Trial Outcomes & Findings for Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B (NCT NCT02141074)
NCT ID: NCT02141074
Last Updated: 2025-12-23
Results Overview
Number of participants with incidence of inihibitory antibodies against FIX after 50 ED is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
54 participants
Primary outcome timeframe
When minimum 20 previously untreated patients (PUPs) have reached at least 50 exposure days (ED) (up to 156 weeks)
Results posted on
2025-12-23
Participant Flow
The trial was conducted at 29 sites in 11 countries as follows (number of sites that screened participants/ number of sites that randomised participants): Australia (1/1); Austria (2/2); Canada (1/1); Israel (1/1); Japan (1/1); Malaysia (4/4); Spain (3/3); Taiwan (2/2); Thailand (2/2); United Kingdom (3/3); United States (9/9).
Trial consists of main phase including pre-prophylaxis \& prophylaxis, extension phase \& prophylaxis period until end of treatment. Pre-prophylaxis was optional and allowed participants to receive treatment until 24 months of age/upon reaching 20EDs, whichever came first. Other participants directly started on prophylaxis treatment at visit 1.
Participant milestones
| Measure |
Pre-prophylaxis
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
Pre-prophylaxis
STARTED
|
34
|
0
|
|
Pre-prophylaxis
COMPLETED
|
31
|
0
|
|
Pre-prophylaxis
NOT COMPLETED
|
3
|
0
|
|
Prophylaxis
STARTED
|
0
|
51
|
|
Prophylaxis
COMPLETED
|
0
|
41
|
|
Prophylaxis
NOT COMPLETED
|
0
|
10
|
Reasons for withdrawal
| Measure |
Pre-prophylaxis
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
Pre-prophylaxis
Adverse Event
|
3
|
0
|
|
Prophylaxis
Withdrawal by parent/guardian
|
0
|
2
|
|
Prophylaxis
Adverse Event
|
0
|
4
|
|
Prophylaxis
Withdrawn due to closure of trial
|
0
|
3
|
|
Prophylaxis
Withdrawal by Subject
|
0
|
1
|
Baseline Characteristics
Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B
Baseline characteristics by cohort
| Measure |
Nonacog Beta Pegol
n=54 Participants
Participants received pre-prophylaxis treatment of nonacog beta pegol 40 IU/kg intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED) whichever came first in the main phase. After which they switched to prophylaxis treatment. In prophylaxis, participants received nonacog beta pegol 40 IU/kg intravenous injection once weekly in the main phase, extension phase and until end of treatment.
|
|---|---|
|
Age, Continuous
|
0.8 years
STANDARD_DEVIATION 1.1 • n=9 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=9 Participants
|
|
Sex: Female, Male
Male
|
54 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
52 Participants
n=9 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=9 Participants
|
|
Race/Ethnicity, Customized
Asian
|
19 Participants
n=9 Participants
|
|
Race/Ethnicity, Customized
Black or African American
|
7 Participants
n=9 Participants
|
|
Race/Ethnicity, Customized
White
|
25 Participants
n=9 Participants
|
|
Race/Ethnicity, Customized
Other
|
3 Participants
n=9 Participants
|
PRIMARY outcome
Timeframe: When minimum 20 previously untreated patients (PUPs) have reached at least 50 exposure days (ED) (up to 156 weeks)Population: Safety analysis set included all participants exposed to nonacog beta pegol. Overall Number of Participants Analyzed = participants with available data for this outcome measure.
Number of participants with incidence of inihibitory antibodies against FIX after 50 ED is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.
Outcome measures
| Measure |
Pre-prophylaxis
n=34 Participants
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
n=28 Participants
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
Number of Participants With Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) (50 Exposure Days)
|
2 Participants
|
0 Participants
|
PRIMARY outcome
Timeframe: When minimum 40 PUPs have reached at least 100 ED (up to 208 weeks)Population: Safety analysis set included all participants exposed to nonacog beta pegol. Overall Number of Participants Analyzed = participants with available data for this outcome measure.
Number of participants with incidence of inihibitory antibodies against FIX after 100 ED is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.
Outcome measures
| Measure |
Pre-prophylaxis
n=19 Participants
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
n=47 Participants
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
Number of Participants With Incidence of Inhibitory Antibodies Against FIX (100 ED)
|
2 Participants
|
2 Participants
|
PRIMARY outcome
Timeframe: At end of trial (up to 434 weeks)Population: Safety analysis set included all participants exposed to nonacog beta pegol. Overall Number of Participants Analyzed = participants with available data for this outcome measure.
Number of participants with incidence of inihibitory antibodies against FIX at end of trial is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.
Outcome measures
| Measure |
Pre-prophylaxis
n=20 Participants
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
n=51 Participants
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
Number of Participants With Incidence of Inhibitory Antibodies Against FIX (At End of Trial)
|
2 Participants
|
2 Participants
|
SECONDARY outcome
Timeframe: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)Population: Safety analysis set included all participants exposed to nonacog beta pegol. Overall Number of Participants Analyzed = participants with available data for this outcome measure.
Number of adverse events after 50 ED, after 100 ED, and at end of trial is presented. An adverse event was defined as any untoward medical occurrence in a participant who was administered a product, and which does not necessarily have a causal relationship with this treatment. All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment.
Outcome measures
| Measure |
Pre-prophylaxis
n=34 Participants
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
n=51 Participants
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
Number of Adverse Events
50 ED
|
86 Events
|
291 Events
|
|
Number of Adverse Events
100 ED
|
131 Events
|
610 Events
|
|
Number of Adverse Events
End of trial (Week 434)
|
134 Events
|
794 Events
|
SECONDARY outcome
Timeframe: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)Population: Safety analysis set included all participants exposed to nonacog beta pegol. Overall Number of Participants Analyzed = participants with available data for this outcome measure.
Frequency of adverse events after 50 ED, after 100 ED, and at end of trial is presented. Frequency of adverse events was expressed as number of adverse events per participant years of exposure (total number of events /total time in trial). An adverse event was defined as any untoward medical occurrence in a participant who was administered a product, and which does not necessarily have a causal relationship with this treatment. All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment.
Outcome measures
| Measure |
Pre-prophylaxis
n=34 Participants
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
n=51 Participants
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
Frequency of Adverse Events
50 ED
|
5.59 Events per participant years of exposure
|
5.87 Events per participant years of exposure
|
|
Frequency of Adverse Events
100 ED
|
5.92 Events per participant years of exposure
|
5.08 Events per participant years of exposure
|
|
Frequency of Adverse Events
End of trial (Week 434)
|
5.52 Events per participant years of exposure
|
4.10 Events per participant years of exposure
|
SECONDARY outcome
Timeframe: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)Population: Safety analysis set included all participants exposed to nonacog beta pegol. Overall Number of Participants Analyzed = participants with available data for this outcome measure.
Number of serious adverse events after 50 ED, after 100 ED, and at end of trial is presented. A serious adverse event was an experience that at any dose resulted in: death; life-threatening experience; in-patient hospitalisation or prolongation of existing hospitalisation; a persistent or significant disability/incapacity; congenital anomaly/birth defect; important medical events that may not result in death, be life-threatening/require hospitalisation could be considered a serious adverse event based upon appropriate medical judgement.
Outcome measures
| Measure |
Pre-prophylaxis
n=34 Participants
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
n=51 Participants
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
Number of Serious Adverse Events
50 ED
|
9 Events
|
14 Events
|
|
Number of Serious Adverse Events
100 ED
|
13 Events
|
27 Events
|
|
Number of Serious Adverse Events
End of trial (Week 434)
|
14 Events
|
30 Events
|
SECONDARY outcome
Timeframe: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)Population: Safety analysis set included all participants exposed to nonacog beta pegol. Overall Number of Participants Analyzed = participants with available data for this outcome measure.
Frequency of serious adverse events after 50 ED, after 100 ED, and at end of trial is presented. Frequency of serious adverse events was expressed as number of serious adverse events per participant years of exposure (total number of events /total time in trial). A serious adverse event was an experience that at any dose resulted in: death; life-threatening experience; in-patient hospitalisation or prolongation of existing hospitalisation; a persistent or significant disability/incapacity; congenital anomaly/birth defect; important medical events that may not result in death, be life-threatening/require hospitalisation could be considered a serious adverse event based upon appropriate medical judgement.
Outcome measures
| Measure |
Pre-prophylaxis
n=34 Participants
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
n=51 Participants
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
Frequency of Serious Adverse Events
50 ED
|
0.59 Events per participant years of exposure
|
0.28 Events per participant years of exposure
|
|
Frequency of Serious Adverse Events
100 ED
|
0.59 Events per participant years of exposure
|
0.22 Events per participant years of exposure
|
|
Frequency of Serious Adverse Events
End of trial (Week 434)
|
0.58 Events per participant years of exposure
|
0.15 Events per participant years of exposure
|
SECONDARY outcome
Timeframe: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)Population: Safety analysis set included all participants exposed to nonacog beta pegol. Overall Number of Participants Analyzed = participants with available data for this outcome measure.
Number of medical events of special interest after 50 ED, after 100 ED, and at end of trial is presented. A medical event of special interest (MESI) was an event that, in the evaluation of safety, has a special focus. A MESI was an adverse event (serious or non-serious adverse event) that fulfils one or more of the following MESI criteria: 1. Medication errors concerning the trial product; 2. Inhibitor formation against FIX; 3. Thromboembolic events; 4. Anaphylactic reaction; 5. Allergic reaction including, but not limited to, any acute immunoglobulin E mediated reaction of delayed-type hypersensitivity (clinical signs may include various types of skin rashes) that does not meet the definition of anaphylaxis; 6. CNS-related adverse events including, but not limited to, any learning and behavioral deficits; 7. Renal adverse events including new onset of renal disorder or renal impairment or acute and chronic renal failure.
Outcome measures
| Measure |
Pre-prophylaxis
n=34 Participants
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
n=51 Participants
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
Number of Medical Events of Special Interest
50 ED
|
5 Events
|
9 Events
|
|
Number of Medical Events of Special Interest
100 ED
|
6 Events
|
31 Events
|
|
Number of Medical Events of Special Interest
End of trial (Week 434)
|
6 Events
|
38 Events
|
SECONDARY outcome
Timeframe: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)Population: Safety analysis set included all participants exposed to nonacog beta pegol. Overall Number of Participants Analyzed = participants with available data for this outcome measure.
Frequency of medical events of special interest after 50 ED, after 100 ED, and at end of trial is presented. It was expressed as number of MESI per participant years of exposure (total number of events/total time in trial). A MESI was an event that, in the evaluation of safety, has a special focus. A MESI was an adverse event that fulfils one or more of the MESI criteria: 1. Medication errors concerning the trial product; 2. Inhibitor formation against FIX; 3. Thromboembolic events; 4. Anaphylactic reaction; 5. Allergic reaction including, but not limited to, any acute immunoglobulin E mediated reaction of delayed-type hypersensitivity (clinical signs may include various types of skin rashes) that does not meet the definition of anaphylaxis; 6. CNS-related adverse events including, but not limited to, any learning and behavioral deficits; 7. Renal adverse events including new onset of renal disorder or renal impairment or acute and chronic renal failure.
Outcome measures
| Measure |
Pre-prophylaxis
n=34 Participants
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
n=51 Participants
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
Frequency of Medical Events of Special Interest
50 ED
|
0.33 Events per participant years of exposure
|
0.18 Events per participant years of exposure
|
|
Frequency of Medical Events of Special Interest
100 ED
|
0.27 Events per participant years of exposure
|
0.26 Events per participant years of exposure
|
|
Frequency of Medical Events of Special Interest
End of trial (Week 434)
|
0.25 Events per participant years of exposure
|
0.20 Events per participant years of exposure
|
SECONDARY outcome
Timeframe: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)Population: Full analysis set included all participants exposed to nonacog beta pegol. Overall Number of Participants Analyzed = participants with available data for this outcome measure.
Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate) after 50 ED, after 100 ED, and at end of trial is presented. Annualised bleeding rate is the number of bleeding episodes per year.
Outcome measures
| Measure |
Pre-prophylaxis
n=137 bleeding episodes
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
Number of Breakthrough Bleeding Episodes During Prophylaxis (Annualised Bleeding Rate)
50 ED
|
0.00 bleeds/participant/year
Interval 0.0 to 2.6
|
—
|
|
Number of Breakthrough Bleeding Episodes During Prophylaxis (Annualised Bleeding Rate)
100 ED
|
0.25 bleeds/participant/year
Interval 0.0 to 17.39
|
—
|
|
Number of Breakthrough Bleeding Episodes During Prophylaxis (Annualised Bleeding Rate)
End of trial (Week 434)
|
0.33 bleeds/participant/year
Interval 0.0 to 17.39
|
—
|
SECONDARY outcome
Timeframe: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)Population: Full analysis set included all participants exposed to nonacog beta pegol. Overall Number of Participants Analyzed = participants with available data for this outcome measure.
Haemostatic effect of nonacog beta pegol in treatment of bleeding episodes by 4-point haemostatic response scale after 50 ED, after 100 ED, and at end of trial is presented. The haemostatic response after treatment of a bleed with nonacog beta pegol was evaluated on a 4-point scale as excellent, good, moderate or poor. Excellent: abrupt pain relief and/or clear improvement in objective signs of bleeding; Good: noticeable pain relief and/or improvement in signs of bleeding; Moderate: probable or slight beneficial effect after the first injection; Poor: no improvement or worsening of symptoms. If the haemostatic response was rated as excellent or good, the treatment of the bleed was considered a success. If the haemostatic response was rated as moderate or poor, the treatment was considered a failure.
Outcome measures
| Measure |
Pre-prophylaxis
n=65 Bleeding episodes
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
n=135 Bleeding episodes
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
Haemostatic Effect of Nonacog Beta Pegol in Treatment of Bleeding Episodes by 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "Poor")
50 ED Excellent
|
23 Number of bleeds treated
|
9 Number of bleeds treated
|
|
Haemostatic Effect of Nonacog Beta Pegol in Treatment of Bleeding Episodes by 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "Poor")
50 ED Good
|
8 Number of bleeds treated
|
5 Number of bleeds treated
|
|
Haemostatic Effect of Nonacog Beta Pegol in Treatment of Bleeding Episodes by 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "Poor")
50 ED Moderate
|
2 Number of bleeds treated
|
1 Number of bleeds treated
|
|
Haemostatic Effect of Nonacog Beta Pegol in Treatment of Bleeding Episodes by 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "Poor")
50 ED Poor
|
0 Number of bleeds treated
|
0 Number of bleeds treated
|
|
Haemostatic Effect of Nonacog Beta Pegol in Treatment of Bleeding Episodes by 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "Poor")
100 ED Excellent
|
44 Number of bleeds treated
|
44 Number of bleeds treated
|
|
Haemostatic Effect of Nonacog Beta Pegol in Treatment of Bleeding Episodes by 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "Poor")
100 ED Good
|
17 Number of bleeds treated
|
30 Number of bleeds treated
|
|
Haemostatic Effect of Nonacog Beta Pegol in Treatment of Bleeding Episodes by 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "Poor")
100 ED Moderate
|
2 Number of bleeds treated
|
3 Number of bleeds treated
|
|
Haemostatic Effect of Nonacog Beta Pegol in Treatment of Bleeding Episodes by 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "Poor")
100 ED Poor
|
0 Number of bleeds treated
|
0 Number of bleeds treated
|
|
Haemostatic Effect of Nonacog Beta Pegol in Treatment of Bleeding Episodes by 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "Poor")
End of trial (Week 434) Excellent
|
45 Number of bleeds treated
|
86 Number of bleeds treated
|
|
Haemostatic Effect of Nonacog Beta Pegol in Treatment of Bleeding Episodes by 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "Poor")
End of trial (Week 434) Good
|
18 Number of bleeds treated
|
45 Number of bleeds treated
|
|
Haemostatic Effect of Nonacog Beta Pegol in Treatment of Bleeding Episodes by 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "Poor")
End of trial (Week 434) Moderate
|
2 Number of bleeds treated
|
4 Number of bleeds treated
|
|
Haemostatic Effect of Nonacog Beta Pegol in Treatment of Bleeding Episodes by 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "Poor")
End of trial (Week 434) Poor
|
0 Number of bleeds treated
|
0 Number of bleeds treated
|
SECONDARY outcome
Timeframe: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks)Population: Full analysis set included all participants exposed to nonacog beta pegol. Overall Number of Participants Analyzed = participants with available data for this outcome measure.
Incremental recovery 30 minutes post dosing (IR30min) after 50 ED, after 100 ED is presented. IR30min was defined as the rise in FIX activity per international units per kilogram (IU/kg) administered and was recorded 30 minutes after the end of nonacog beta pegol injection. It was calculated as the baseline adjusted FIX activity recorded 30 minutes after ended nonacog beta pegol injection divided by the administered dose (IU/kg body weight). It was recorded as international units per milliliter (IU/mL)/international units per kilogram (IU/kg).
Outcome measures
| Measure |
Pre-prophylaxis
n=25 Participants
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
Incremental Recovery at 30 Minutes (IR30min)
50 ED
|
0.012 (IU/mL)/(IU/kg)
Geometric Coefficient of Variation 13.9
|
—
|
|
Incremental Recovery at 30 Minutes (IR30min)
100 ED
|
0.014 (IU/mL)/(IU/kg)
Geometric Coefficient of Variation 20.1
|
—
|
SECONDARY outcome
Timeframe: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks)Population: Full analysis set included all participants exposed to nonacog beta pegol. Overall Number of Participants Analyzed = participants with available data for this outcome measure.
FIX Activity 30 minutes post dosing (C30min) after 50 ED, after 100 ED is presented. The FIX activity was measured by one-stage clotting assay - a modified activated partial thromboplastin time (aPTT) assay.
Outcome measures
| Measure |
Pre-prophylaxis
n=23 Participants
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
FIX Activity at 30 Minutes (C30min)
50 ED
|
0.652 IU/mL
Geometric Coefficient of Variation 39.5
|
—
|
|
FIX Activity at 30 Minutes (C30min)
100 ED
|
0.824 IU/mL
Geometric Coefficient of Variation 21.6
|
—
|
SECONDARY outcome
Timeframe: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)Population: Full analysis set included all participants exposed to nonacog beta pegol. Overall Number of Participants Analyzed = participants with available data for this outcome measure.
FIX trough levels after 50 ED, after 100 ED, and at end of trial is presented. FIX trough level was defined as the activity recorded immediately before nonacog beta pegol injection was given and was measured by one-stage clotting assay - a modified activated partial thromboplastin time (aPTT) assay. The analysis is based on a mixed model on the log-transformed plasma concentrations with participant as a random effect. The mean trough level is presented back-transformed to the natural scale.
Outcome measures
| Measure |
Pre-prophylaxis
n=51 Participants
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
FIX Trough Levels
50 ED
|
0.150 IU/mL
Interval 0.141 to 0.16
|
—
|
|
FIX Trough Levels
100 ED
|
0.156 IU/mL
Interval 0.144 to 0.17
|
—
|
|
FIX Trough Levels
End of trial (Week 434)
|
0.167 IU/mL
Interval 0.155 to 0.179
|
—
|
SECONDARY outcome
Timeframe: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)Population: Full analysis set included all participants exposed to nonacog beta pegol. Overall Number of Participants Analyzed = participants with available data for this outcome measure.
Amount of nonacog beta pegol administered (average dose of nonacog beta pegol) to treat a bleeding episode after 50 ED, after 100 ED, and at end of trial is presented as international units per kilogram per bleed (IU/kg/bleed).
Outcome measures
| Measure |
Pre-prophylaxis
n=65 Bleeding episodes
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
n=135 Bleeding episodes
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
Amount of Drug Administered to Treat a Bleeding Episode
50 ED
|
55.0 IU/kg/bleed
Standard Deviation 28.7
|
48.8 IU/kg/bleed
Standard Deviation 16.9
|
|
Amount of Drug Administered to Treat a Bleeding Episode
100 ED
|
47.7 IU/kg/bleed
Standard Deviation 17.6
|
58.5 IU/kg/bleed
Standard Deviation 70.0
|
|
Amount of Drug Administered to Treat a Bleeding Episode
End of trial (Week 434)
|
47.5 IU/kg/bleed
Standard Deviation 17.4
|
54.0 IU/kg/bleed
Standard Deviation 53.5
|
SECONDARY outcome
Timeframe: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)Population: Full analysis set included all participants exposed to nonacog beta pegol. Overall Number of Participants Analyzed = participants with available data for this outcome measure.
The mean number of injections needed to treat a bleeding episode is presented.
Outcome measures
| Measure |
Pre-prophylaxis
n=65 Bleeding episodes
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
n=135 Bleeding episodes
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
Number of Injections Needed to Treat a Bleeding Episode
50 ED
|
1.3 Injections per bleed
Standard Deviation 0.7
|
1.0 Injections per bleed
Standard Deviation 0.0
|
|
Number of Injections Needed to Treat a Bleeding Episode
100 ED
|
1.1 Injections per bleed
Standard Deviation 0.4
|
1.3 Injections per bleed
Standard Deviation 1.5
|
|
Number of Injections Needed to Treat a Bleeding Episode
End of trial (Week 434)
|
1.1 Injections per bleed
Standard Deviation 0.4
|
1.2 Injections per bleed
Standard Deviation 1.2
|
Adverse Events
Pre-prophylaxis
Serious events: 9 serious events
Other events: 22 other events
Deaths: 1 deaths
Prophylaxis
Serious events: 19 serious events
Other events: 46 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Pre-prophylaxis
n=34 participants at risk
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
n=51 participants at risk
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
Immune system disorders
Anaphylactic reaction
|
2.9%
1/34 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
0.00%
0/51 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Psychiatric disorders
Autism spectrum disorder
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Bacteraemia
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Investigations
Blood culture positive
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Bronchiolitis
|
2.9%
1/34 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
0.00%
0/51 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Bronchitis
|
2.9%
1/34 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
0.00%
0/51 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Catheter site infection
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Investigations
Catheterisation cardiac
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Cellulitis
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Cellulitis of male external genital organ
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Gastrointestinal disorders
Dental caries
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
3.9%
2/51 • Number of events 2 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Device related infection
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Blood and lymphatic system disorders
Factor IX inhibition
|
5.9%
2/34 • Number of events 2 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
3.9%
2/51 • Number of events 2 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Injury, poisoning and procedural complications
Fall
|
2.9%
1/34 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
0.00%
0/51 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Gastroenteritis salmonella
|
2.9%
1/34 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
0.00%
0/51 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Gastroenteritis viral
|
2.9%
1/34 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
0.00%
0/51 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Renal and urinary disorders
Haematuria
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Nervous system disorders
Haemorrhage intracranial
|
5.9%
2/34 • Number of events 2 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Injury, poisoning and procedural complications
Head injury
|
2.9%
1/34 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Skin and subcutaneous tissue disorders
Henoch-Schonlein purpura
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Immune system disorders
Hypersensitivity
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Influenza
|
2.9%
1/34 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Nervous system disorders
Language disorder
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Otitis media viral
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Pharyngitis
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Blood and lymphatic system disorders
Platelet dysfunction
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Pneumonia
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
7.8%
4/51 • Number of events 4 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Vascular disorders
Poor venous access
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Respiratory, thoracic and mediastinal disorders
Tonsillar hypertrophy
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
2.0%
1/51 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Upper respiratory tract infection
|
2.9%
1/34 • Number of events 2 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
0.00%
0/51 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
Other adverse events
| Measure |
Pre-prophylaxis
n=34 participants at risk
Participants received nonacog beta pegol 40 international units/kilogram (IU/kg) intravenous injection at intervals longer than a week on-demand for bleeding episodes until they were 24 months of age or until 20 exposure days (ED), whichever came first, in the main phase. Participants switched from pre-prophylaxis treatment to prophylaxis treatment no later than 24 months of age/20 ED, whichever came first.
|
Prophylaxis
n=51 participants at risk
Participants who started with pre-prophylaxis were switched to prophylaxis no later than 24 months of age or upon reaching 20EDs, whichever came first. These participants that switched from pre-prophylaxis and other participants who started directly on prophylaxis since visit 1 received once weekly dosing of nonacog beta pegol 40 IU/kg intravenous injection in the main phase, extension phase, and until the end of treatment.
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
9.8%
5/51 • Number of events 6 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
7.8%
4/51 • Number of events 4 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
11.8%
6/51 • Number of events 7 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Bronchitis
|
2.9%
1/34 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
11.8%
6/51 • Number of events 8 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
COVID-19
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
11.8%
6/51 • Number of events 7 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Conjunctivitis
|
2.9%
1/34 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
7.8%
4/51 • Number of events 6 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Eye disorders
Conjunctivitis allergic
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
5.9%
3/51 • Number of events 4 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
7.8%
4/51 • Number of events 8 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Injury, poisoning and procedural complications
Contusion
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
11.8%
6/51 • Number of events 16 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
11.8%
4/34 • Number of events 5 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
33.3%
17/51 • Number of events 34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Gastrointestinal disorders
Dental caries
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
9.8%
5/51 • Number of events 7 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Gastrointestinal disorders
Diarrhoea
|
8.8%
3/34 • Number of events 3 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
21.6%
11/51 • Number of events 13 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Immune system disorders
Drug hypersensitivity
|
2.9%
1/34 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
7.8%
4/51 • Number of events 5 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Ear infection
|
8.8%
3/34 • Number of events 4 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
17.6%
9/51 • Number of events 13 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
5.9%
3/51 • Number of events 5 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
2.9%
1/34 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
5.9%
3/51 • Number of events 3 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Skin and subcutaneous tissue disorders
Erythema
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
5.9%
3/51 • Number of events 3 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Injury, poisoning and procedural complications
Fall
|
2.9%
1/34 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
15.7%
8/51 • Number of events 14 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
7.8%
4/51 • Number of events 4 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Hand-foot-and-mouth disease
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
7.8%
4/51 • Number of events 4 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Injury, poisoning and procedural complications
Head injury
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
13.7%
7/51 • Number of events 10 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Nervous system disorders
Headache
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
5.9%
3/51 • Number of events 11 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Influenza
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
9.8%
5/51 • Number of events 6 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Injury, poisoning and procedural complications
Lip injury
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
5.9%
3/51 • Number of events 3 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
2.9%
1/34 • Number of events 2 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
7.8%
4/51 • Number of events 16 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Nasopharyngitis
|
14.7%
5/34 • Number of events 12 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
45.1%
23/51 • Number of events 68 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
7.8%
4/51 • Number of events 8 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Otitis media
|
5.9%
2/34 • Number of events 2 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
11.8%
6/51 • Number of events 12 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Otitis media acute
|
5.9%
2/34 • Number of events 4 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
9.8%
5/51 • Number of events 5 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Pharyngitis
|
2.9%
1/34 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
7.8%
4/51 • Number of events 4 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Congenital, familial and genetic disorders
Phimosis
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
5.9%
3/51 • Number of events 3 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
General disorders
Pyrexia
|
32.4%
11/34 • Number of events 23 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
52.9%
27/51 • Number of events 74 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Skin and subcutaneous tissue disorders
Rash
|
8.8%
3/34 • Number of events 3 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
11.8%
6/51 • Number of events 14 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Rhinitis
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
5.9%
3/51 • Number of events 4 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
5.9%
3/51 • Number of events 3 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
8.8%
3/34 • Number of events 5 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
17.6%
9/51 • Number of events 21 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Sinusitis
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
5.9%
3/51 • Number of events 5 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Injury, poisoning and procedural complications
Skin laceration
|
2.9%
1/34 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
7.8%
4/51 • Number of events 9 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Nervous system disorders
Speech disorder developmental
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
9.8%
5/51 • Number of events 5 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Gastrointestinal disorders
Teething
|
2.9%
1/34 • Number of events 3 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
7.8%
4/51 • Number of events 7 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Injury, poisoning and procedural complications
Thermal burn
|
0.00%
0/34 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
5.9%
3/51 • Number of events 3 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Tonsillitis
|
2.9%
1/34 • Number of events 1 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
9.8%
5/51 • Number of events 8 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Upper respiratory tract infection
|
14.7%
5/34 • Number of events 6 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
37.3%
19/51 • Number of events 33 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Infections and infestations
Viral infection
|
8.8%
3/34 • Number of events 3 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
9.8%
5/51 • Number of events 10 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
|
Gastrointestinal disorders
Vomiting
|
5.9%
2/34 • Number of events 2 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
21.6%
11/51 • Number of events 15 • Up to Week 434
All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment. Safety analysis set included all participants exposed to nonacog beta pegol.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee At the end of the trial, one or more scientific publications may be prepared collaboratively by the investigator(s) and Novo Nordisk. Novo Nordisk reserves the right to postpone publication and/or communication for up to 60 days to protect intellectual property.
- Publication restrictions are in place
Restriction type: OTHER