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Trial record 48 of 82 for:    bleeding episodes | ( Map: France )

PROPACT: Retrospective Prophylaxis Patient Case Collection (PROPACT)

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ClinicalTrials.gov Identifier: NCT00882778
Recruitment Status : Completed
First Posted : April 16, 2009
Results First Posted : July 25, 2011
Last Update Posted : January 9, 2017
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

Study Type Observational
Study Design Observational Model: Case-Only;   Time Perspective: Retrospective
Conditions Congenital Bleeding Disorder
Haemophilia A With Inhibitors
Haemophilia B With Inhibitors
Intervention Drug: eptacog alfa (activated)
Enrollment 86
Recruitment Details Participating sites: Argentina (1), Canada (1), Croatia (1), Czech Republic (1), Germany (1), France (1), Ireland (1), Italy (4), Slovakia (1), Spain (3), Sweden (2), Switzerland (2), United Kingdom (1), and the United States of America (11)
Pre-assignment Details  
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Period Title: Overall Study
Started 86
Completed 86
Not Completed 0
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Baseline Participants 86
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous   [1] 
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 86 participants
All, n=86 11.6  (13.03)
Bleeding population, n=74 10.9  (12.45)
Frequent bleeding population, n=36 12.1  (12.67)
[1]
Measure Description: All = participants from bleeding population and frequent bleeding population (FBP). The FBP is a subset of the bleeding population with at least one bleed per month in the pre-prophylactic period of approximately 6 months.
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 86 participants
Below 12 years 61
Between 12 and 17 years 7
18 years or above 18
Gender  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 86 participants
Female
0
   0.0%
Male
86
 100.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 86 participants
Hispanic or Latino
12
  14.0%
Not Hispanic or Latino
33
  38.4%
Unknown or Not Reported
41
  47.7%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 86 participants
American Indian or Alaska Native
1
   1.2%
Asian
1
   1.2%
Native Hawaiian or Other Pacific Islander
1
   1.2%
Black or African American
7
   8.1%
White
43
  50.0%
More than one race
0
   0.0%
Unknown or Not Reported
33
  38.4%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 86 participants
Argentina 4
Canada 1
Croatia 2
Czech Republic 1
France 17
Germany 3
Ireland 5
Italy 7
Slovakia 1
Spain 10
Sweden 4
Switzerland 2
United Kingdom 1
United States 28
Peak historical inhibitor titre recorded   [1] 
Mean (Standard Deviation)
Unit of measure:  Bethesda Units (BU)/mL
Number Analyzed 86 participants
All, n=81 517.7  (1164.41)
Bleeding population, n=72 571  (1224.88)
Frequent bleeding population, n=35 737.9  (1457)
Missing, n=5 NA [2]   (NA)
[1]
Measure Description: All = participants from bleeding population and frequent bleeding population (FBP). The FBP is a subset of the bleeding population with at least one bleed per month in the pre-prophylactic period of approximately 6 months.
[2]
No data available
Reason for prophylaxis   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 86 participants
Prevention of recurrent bleeding 79
In preparation for surgery 0
Prep. for rehab./planned phys. activity 2
Other 2
Missing 3
[1]
Measure Description: Reason patients were given prophylactic activated recombinant human factor VII
Relationship to timing of Immune Tolerance Induction (ITI)   [1] 
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 86 participants
Pre-ITI 19
During ITI 8
Post-ITI 25
ITI dates unknown 0
Not candidate for ITI 27
Not reported 7
[1]
Measure Description: Timing (pre-, during, or post- Immune Tolerance Induction (ITI)) of prophylaxis determined within a subset of patients who had “prevention of recurrent bleeding” as the reason for prophylaxis
Duration of prophylaxis   [1] 
Median (Full Range)
Unit of measure:  Days
Number Analyzed 86 participants
All, n=86
288
(22 to 3651)
Bleeding population, n=74
293
(35 to 1580)
Frequent bleeding population, n=36
293
(46 to 1580)
[1]
Measure Description: All = participants from bleeding population and frequent bleeding population (FBP). The FBP is a subset of the bleeding population with at least one bleed per month in the pre-prophylactic period of approximately 6 months.
Weekly dose   [1] 
Median (Full Range)
Unit of measure:  Mcg/kg
Number Analyzed 86 participants
All, n=82
710
(94.2 to 3100)
Bleeding population, n=73
742.5
(94.2 to 3100)
Frequent bleeding population, n=36
656.2
(94.2 to 2768)
Missing, n=4
NA [2] 
(NA to NA)
[1]
Measure Description: All = participants from bleeding population and frequent bleeding population (FBP). The FBP is a subset of the bleeding population with at least one bleed per month in the pre-prophylactic period of approximately 6 months.
[2]
No data available
Dose frequency   [1] 
Median (Full Range)
Unit of measure:  Doses per week
Number Analyzed 86 participants
All, n=82
5.2
(1 to 24.3)
Bleeding population, n=73
5.8
(1 to 24.3)
Frequent bleeding population, n=36
5.2
(1 to 22.2)
Missing, n=4
NA [2] 
(NA to NA)
[1]
Measure Description: Average number of doses per week during prophylaxis period. All = participants from bleeding population and frequent bleeding population (FBP). The FBP is a subset of the bleeding population with at least one bleed per month in the pre-prophylactic period of approximately 6 months.
[2]
No data available
1.Primary Outcome
Title Percent Change in Total Bleed Episodes Per Month - Bleeding Population
Hide Description Percent change of bleeds per month in the pre-prophylaxis period and bleeds per month in the prophylaxis period
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 74
Measure Type: Number
Unit of Measure: percent change (%) in bleeds per month
-45
2.Primary Outcome
Title Percent Change in Total Bleed Episodes Per Month - Frequent Bleeding Population
Hide Description Percent change of bleeds per month in the pre-prophylaxis period and bleeds per month in the prophylaxis period
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 36
Measure Type: Number
Unit of Measure: percent change (%) in bleeds per month
-51
3.Primary Outcome
Title Percent Change in Total Bleed Episodes Per Month Per Age Categories - Bleeding Population
Hide Description Percent change of bleeds per month between the pre-prophylaxis period and the prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 74
Measure Type: Number
Unit of Measure: percent change (%) in bleeds per month
Paediatric, n=54 -48
Adolescent, n=7 0
Adult, n=13 -56
4.Primary Outcome
Title Percent Change in Total Bleed Episodes Per Month Per Age Categories - Frequent Bleeding Population
Hide Description Percent change of bleeds per month between the pre-prophylaxis period and the prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 36
Measure Type: Number
Unit of Measure: percent change (%) in bleeds per month
Paediatric, n=27 -56
Adolescent, n=3 5
Adult, n=6 -49
5.Primary Outcome
Title Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Infrequent Dosing
Hide Description Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Infrequent dosing was defined as less than two doses per week. All participants = paediatrics, adolescents and adults.
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 1
Measure Type: Number
Unit of Measure: percent change (%) in bleeds per month
All, n=1 -6
Paediatric, n=1 -6
Adolescent, n=0 0
Adult, n=0 0
6.Primary Outcome
Title Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Dosing Three Times Per Week
Hide Description Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Three times per week dosing was defined as dosing two to four times per week. All participants = paediatrics, adolescents and adults.
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 32
Measure Type: Number
Unit of Measure: percent change (%) in bleeds per month
All, n=32 -52
Paediatric, n=18 -50
Adolescent, n=3 12
Adult, n=11 -72
7.Primary Outcome
Title Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Daily Dosing
Hide Description Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Daily dosing was defined as 5 to 7 doses per week. All participants = paediatrics, adolescents and adults.
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 29
Measure Type: Number
Unit of Measure: percent change (%) in bleeds per month
All, n=29 -48
Paediatric, n=26 -48
Adolescent, n=2 -43
Adult, n=1 -51
8.Primary Outcome
Title Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Bleeding Population, Frequent Dosing
Hide Description Percent change of bleeds per month between the pre-prophylaxis period and the prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Frequent dosing was defined as 7 or more doses per week. All participants = paediatrics, adolescents and adults.
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 11
Measure Type: Number
Unit of Measure: percent change (%) in bleeds per month
All, n=11 -18
Paediatric, n=8 -47
Adolescent, n=2 40
Adult, n=1 35
9.Primary Outcome
Title Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Infrequent Dosing
Hide Description Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Infrequent dosing was defined as less than two doses per week. All participants = paediatrics, adolescents and adults.
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 1
Measure Type: Number
Unit of Measure: percent change (%) in bleeds per month
All, n=1 -6
Paediatric, n=1 -6
Adolescent, n=0 0
Adult, n=0 0
10.Primary Outcome
Title Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Dosing Three Times Per Week
Hide Description Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Three times per week dosing was defined as dosing two to four times per week. All participants = paediatrics, adolescents and adults.
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 17
Measure Type: Number
Unit of Measure: percent change (%) in bleeds per month
All, n=17 -56
Padiatric, n=11 -57
Adolescent, n=2 -10
Adult, n=4 -76
11.Primary Outcome
Title Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Daily Dosing
Hide Description Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Daily dosing was defined as 5 to 7 doses per week. All participants = paediatrics, adolescents and adults.
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 15
Measure Type: Number
Unit of Measure: percent change (%) in bleeds per month
All, n=15 -59
Paediatric, n=13 -62
Adolescent, n=1 47
Adult, n=1 -51
12.Primary Outcome
Title Percent Change in Total Bleed Episodes Per Month by Dosing and Age Categories - Frequent Bleeding Population, Frequent Dosing
Hide Description Percent change of bleeds per month between the pre-prophylaxis period and prophylaxis period. Paediatric patients below 12 years, adolescents 12-17 years, and adults at least 18 years. Frequent dosing was defined as 7 or more doses per week. All participants = paediatrics, adolescents and adults.
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 3
Measure Type: Number
Unit of Measure: percent change (%) in bleeds per month
All, n=3 33
Paediatric, n=2 -13
Adolescent, n=0 0
Adult, n=1 35
13.Secondary Outcome
Title Individual Dose by Dose Regimen and Age Group - Bleeding Population, Paediatric
Hide Description Individual activated recombinant human factor VII dose for paediatric patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).
Time Frame Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 53
Median (Full Range)
Unit of Measure: mcg/kg
Infrequent dosing, n=1
133.3
(133.3 to 133.3)
Dosing three times per week, n=18
185.4
(26.9 to 301.8)
Daily dosing, n=26
141.9
(54.5 to 395.4)
Frequent dosing, n=8
135
(103.3 to 155.5)
14.Secondary Outcome
Title Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adolescent
Hide Description Individual activated recombinant human factor VII dose for adolescent patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).
Time Frame Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 7
Median (Full Range)
Unit of Measure: mcg/kg
Infrequent dosing, n=0
0
(0 to 0)
Dosing three times per week, n=3
198
(77.9 to 301.8)
Daily dosing, n=2
138.9
(54.5 to 395.4)
Frequent dosing, n=2
138
(120 to 155.5)
15.Secondary Outcome
Title Individual Dose by Dose Regimen and Age Group - Bleeding Population, Adult
Hide Description Individual activated recombinant human factor VII dose for adult patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).
Time Frame Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 13
Median (Full Range)
Unit of Measure: mcg/kg
Infrequent dosing, n=0
0
(0 to 0)
Dosing three times per week, n=11
159.7
(26.9 to 290.9)
Daily dosing, n=1
117.6
(117.6 to 117.6)
Frequent dosing, n=1
103.3
(103.3 to 103.3)
16.Secondary Outcome
Title Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Paediatric
Hide Description Individual activated recombinant human factor VII dose for paediatric patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).
Time Frame Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 27
Median (Full Range)
Unit of Measure: mcg/kg
Infrequent dosing, n=1
133.3
(133.3 to 133.3)
Dosing three times per week, n=11
197.2
(77.9 to 270)
Daily dosing, n=13
135.9
(57.3 to 395.4)
Frequent dosing, n=2
131
(127 to 135)
17.Secondary Outcome
Title Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adolescent
Hide Description Individual activated recombinant human factor VII dose for adolescent patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).
Time Frame Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 3
Median (Full Range)
Unit of Measure: mcg/kg
Infrequent dosing, n=0
0
(0 to 0)
Dosing three times per week, n=2
138
(96 to 180)
Daily dosing, n=1
280
(280 to 280)
Frequent dosing, n=0
0
(0 to 0)
18.Secondary Outcome
Title Individual Dose by Dose Regimen and Age Group - Frequent Bleeding Population, Adult
Hide Description Individual activated recombinant human factor VII dose for adult patients by dosing regimen (infrequent dosing = less than 2 doses per week, three times per week = dosing 2-4 times per week, daily = 5-7 doses per week, or frequent dosing = 7 or more doses per week).
Time Frame Data was collected for the period of prophylactic treatment (prophylaxis period), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
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Hide Analysis Population Description
Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 6
Median (Full Range)
Unit of Measure: mcg/kg
Infrequent dosing, n=0
0
(0 to 0)
Dosing three times per week, n=4
162
(26.9 to 240)
Daily dosing, n=1
117.6
(117.6 to 117.6)
Frequent dosing, n=1
103.3
(103.3 to 103.3)
19.Secondary Outcome
Title Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Bleeding Population
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Bleeding population with at least one bleed in the pre-prophylaxis period of approx. 6 months. Patients from the French sites were not included because bleed location was not collected. Bleed episodes with no recorded locations were not included in the analysis
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 56
Measure Type: Number
Unit of Measure: percent change (%) in bleeds per month
All joints -39
Target joints -50
Non-joints -33
20.Secondary Outcome
Title Total Bleed Episodes Per Month by Joint, Target Joint and Non-joint - Frequent Bleeding Population
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approx. 6 months. Patients from the French sites were not included because bleed location was not collected in these patients. Bleed episodes with no recorded locations were not included in the analysis
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 27
Measure Type: Number
Unit of Measure: percent change (%) in bleeds per month
All joints -46
Target joints -56
Non-joints -42
21.Secondary Outcome
Title Healthcare Resource Consumption of Visits, Consultations, and Hospital Admissions Per Month - All Patients
Hide Description Healthcare resource consumption evaluated the absolute change in number of outpatient clinical visits, physician consultations and hospital admissions during the pre-prophylaxis to the prophylaxis period.
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Whole population of all male patients diagnosed with haemophilia A or B with inhibitor, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 83
Mean (Standard Deviation)
Unit of Measure: change in events per month
Out patient clinical visits -0.011  (1.07)
Physician consultations 0.043  (1.66)
Hospital admissions -0.097  (0.27)
22.Secondary Outcome
Title Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Bleeding Population
Hide Description Healthcare resource consumption evaluated the absolute change in number of outpatient clinical visits, physician consultations and hospital admissions during the pre-prophylaxis period to the prophylaxis period.
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Bleeding population with at least one bleed in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 74
Mean (Standard Deviation)
Unit of Measure: change in events per month
Out patient clinical visits 0.0090  (1.117)
Physician consultations 0.053  (1.76)
Hospital admissions -0.11  (0.28)
23.Secondary Outcome
Title Healthcare Resource Consumption of Visits, Consultations and Hospital Admissions Per Month - Frequent Bleeding Population
Hide Description Healthcare resource consumption evaluated the absolute change in number of outpatient clinical visits, physician consultations and hospital admissions during the pre-prophylaxis period to the prophylaxis period.
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 36
Mean (Standard Deviation)
Unit of Measure: change in events per month
Out patient clinical visits 0.0576  (1.39)
Physician consultations 0.213  (2.49)
Hospital admissions -0.11  (0.33)
24.Secondary Outcome
Title Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - All Patients
Hide Description Healthcare resource consumption evaluated the absolute change in number of total hospital length of stay and school/work absences during the pre-prophylaxis period to the prophylaxis period.
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Whole population of all male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (RFVIIa) for at least 30 days.
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 83
Mean (Standard Deviation)
Unit of Measure: change in days per month
Total hospital length of stay -0.654  (3.43)
School/work absences -1.365  (1.99)
25.Secondary Outcome
Title Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Bleeding Population
Hide Description Healthcare resource consumption evaluated the absolute change in number of total hospital length of stay and school/work absences during the pre-prophylaxis period to the prophylaxis period.
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Bleeding population with at least one bleed in the pre-prophylaxis of approximately 6 months.
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 74
Mean (Standard Deviation)
Unit of Measure: change in days per month
Total hospital length of stay -0.794  (3.55)
School/work absences -1.365  (1.99)
26.Secondary Outcome
Title Healthcare Resource Consumption of Total Hospital Length of Stay and School/Work Absences Per Month - Frequent Bleeding Population
Hide Description Healthcare resource consumption evaluated the absolute change in number of total hospital length of stay and school/work absences during the pre-prophylaxis period to the prophylaxis period.
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Frequent bleeding population is a subset of patients in the bleeding population with at least one bleed per month in the pre-prophylaxis period of approximately 6 months.
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 36
Mean (Standard Deviation)
Unit of Measure: change in days per month
Total hospital length of stay -1.064  (2.38)
School/work absences -1.927  (2.29)
27.Secondary Outcome
Title Physician Reported Outcome Assessment in Prophylaxis in Number of Patients
Hide Description Physician's assessment of prophylaxis outcome as successful, partially successful, unsuccessful, or unable to determine
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Whole population of all male patients diagnosed with haemophilia A or B with inhibitor, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 86
Measure Type: Number
Unit of Measure: patients
Successful 35
Partially successful 20
Unsuccessful 7
Unable to determine 9
Data missing or not reported 15
28.Secondary Outcome
Title Number of Physician Reported Outcome Assessment in Prophylaxis in Percentage of Patients
Hide Description Physician's assessment of prophylaxis outcome as successful, partially successful, unsuccessful, or unable to determine
Time Frame Data was collected for an average of 6 months prior to start of prophylaxis (pre-prophylaxis period) and the period of prophylactic treatment (during prophylaxis), which had no time frame limits. Participants were on prophylaxis for a median of 288 days.
Hide Outcome Measure Data
Hide Analysis Population Description
Whole population of all male patients diagnosed with haemophilia A or B with inhibitor, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description:
Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
Overall Number of Participants Analyzed 86
Measure Type: Number
Unit of Measure: percentage of patients
Successful 40
Partially successful 23
Unsuccessful 8
Unable to determine 10
Data missing or not reported 17
Time Frame Onset date 15-Dec-2007, outcome date 22-Dec-2007
Adverse Event Reporting Description Safety population is all patients in the study. This is a non-interventional study, and thus only adverse events (AEs) and serious adverse events (SAEs) reported by the investigator is possible or probably related to drug and/or medical events of special interest (MESI) are included in the table
 
Arm/Group Title Activated Recombinant Human Factor VII
Hide Arm/Group Description Male patients diagnosed with haemophilia A or B with inhibitors, who were prescribed activated recombinant human factor VII (rFVIIa) for at least 30 days. All direction for rFVIIa medication usage was at the sole discretion of the physician in accordance within their usual practice. Data was collected for approximately 6 months of pre-prophylaxis, while the prophylaxis period had no limits.
All-Cause Mortality
Activated Recombinant Human Factor VII
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Activated Recombinant Human Factor VII
Affected / at Risk (%) # Events
Total   1/86 (1.16%)    
Blood and lymphatic system disorders   
Septicaemia of Candida albicans  1  1/86 (1.16%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA 13.1
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Activated Recombinant Human Factor VII
Affected / at Risk (%) # Events
Total   0/86 (0.00%)    
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.
Results Point of Contact
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Name/Title: Public Access to Clinical Trials
Organization: Novo Nordisk A/S
EMail: clinicaltrials@novonordisk.com
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Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT00882778     History of Changes
Other Study ID Numbers: F7HAEM-3695
First Submitted: April 14, 2009
First Posted: April 16, 2009
Results First Submitted: May 11, 2011
Results First Posted: July 25, 2011
Last Update Posted: January 9, 2017