0.45)
8.Secondary Outcome
Title Clinical Laboratory Assessments: Haematology: Leucocytes
Hide Description Clinical laboratory values for leucocytes collected from week 0 to end of trial visit.
Time Frame Every 6th month, from week 0 to end of trial visit (week 173).
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set included all subjects who received at least one dose of the trial product.
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: 10^9 cells/L
Week 0 (pre-dose), N=6 8.367  (2.20)
Week 24 (pre-dose), N=4 7.373  (1.88)
Week 48 (pre-dose), N=3 5.387  (1.06)
Week 72 (pre-dose), N=4 5.863  (1.96)
Week 96 (pre-dose), N=4 6.643  (1.89)
Week 120(pre-dose), N=3 5.027  (2.12)
Week 144(pre-dose), N=3 4.850  (3.00)
Week 168(pre-dose), N=2 4.275  (2.65)
End of trial (week 173) pre-dose, N=6 7.605  (1.25)
9.Secondary Outcome
Title Clinical Laboratory Assessments: Haematology: Thrombocytes
Hide Description Clinical laboratory values for thrombocytes collected from week 0 to end of trial visit.
Time Frame Every 6th month, from week 0 to end of trial visit (week 173).
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set included all subjects who received at least one dose of the trial product.
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: 10^9 cells/L
Week 0 (pre-dose), N=6 316.3  (50.75)
Week 24 (pre-dose), N=4 296.0  (54.17)
Week 48 (pre-dose), N=3 277.0  (93.72)
Week 72 (pre-dose), N=4 283.3  (41.63)
Week 96 (pre-dose), N=4 332.3  (107.3)
Week 120 (pre-dose), N=3 269.7  (102.2)
Week 144 (pre-dose), N=3 324.3  (44.64)
Week 168 (pre-dose), N=2 307.0  (15.56)
End of trial (week 173) pre-dose, N=6 321.3  (60.45)
10.Secondary Outcome
Title Clinical Laboratory Assessments: Haematology: Erythrocytes
Hide Description Clinical laboratory values for erythrocytes collected from week 0 to end of trial visit.
Time Frame Every 6th month, from week 0 to end of trial visit (week 173).
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set included all subjects who received at least one dose of the trial product.
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: 10^12 cells/L
Week 0 (pre-dose), N=6 4.863  (0.43)
Week 24 (pre-dose), N=4 4.745  (0.18)
Week 48 (pre-dose), N=3 4.587  (0.12)
Week 72 (pre-dose), N=4 4.505  (0.22)
Week 96 (pre-dose), N=4 4.628  (0.26)
Week 120 (pre-dose), N=3 4.247  (1.19)
Week 144 (pre-dose), N=3 4.627  (0.11)
Week 168 (pre-dose), N=2 4.875  (0.11)
End of trial (week 173), N=6 4.662  (0.21)
11.Secondary Outcome
Title Clinical Laboratory Assessments: Haematology: Haematocrit
Hide Description Clinical laboratory values for haematocrit collected from week 0 to end of trial visit.
Time Frame Every 6th month, from week 0 to end of trial visit (week 173).
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set included all subjects who received at least one dose of the trial product.
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: percentage of red blood cells
Week 0 (pre-dose), N=6 35.07  (0.99)
Week 24 (pre-dose), N=4 36.05  (1.90)
Week 48 (pre-dose), N=3 32.83  (1.76)
Week 72 (pre-dose), N=4 34.80  (2.49)
Week 96 (pre-dose), N=4 36.85  (1.07)
Week 120 (pre-dose), N=3 32.83  (8.61)
Week 144 (pre-dose), N=3 36.6  (3.44)
Week 168 (pre-dose), N=2 36.40  (2.26)
End of trial (week 173), N=6 37.45  (3.22)
12.Secondary Outcome
Title Physical Examinations
Hide Description Number of subjects in percentage with changes in values of physical examinations from week 0 to end of trial visit were collected.
Time Frame Week 0 to end of trial visit (week 173).
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: percentage of subjects
Skin 3
Musculo-skeletal system 1
13.Secondary Outcome
Title Vital Signs: Systolic BP (Blood Pressure)
Hide Description Values collected for systolic BP from week 0 to end of trial visit.
Time Frame Week 0 to end of trial visit (week 173).
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set included all subjects who received at least one dose of the trial product.
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mmHg
Week 0, N=6 107.0  (13.7)
Week 4, N=6 101.0  (8.4)
Week 8, N=6 99.0  (9.4)
Week 12, N=6 106.3  (9.4)
Week 16, N=6 106.0  (4.1)
Week 20, N=6 100.7  (10.0)
Week 24, N=6 112.0  (12.6)
Week 28, N=6 101.3  (11.4)
Week 32, N=6 103.7  (20.6)
Week 36, N=6 102.3  (11.9)
Week 40, N=6 99.7  (8.3)
Week 44, N=6 99.7  (13.0)
Week 48, N=6 103.5  (19.4)
Week 60, N=5 105.4  (7.8)
Week 72, N=6 101.7  (7.3)
Week 84, N=6 100.7  (10.7)
Week 96, N=5 101.2  (4.8)
Week 108, N=5 109.6  (10.9)
Week 120, N=4 107.0  (7.0)
Week 132, N=4 99.3  (6.2)
Week 144, N=3 101.7  (7.8)
Week 156, N=3 110.7  (7.0)
Week 168, N=2 107.0  (7.1)
End of trial (week 173), N=6 96.5  (4.8)
14.Secondary Outcome
Title Vital Signs: Diastolic BP (Blood Pressure)
Hide Description Values collected for diastolic BP from week 0 to end of trial visit.
Time Frame Week 0 to end of trial visit (week 173).
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis set one dose of the trial product.
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: mmHg
Week 0, N=6 64.0  (7.6)
Week 4, N=6 62.7  (11.4)
Week 8, N=6 58.3  (19.1)
Week 12, N=6 57.5  (7.9)
Week 16, N=6 52.0  (3.3)
Week 20, N=6 65.2  (6.5)
Week 24, N=6 64.0  (4.4)
Week 28, N=6 58.3  (10.4)
Week 32, N=6 61.7  (16.4)
Week 36, N=6 66.2  (11.1)
Week 40, N=6 62.5  (6.4)
Week 44, N=6 60.3  (7.3)
Week 48, N=6 64.7  (10.9)
Week 60, N=5 69.4  (10.9)
Week 72, N=6 58.2  (4.6)
Week 84, N=6 57.5  (8.1)
Week 96, N=5 62.0  (6.3)
Week 108, N=5 57.6  (10.4)
Week 120, N=4 62.0  (3.4)
Week 132, N=4 55.8  (8.4)
Week 144, N=3 52.0  (9.2)
Week 156, N=3 57.7  (6.7)
Week 168, N=2 53.5  (2.1)
End of trial (week 173), N=6 56.2  (5.5)
15.Secondary Outcome
Title Vital Signs: Pulse
Hide Description Values collected for pulse from week 0 to end of trial visit.
Time Frame Week 0 to end of trial visit (week 173).
Hide Outcome Measure Data
Hide Analysis Population Description
The safety analysis included all subjects who received at least one dose of the trial product.
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: beats/minute
Week 0, N=6 113.2  (10.6)
Week 4, N=6 97.7  (16.9)
Week 8, N=6 106.2  (4.6)
Week 12, N=6 104.5  (7.3)
Week 16, N=6 109.8  (9.8)
Week 20, N=6 105.3  (19.7)
Week 24, N=6 103.7  (6.4)
Week 28, N=6 102.2  (9.3)
Week 32, N=6 108.3  (20.5)
Week 36, N=6 95.5  (22.2)
Week 40, N=6 93.2  (19.5)
Week 44, N=6 95.5  (23.2)
Week 48, N=6 100.5  (7.9)
Week 60, N=5 109.0  (15.1)
Week 72, N=6 100.8  (11.4)
Week 84, N=6 99.3  (7.9)
Week 96, N=4 96.5  (16.2)
Week 108, N=5 98.4  (18.0)
Week 120, N=4 85.0  (16.4)
Week 132, N=4 86.3  (16.7)
Week 144, N=3 88.0  (11.1)
Week 156, N=3 100.7  (11.0)
Week 168, N=2 92.0  (9.9)
End of trial (week 173), N=6 94.0  (16.8)
16.Secondary Outcome
Title Rate (Number Per Subject Year) of All Bleeding Episodes Requiring Treatment With a FXIII Containing Product Other Than Recombinant Factor XIII.
Hide Description 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.
Time Frame Weeks 0 to end of trial visit (week 173).
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame 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.
Adverse Event Reporting Description The safety analysis set included all subjects who received at least one dose of the trial product (rFXIII).
 
Arm/Group Title rFXIII 35 IU/kg
Hide Arm/Group Description 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.
All-Cause Mortality
rFXIII 35 IU/kg
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
rFXIII 35 IU/kg
Affected / at Risk (%) # Events
Total   1/6 (16.67%)    
Injury, poisoning and procedural complications   
Head injury  1  1/6 (16.67%)  2
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 16.1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
rFXIII 35 IU/kg
Affected / at Risk (%) # Events
Total   6/6 (100.00%)    
Blood and lymphatic system disorders   
Lymphopenia  1  1/6 (16.67%)  1
Gastrointestinal disorders   
Abdominal pain upper  1  1/6 (16.67%)  1
Diarrhoea  1  3/6 (50.00%)  5
Nausea  1  2/6 (33.33%)  2
Tooth loss  1  1/6 (16.67%)  1
Vomiting  1  2/6 (33.33%)  4
General disorders   
Fatigue  1  1/6 (16.67%)  1
Infusion site extravasation  1  2/6 (33.33%)  2
Infusion site rash  1  1/6 (16.67%)  1
Pain  1  2/6 (33.33%)  2
Pyrexia  1  4/6 (66.67%)  6
Infections and infestations   
Acute tonsillitis  1  1/6 (16.67%)  1
Gastroenteritis  1  2/6 (33.33%)  3
Gastroenteritis viral  1  1/6 (16.67%)  1
Influenza  1  1/6 (16.67%)  1
Nasopharyngitis  1  1/6 (16.67%)  1
Otitis media acute  1  1/6 (16.67%)  2
Pharyngitis streptococcal  1  1/6 (16.67%)  1
Upper respiratory tract infection  1  3/6 (50.00%)  7
Varicella  1  1/6 (16.67%)  1
Viral infection  1  1/6 (16.67%)  1
Injury, poisoning and procedural complications   
Arthropod bite  1  1/6 (16.67%)  1
Contusion  1  3/6 (50.00%)  3
Fall  1  3/6 (50.00%)  7
Incorrect dose administered  1  1/6 (16.67%)  1
Joint injury  1  1/6 (16.67%)  1
Laceration  1  1/6 (16.67%)  2
Limb injury  1  1/6 (16.67%)  1
Skin abrasion  1  2/6 (33.33%)  2
Thermal burn  1  1/6 (16.67%)  1
Traumatic haematoma  1  1/6 (16.67%)  1
Traumatic haemorrhage  1  1/6 (16.67%)  1
Investigations   
Bacterial test positive  1  1/6 (16.67%)  1
Metabolism and nutrition disorders   
Decreased appetite  1  1/6 (16.67%)  1
Musculoskeletal and connective tissue disorders   
Arthralgia  1  1/6 (16.67%)  1
Nervous system disorders   
Headache  1  3/6 (50.00%)  3
Respiratory, thoracic and mediastinal disorders   
Cough  1  2/6 (33.33%)  3
Epistaxis  1  1/6 (16.67%)  1
Nasal congestion  1  1/6 (16.67%)  2
Oropharyngeal pain  1  1/6 (16.67%)  1
Respiratory disorder  1  1/6 (16.67%)  1
Rhinitis allergic  1  1/6 (16.67%)  1
Rhinorrhoea  1  2/6 (33.33%)  7
Sneezing  1  2/6 (33.33%)  3
Snoring  1  1/6 (16.67%)  1
Skin and subcutaneous tissue disorders   
Dry skin  1  1/6 (16.67%)  1
Rash  1  1/6 (16.67%)  3
Vascular disorders   
Haematoma  1  2/6 (33.33%)  2
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 16.1
Limitations of the trial included the small number of subjects analysed and the sensitivity of the Berichrom® FXIII activity assay. However, congenital FXIII deficiency is a rare disease and there were no bleeds requiring haemostatic treatment.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Name/Title: Public Access to Clinical Trials
Organization: Novo Nordisk A/S
Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT01253811     History of Changes
Other Study ID Numbers: F13CD-3835
U1111-1117-1063 ( Other Identifier: WHO )
2010-020192-23 ( EudraCT Number )
First Submitted: December 1, 2010
First Posted: December 3, 2010
Results First Submitted: March 29, 2016
Results First Posted: June 24, 2016
Last Update Posted: June 24, 2016