Trial Outcomes & Findings for Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia A (NCT NCT01493778)
NCT ID: NCT01493778
Last Updated: 2020-11-30
Results Overview
The incidence rate (percentage of participants with inhibitors) of inhibitors defined as inhibitor titres ≥0.6 BU for main phase of the trial.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
60 participants
Primary outcome timeframe
From Visit 2 (21 days after screening) to Visit 5 (50-55 exposure day)
Results posted on
2020-11-30
Participant Flow
Participants were enrolled at 40 sites in 15 countries: Algeria (1 site), Austria (2 sites), China (6 sites), Denmark (1 site), Greece (2 sites), Hong Kong (1 site), Hungary (1 site), Japan (2 sites), Lithuania (1 site), Poland (2 sites), Russian Federation (2 sites), Serbia (1 site), Spain (3 sites), Turkey (3 sites), United States (12).
Sixty (60) participants were enrolled in the trial and received at least one dose of turoctocog alfa.The trial consisted of two phases: the main phase including the screening visit (visit 1) and 4 subsequent visits, and an extension phase with a rolling visit schedule consisting of 6 planned visits including 4 dispensing visits) per year.
Participant milestones
| Measure |
Turoctocog Alfa (N8) (Preventive+On-demand Treatment)
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Participants who enrolled into the trial initiated the preventive treatment no later than their second birthday or after a maximum of 2 bleeding episodes requiring treatment, whichever came first. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
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|---|---|
|
Overall Study
STARTED
|
60
|
|
Overall Study
COMPLETED
|
52
|
|
Overall Study
NOT COMPLETED
|
8
|
Reasons for withdrawal
| Measure |
Turoctocog Alfa (N8) (Preventive+On-demand Treatment)
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Participants who enrolled into the trial initiated the preventive treatment no later than their second birthday or after a maximum of 2 bleeding episodes requiring treatment, whichever came first. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
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|---|---|
|
Overall Study
Withdrawal by Subject
|
8
|
Baseline Characteristics
Safety and Efficacy of Turoctocog Alfa in Prevention and Treatment of Bleeds in Previously Untreated Children With Haemophilia A
Baseline characteristics by cohort
| Measure |
Turoctocog Alfa (N8) (Preventive+On-demand)
n=60 Participants
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Participants who enrolled into the trial initiated the preventive treatment no later than their second birthday or after a maximum of 2 bleeding episodes requiring treatment, whichever came first. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
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|---|---|
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Age, Continuous
|
10.2 Months
STANDARD_DEVIATION 7.88 • n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
60 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
55 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
12 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
44 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
4 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: From Visit 2 (21 days after screening) to Visit 5 (50-55 exposure day)Population: Analysis was based on participants who completed the main phase of the trial.
The incidence rate (percentage of participants with inhibitors) of inhibitors defined as inhibitor titres ≥0.6 BU for main phase of the trial.
Outcome measures
| Measure |
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=58 Participants
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Extension Phase (Preventive Treatment)
Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).
|
Turoctocog Alfa (N8): Combined-Main+Ext, Preventive Treatment
Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
|
Turoctocog Alfa (N8): Inhibitor Cohort
Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.
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|---|---|---|---|---|---|
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Incidence Rate of Factor VIII Inhibitors (Above or Equal to 0.6 BU (Bethesda Units)/mL) for the Main Phase of the Trial
|
43.1 Percentage of participants
Interval 30.2 to 56.8
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—
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—
|
—
|
—
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SECONDARY outcome
Timeframe: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trialPopulation: The full analysis set included all dosed participants with data after dosing.
The haemostatic effect of turoctocog alfa was summarised by frequency tables containing count of all bleeds and assessed on a predefined four point scale: Excellent, Good, Moderate and None. The analysis was based on the total number of bleeds and their response to treatment. The analysis was performed for the main phase (from Visit 2 to Visit 5 \[50-55 exposure day\], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Outcome measures
| Measure |
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=98 bleeds
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=133 bleeds
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Extension Phase (Preventive Treatment)
n=269 bleeds
Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).
|
Turoctocog Alfa (N8): Combined-Main+Ext, Preventive Treatment
n=402 bleeds
Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
|
Turoctocog Alfa (N8): Inhibitor Cohort
n=179 bleeds
Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.
|
|---|---|---|---|---|---|
|
Haemostatic Effect of Turoctocog Alfa on Treatment of Bleeds Assessed on a Predefined Four Point Scale: Excellent, Good, Moderate and None
Excellent
|
53 bleeds
|
83 bleeds
|
161 bleeds
|
244 bleeds
|
9 bleeds
|
|
Haemostatic Effect of Turoctocog Alfa on Treatment of Bleeds Assessed on a Predefined Four Point Scale: Excellent, Good, Moderate and None
Good
|
36 bleeds
|
29 bleeds
|
73 bleeds
|
102 bleeds
|
19 bleeds
|
|
Haemostatic Effect of Turoctocog Alfa on Treatment of Bleeds Assessed on a Predefined Four Point Scale: Excellent, Good, Moderate and None
Moderate
|
5 bleeds
|
12 bleeds
|
31 bleeds
|
43 bleeds
|
10 bleeds
|
|
Haemostatic Effect of Turoctocog Alfa on Treatment of Bleeds Assessed on a Predefined Four Point Scale: Excellent, Good, Moderate and None
None
|
0 bleeds
|
1 bleeds
|
1 bleeds
|
2 bleeds
|
4 bleeds
|
|
Haemostatic Effect of Turoctocog Alfa on Treatment of Bleeds Assessed on a Predefined Four Point Scale: Excellent, Good, Moderate and None
Missing
|
4 bleeds
|
8 bleeds
|
3 bleeds
|
11 bleeds
|
137 bleeds
|
SECONDARY outcome
Timeframe: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trialPopulation: The full analysis (FAS) set included all dosed participants with data after dosing. Participants in FAS with only one exposure day were excluded from preventive treatment in main when calculating the Poisson estimates of ABR as the small amount of information introduces uncertainty to the estimated ABR.
Annualised bleeding rate defined as number of bleeds in total per patient per year following treatment were estimated by a Poisson model allowing for over-dispersion. The Poisson estimate is presented with a 95% confidence interval (CI). The analysis was performed for the main phase (from Visit 2 to Visit 5 \[50-55 exposure day\], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Outcome measures
| Measure |
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=98 bleeding episodes
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=133 bleeding episodes
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Extension Phase (Preventive Treatment)
n=269 bleeding episodes
Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).
|
Turoctocog Alfa (N8): Combined-Main+Ext, Preventive Treatment
n=402 bleeding episodes
Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
|
Turoctocog Alfa (N8): Inhibitor Cohort
n=179 bleeding episodes
Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.
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|---|---|---|---|---|---|
|
Annualised Bleeding Rate (ABR)
|
4.27 bleeds/patient/year
Interval 3.09 to 5.89
|
5.63 bleeds/patient/year
Interval 4.27 to 7.43
|
3.81 bleeds/patient/year
Interval 2.84 to 5.11
|
4.26 bleeds/patient/year
Interval 3.34 to 5.44
|
6.09 bleeds/patient/year
Interval 3.77 to 9.84
|
SECONDARY outcome
Timeframe: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trialPopulation: Full Analysis Set included all dosed participants with data after dosing. Number of participants analysed=number of participants with bleeding episodes.
The number of injections of turoctocog alfa (N8) required per bleed was calculated as the number of injections of turoctocog alfa used in the time period from start of the bleed to stop of the bleed. The mean number of turoctocog alfa injections required to stop the bleed is presented. The analysis was based on the total number of bleeds. The analysis was performed for the main phase (from Visit 2 to Visit 5 \[50-55 exposure day\], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Outcome measures
| Measure |
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=98 bleeds
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=133 bleeds
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Extension Phase (Preventive Treatment)
n=269 bleeds
Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).
|
Turoctocog Alfa (N8): Combined-Main+Ext, Preventive Treatment
n=402 bleeds
Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
|
Turoctocog Alfa (N8): Inhibitor Cohort
n=179 bleeds
Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.
|
|---|---|---|---|---|---|
|
Number of Turoctocog Alfa (N8) Injections Required Per Bleed
|
2.1 injections/bleed
Standard Deviation 2.18
|
1.8 injections/bleed
Standard Deviation 2.98
|
2.4 injections/bleed
Standard Deviation 3.04
|
2.2 injections/bleed
Standard Deviation 3.03
|
2.8 injections/bleed
Standard Deviation 6.24
|
SECONDARY outcome
Timeframe: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trialPopulation: Full Analysis Set included all dosed participants with data after dosing.
Mean total consumption of turoctocog alfa (N8) used for treatment (includes all injections given: prevention, treatment of bleeds and during surgery) per patient per month. The analysis was performed for the main phase (from Visit 2 to Visit 5 \[50-55 exposure day\], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Outcome measures
| Measure |
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=51 Participants
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=58 Participants
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Extension Phase (Preventive Treatment)
n=49 Participants
Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).
|
Turoctocog Alfa (N8): Combined-Main+Ext, Preventive Treatment
n=59 Participants
Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
|
Turoctocog Alfa (N8): Inhibitor Cohort
n=26 Participants
Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.
|
|---|---|---|---|---|---|
|
Total Consumption of Turoctocog Alfa (N8) Per Patient (Prevention, Treatment of Bleeds and During Surgery) Per Month
|
117.6 IU/kg/month/patient
Standard Deviation 190.7
|
359.8 IU/kg/month/patient
Standard Deviation 323.2
|
476.9 IU/kg/month/patient
Standard Deviation 209.3
|
437.2 IU/kg/month/patient
Standard Deviation 244.4
|
1815.2 IU/kg/month/patient
Standard Deviation 1653.8
|
SECONDARY outcome
Timeframe: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trialPopulation: Full Analysis Set included all dosed participants with data after dosing.
Mean total consumption of turoctocog alfa (N8) used for treatment (includes all injections given: prevention, treatment of bleeds and during surgery) per patient per year. The analysis was performed for the main phase (from Visit 2 to Visit 5 \[50-55 exposure day\], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Outcome measures
| Measure |
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=51 Participants
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=58 Participants
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Extension Phase (Preventive Treatment)
n=49 Participants
Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).
|
Turoctocog Alfa (N8): Combined-Main+Ext, Preventive Treatment
n=59 Participants
Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
|
Turoctocog Alfa (N8): Inhibitor Cohort
n=26 Participants
Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.
|
|---|---|---|---|---|---|
|
Total Consumption of Turoctocog Alfa (N8) Per Patient (Prevention, Treatment of Bleeds and During Surgery) Per Year
|
1410.8 IU/kg/year/patient
Standard Deviation 2288.4
|
4317.2 IU/kg/year/patient
Standard Deviation 3878.9
|
5722.5 IU/kg/year/patient
Standard Deviation 2512.0
|
5245.9 IU/kg/year/patient
Standard Deviation 2933.1
|
21782.8 IU/kg/year/patient
Standard Deviation 19845.6
|
SECONDARY outcome
Timeframe: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trialPopulation: The full analysis set included all dosed participants with data after dosing.
Mean consumption of turoctocog alfa (N8) used for treatment of bleed from start to stop of bleed. The analysis was based on number of bleeds. The analysis was performed for the main phase (from Visit 2 to Visit 5 \[50-55 exposure day\], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Outcome measures
| Measure |
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=98 bleeds
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=133 bleeds
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Extension Phase (Preventive Treatment)
n=269 bleeds
Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).
|
Turoctocog Alfa (N8): Combined-Main+Ext, Preventive Treatment
n=59 Participants
Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
|
Turoctocog Alfa (N8): Inhibitor Cohort
n=179 bleeds
Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.
|
|---|---|---|---|---|---|
|
Consumption of Turoctocog Alfa (N8) (IU/kg/Bleed) Per Bleed
|
86.1 IU/kg/bleed
Standard Deviation 116.8
|
80.8 IU/kg/bleed
Standard Deviation 126.4
|
107.9 IU/kg/bleed
Standard Deviation 137.7
|
98.9 IU/kg/bleed
Standard Deviation 134.5
|
279.1 IU/kg/bleed
Standard Deviation 821.7
|
SECONDARY outcome
Timeframe: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trialPopulation: The full analysis set included all dosed participants with data after dosing. Participants in on-demand treatment did not receive treatment for bleed prevention and therefore are not included in the analysis.
Mean consumption of turoctocog alfa (N8) used for preventive treatment per month per patient. The analysis was performed for the main phase (from Visit 2 to Visit 5 \[50-55 exposure day\], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Outcome measures
| Measure |
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=58 Participants
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Extension Phase (Preventive Treatment)
n=49 Participants
Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).
|
Turoctocog Alfa (N8): Combined-Main+Ext, Preventive Treatment
n=59 Participants
Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
|
Turoctocog Alfa (N8): Inhibitor Cohort
n=26 Participants
Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.
|
|---|---|---|---|---|---|
|
Consumption of Turoctocog Alfa (N8) (IU/kg/Months) for Bleed Prevention
|
—
|
293.5 IU/kg/month/patient
Standard Deviation 281.1
|
446.3 IU/kg/month/patient
Standard Deviation 206.2
|
399.0 IU/kg/month/patient
Standard Deviation 234.7
|
1692.6 IU/kg/month/patient
Standard Deviation 1553.9
|
SECONDARY outcome
Timeframe: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trialPopulation: The safety analysis set included all dosed participants with data after dosing. The arm group - 'main phase' includes subjects who were treated both on-demand and preventively in the main phase. 'On-Demand' and 'Preventive' were not described as separate arms/groups in the protocol. These were not analysed separately for this endpoint.
Number of adverse events and serious adverse events per patient years of exposure. The analysis was performed for the main phase (from Visit 2 to Visit 5 \[50-55 exposure day\], extension phase (from Visit 6 to end of trial (exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Outcome measures
| Measure |
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=60 Participants
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=49 Participants
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Extension Phase (Preventive Treatment)
n=60 Participants
Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).
|
Turoctocog Alfa (N8): Combined-Main+Ext, Preventive Treatment
n=26 Participants
Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
|
Turoctocog Alfa (N8): Inhibitor Cohort
Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.
|
|---|---|---|---|---|---|
|
Frequency of Adverse Events (AEs) and Serious Adverse Events (SAEs) Reported During the Trial Period
All adverse events
|
5.85 events per patient years of exposure
|
3.76 events per patient years of exposure
|
5.12 events per patient years of exposure
|
7.41 events per patient years of exposure
|
—
|
|
Frequency of Adverse Events (AEs) and Serious Adverse Events (SAEs) Reported During the Trial Period
Serious adverse events
|
1.02 events per patient years of exposure
|
0.24 events per patient years of exposure
|
0.73 events per patient years of exposure
|
1.52 events per patient years of exposure
|
—
|
SECONDARY outcome
Timeframe: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trialPopulation: The full analysis set included all dosed participants with data after dosing. The arm group - 'main phase' includes subjects who were treated both on-demand and preventively in the main phase. 'On-Demand' and 'Preventive' were not described as separate arms/groups in the protocol. These were not analysed separately for this endpoint.
The incidence rates (number of patients with new inhibitor in the period/number of participants at risk, expressed in percentage) of clinically relevant inhibitors were defined as an inhibitor titre (≥ 0.6 BU) combined with a decreased recovery (\<66% of expected level). Incidence rate of clinically relevant inhibitors according to the type of assay used is presented. The analysis was performed for the main phase (from Visit 2 to Visit 5 \[50-55 exposure day\], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation) and the combined main and extension phase (from Visit 2 to end of trial).
Outcome measures
| Measure |
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=58 Participants
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=33 Participants
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Extension Phase (Preventive Treatment)
n=58 Participants
Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).
|
Turoctocog Alfa (N8): Combined-Main+Ext, Preventive Treatment
Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
|
Turoctocog Alfa (N8): Inhibitor Cohort
Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.
|
|---|---|---|---|---|---|
|
Incidence Rate of Clinically Relevant Inhibitors Defined as an Inhibitor Titre (≥ 0.6 BU/mL) Combined With a Decreased Recovery (<66% of Expected Level)
One-stage FVIII clotting activity assay
|
36.2 Percentage of participants
Interval 24.0 to 49.9
|
3.0 Percentage of participants
Interval 0.1 to 15.8
|
37.9 Percentage of participants
Interval 25.5 to 51.6
|
—
|
—
|
|
Incidence Rate of Clinically Relevant Inhibitors Defined as an Inhibitor Titre (≥ 0.6 BU/mL) Combined With a Decreased Recovery (<66% of Expected Level)
Two-stage chromogenic assay
|
36.2 Percentage of participants
Interval 24.0 to 49.9
|
3.0 Percentage of participants
Interval 0.1 to 15.8
|
37.9 Percentage of participants
Interval 25.5 to 51.6
|
—
|
—
|
SECONDARY outcome
Timeframe: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trialPopulation: The full analysis set included all dosed participants with data after dosing. The arm group - 'main phase' includes subjects who were treated both on-demand and preventively in the main phase. 'On-Demand' and 'Preventive' were not described as separate arms/groups in the protocol. These were not analysed separately for this endpoint.
Incidence rate (number of patients with new inhibitor in the period/number of participants at risk, expressed in percentage) of high-titre inhibitors defined as inhibitor titre ≥ 5 BU (Bethesda Units)/mL). The inhibitors were evaluated for the main phase (from Visit 2 to Visit 5 \[50-55 exposure day\], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation) and the combined main and extension phase (from Visit 2 to end of trial).
Outcome measures
| Measure |
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=58 Participants
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=33 Participants
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Extension Phase (Preventive Treatment)
n=58 Participants
Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).
|
Turoctocog Alfa (N8): Combined-Main+Ext, Preventive Treatment
Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
|
Turoctocog Alfa (N8): Inhibitor Cohort
Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.
|
|---|---|---|---|---|---|
|
Incidence Rate of High-titre Inhibitors Defined as Inhibitor Titre ≥ 5 BU (Bethesda Units)/mL)
|
27.6 Percentage of participants
Interval 16.7 to 40.9
|
0.0 Percentage of participants
Interval 0.0 to 10.6
|
27.6 Percentage of participants
Interval 16.7 to 40.9
|
—
|
—
|
SECONDARY outcome
Timeframe: Visit 3 (10th-15th ED); Visit 5 (50th-55th ED); End of trial (within 8 weeks of their last scheduled visit in the extension phase)Population: The full analysis set included all dosed participants with data after dosing. Number of participants analysed=number of participants who answered the questionnaire
The caregivers/parent reported treatment satisfaction is assessed by the parents using the haemophilia satisfaction questionnaire (HEMO-SAT). The questionnaire contained questions related to treatment that covered 6 domains (ease and convenience, efficacy, burden, specialist, centre and general satisfaction). Adults completing the questionnaire could achieve a score from 0 to 100, with lower scores reflecting greater treatment satisfaction. The scale range for each of the 6 domains was 0-100 with lower scores reflecting greater treatment satisfaction. The scores of the domains at visit 3 (10th-15th ED), visit 5 (50th-55th ED) and end of trial (EoT) are presented.
Outcome measures
| Measure |
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=59 Participants
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Extension Phase (Preventive Treatment)
Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).
|
Turoctocog Alfa (N8): Combined-Main+Ext, Preventive Treatment
Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
|
Turoctocog Alfa (N8): Inhibitor Cohort
Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.
|
|---|---|---|---|---|---|
|
Change in Total Scores for Parent Reported Treatment Satisfaction
Ease/convenience visit 3 Range: 0 - 100
|
27.1 Score on a scale
Standard Deviation 14.6
|
—
|
—
|
—
|
—
|
|
Change in Total Scores for Parent Reported Treatment Satisfaction
Ease and convenience - visit 5 Score range: 0-100
|
21.4 Score on a scale
Standard Deviation 14.9
|
—
|
—
|
—
|
—
|
|
Change in Total Scores for Parent Reported Treatment Satisfaction
Ease and convenience - EoT Score range:0-100
|
21.9 Score on a scale
Standard Deviation 13.4
|
—
|
—
|
—
|
—
|
|
Change in Total Scores for Parent Reported Treatment Satisfaction
Efficacy - visit 3 Score range: 0-100
|
10.2 Score on a scale
Standard Deviation 12.8
|
—
|
—
|
—
|
—
|
|
Change in Total Scores for Parent Reported Treatment Satisfaction
Efficacy - visit 5 Score range: 0-100
|
9.8 Score on a scale
Standard Deviation 10.5
|
—
|
—
|
—
|
—
|
|
Change in Total Scores for Parent Reported Treatment Satisfaction
Efficacy - end of trial Score range: 0-100
|
11.7 Score on a scale
Standard Deviation 17.4
|
—
|
—
|
—
|
—
|
|
Change in Total Scores for Parent Reported Treatment Satisfaction
Burden - visit 3 Score range: 0-100
|
20.5 Score on a scale
Standard Deviation 21.0
|
—
|
—
|
—
|
—
|
|
Change in Total Scores for Parent Reported Treatment Satisfaction
Burden - visit 5 Score range: 0-100
|
15.4 Score on a scale
Standard Deviation 17.4
|
—
|
—
|
—
|
—
|
|
Change in Total Scores for Parent Reported Treatment Satisfaction
Burden - end of trial Score range: 0-100
|
16.9 Score on a scale
Standard Deviation 16.6
|
—
|
—
|
—
|
—
|
|
Change in Total Scores for Parent Reported Treatment Satisfaction
Specialist/Nurses- visit 3 Score range: 0-100
|
4.1 Score on a scale
Standard Deviation 8.4
|
—
|
—
|
—
|
—
|
|
Change in Total Scores for Parent Reported Treatment Satisfaction
Specialist/Nurses- visit 5 Score range: 0-100
|
4.0 Score on a scale
Standard Deviation 6.6
|
—
|
—
|
—
|
—
|
|
Change in Total Scores for Parent Reported Treatment Satisfaction
Specialist/Nurses- end of trial Score range: 0-100
|
4.9 Score on a scale
Standard Deviation 8.2
|
—
|
—
|
—
|
—
|
|
Change in Total Scores for Parent Reported Treatment Satisfaction
Centre/Hospital - visit 3 Score range: 0-100
|
2.8 Score on a scale
Standard Deviation 5.7
|
—
|
—
|
—
|
—
|
|
Change in Total Scores for Parent Reported Treatment Satisfaction
Centre/Hospital - visit 5 Score range: 0-100
|
3.1 Score on a scale
Standard Deviation 5.7
|
—
|
—
|
—
|
—
|
|
Change in Total Scores for Parent Reported Treatment Satisfaction
Centre/Hospital - end of trial Score range: 0-100
|
5.0 Score on a scale
Standard Deviation 8.9
|
—
|
—
|
—
|
—
|
|
Change in Total Scores for Parent Reported Treatment Satisfaction
General satisfaction - visit 3 Score range: 0-100
|
5.7 Score on a scale
Standard Deviation 10.7
|
—
|
—
|
—
|
—
|
|
Change in Total Scores for Parent Reported Treatment Satisfaction
General satisfaction - visit 5 Score range: 0-100
|
3.8 Score on a scale
Standard Deviation 8.5
|
—
|
—
|
—
|
—
|
|
Change in Total Scores for Parent Reported Treatment Satisfaction
General satisfaction - end of trial Range: 0-100
|
6.4 Score on a scale
Standard Deviation 15.3
|
—
|
—
|
—
|
—
|
SECONDARY outcome
Timeframe: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trialPopulation: The full analysis set included all dosed participants with data after dosing. Number of participants analysed=number of participants with data available
Resource utilisation and caregiver burden are analysed in terms of average number of days absent from work and use of mobility aids (e.g. wheelchair or crutches) as a consequence of bleeds. The analysis was performed for the main phase (from Visit 2 to Visit 5 \[50-55 exposure day\], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Outcome measures
| Measure |
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=51 Participants
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=58 Participants
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Extension Phase (Preventive Treatment)
n=49 Participants
Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).
|
Turoctocog Alfa (N8): Combined-Main+Ext, Preventive Treatment
n=59 Participants
Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
|
Turoctocog Alfa (N8): Inhibitor Cohort
n=26 Participants
Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.
|
|---|---|---|---|---|---|
|
Health Resource Utilization and Caregiver Burden Associated With Bleeds (Per Month Per Patient)
Caregiver/parent's absence from work
|
0.011 days/month/patient
Standard Deviation 0.050
|
0.533 days/month/patient
Standard Deviation 1.645
|
0.007 days/month/patient
Standard Deviation 0.027
|
0.232 days/month/patient
Standard Deviation 1.117
|
0.271 days/month/patient
Standard Deviation 0.641
|
|
Health Resource Utilization and Caregiver Burden Associated With Bleeds (Per Month Per Patient)
Patient's use of mobility aids
|
0.0 days/month/patient
Standard Deviation 0.0
|
0.0 days/month/patient
Standard Deviation 0.0
|
0.0 days/month/patient
Standard Deviation 0.0
|
0.0 days/month/patient
Standard Deviation 0.0
|
0.025 days/month/patient
Standard Deviation 0.084
|
SECONDARY outcome
Timeframe: From Visit 2 to Visit 5 (50-55 exposure day); From Visit 6 to end of trial, extension phase of the trial; From Visit 2 to end of trial, the combined main and extension phases of the trialPopulation: The full analysis set included all dosed participants with data after dosing. Number of participants analysed=number of participants with data available.
Resource utilisation and caregiver burden are analysed in terms of average number of days of absence from work and use of mobility aids (e.g. wheelchair or crutches) as a consequence of bleeds. The analysis was performed for the main phase (from Visit 2 to Visit 5 \[50-55 exposure day\], extension phase (from Visit 6 to end of trial; exposure day 100, expected to occur between 6 and 48 months of trial participation), the combined main and extension phase (from Visit 2 to end of trial) and for the inhibitor cohort.
Outcome measures
| Measure |
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=51 Participants
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Main Phase (Preventive Treatment)
n=58 Participants
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Subjects received prophylactic factor VIII replacement therapy (preventive treatment) until Visit 5 or until development of inhibitor, whichever came first. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors.
|
Turoctocog Alfa (N8): Extension Phase (Preventive Treatment)
n=49 Participants
Participants received prophylactic treatment administered from completion of the main phase or completion of the inhibitor cohort until the end-of-trial visit in the extension phase. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. After completing the main phase, participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment).
|
Turoctocog Alfa (N8): Combined-Main+Ext, Preventive Treatment
n=59 Participants
Participants who started preventive treatment in the main and extension phase are included in this arm. They received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the child's clinical profile. The trial consisted of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reached a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which translated into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
|
Turoctocog Alfa (N8): Inhibitor Cohort
n=26 Participants
Participants who developed an inhibitor during the course of the trial and followed an alternative treatment regimen are included in this arm. They were offered continued treatment with turoctocog alfa for up to 24 months.
|
|---|---|---|---|---|---|
|
Health Resource Utilization and Caregiver Burden Associated With Bleeds (Per Year Per Patient)
Caregiver/parent's absence from work
|
0.127 days/year/patient
Standard Deviation 0.601
|
6.396 days/year/patient
Standard Deviation 19.736
|
0.082 days/year/patient
Standard Deviation 0.319
|
2.779 days/year/patient
Standard Deviation 13.406
|
3.252 days/year/patient
Standard Deviation 7.691
|
|
Health Resource Utilization and Caregiver Burden Associated With Bleeds (Per Year Per Patient)
Patient's use of mobility aids
|
0.0 days/year/patient
Standard Deviation 0.0
|
0.0 days/year/patient
Standard Deviation 0.0
|
0.0 days/year/patient
Standard Deviation 0.0
|
0.0 days/year/patient
Standard Deviation 0.0
|
0.296 days/year/patient
Standard Deviation 1.006
|
Adverse Events
Turoctocog Alfa
Serious events: 36 serious events
Other events: 55 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Turoctocog Alfa
n=60 participants at risk
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Participants who enrolled into the trial initiated the preventive treatment no later than their second birthday or after a maximum of 2 bleeding episodes requiring treatment, whichever came first. The trial consists of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reach a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which will translate into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
|
|---|---|
|
Injury, poisoning and procedural complications
Accidental overdose
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Blood and lymphatic system disorders
Anaemia
|
5.0%
3/60 • Number of events 3 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Bacteraemia
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Bronchitis
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
General disorders
Catheter site haematoma
|
3.3%
2/60 • Number of events 2 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Catheter site infection
|
3.3%
2/60 • Number of events 6 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
General disorders
Catheter site oedema
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Surgical and medical procedures
Central venous catheter removal
|
1.7%
1/60 • Number of events 2 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Surgical and medical procedures
Central venous catheterisation
|
3.3%
2/60 • Number of events 2 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Surgical and medical procedures
Circumcision
|
3.3%
2/60 • Number of events 2 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Injury, poisoning and procedural complications
Concussion
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Congenital, familial and genetic disorders
Cryptorchism
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Product Issues
Device damage
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Product Issues
Device dislocation
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Device related infection
|
5.0%
3/60 • Number of events 5 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Immune system disorders
Drug hypersensitivity
|
3.3%
2/60 • Number of events 2 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Blood and lymphatic system disorders
Factor VIII inhibition
|
41.7%
25/60 • Number of events 27 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Gastrointestinal disorders
Gastritis
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Gastroenteritis
|
3.3%
2/60 • Number of events 3 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Gastrointestinal infection
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Vascular disorders
Haematoma
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Nervous system disorders
Haemorrhage intracranial
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Injury, poisoning and procedural complications
Head injury
|
5.0%
3/60 • Number of events 3 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Surgical and medical procedures
Hospitalisation
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Congenital, familial and genetic disorders
Hydrocele
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Injection site infection
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Nervous system disorders
Intracranial haematoma
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Musculoskeletal and connective tissue disorders
Joint effusion
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Gastrointestinal disorders
Mouth haemorrhage
|
1.7%
1/60 • Number of events 2 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Nasopharyngitis
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Otitis media acute
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Vascular disorders
Phlebitis deep
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Vascular disorders
Poor venous access
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
General disorders
Pyrexia
|
6.7%
4/60 • Number of events 4 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Respiratory tract infection
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Staphylococcal bacteraemia
|
3.3%
2/60 • Number of events 3 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Staphylococcal infection
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Respiratory, thoracic and mediastinal disorders
Status asthmaticus
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Streptococcal bacteraemia
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Systemic bacterial infection
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Injury, poisoning and procedural complications
Traumatic haemorrhage
|
6.7%
4/60 • Number of events 5 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Upper respiratory tract infection
|
3.3%
2/60 • Number of events 2 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Vascular device infection
|
3.3%
2/60 • Number of events 2 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Viral infection
|
1.7%
1/60 • Number of events 1 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
Other adverse events
| Measure |
Turoctocog Alfa
n=60 participants at risk
Participants received turoctocog alfa doses of 15-60 IU/kg body weight (BW) administered as an intravenous injection for the prevention of bleeding. The investigator decided what dose of turoctocog alfa to give based on the participant's clinical profile. Participants who enrolled into the trial initiated the preventive treatment no later than their second birthday or after a maximum of 2 bleeding episodes requiring treatment, whichever came first. The trial consists of two phases. In the main phase, an aggregated number of at least 50 participants received preventive treatment with turoctocog alfa until they reach a minimum of 50 exposure days (ED) or until they developed inhibitors. After that the participants continued in the extension phase where at least 50 participants achieved at least 100 ED, which will translate into a maximum trial duration of up to 5 years (including any potential inhibitor treatment). The estimated total duration of the trial was approximately 7 years.
|
|---|---|
|
Blood and lymphatic system disorders
Anaemia
|
10.0%
6/60 • Number of events 8 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
6.7%
4/60 • Number of events 5 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Reproductive system and breast disorders
Balanoposthitis
|
5.0%
3/60 • Number of events 3 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Investigations
Body temperature increased
|
5.0%
3/60 • Number of events 4 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Bronchitis
|
11.7%
7/60 • Number of events 7 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Catheter site infection
|
5.0%
3/60 • Number of events 7 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
General disorders
Catheter site inflammation
|
5.0%
3/60 • Number of events 3 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Conjunctivitis
|
5.0%
3/60 • Number of events 3 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Gastrointestinal disorders
Constipation
|
5.0%
3/60 • Number of events 3 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Injury, poisoning and procedural complications
Contusion
|
10.0%
6/60 • Number of events 9 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
25.0%
15/60 • Number of events 26 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
5.0%
3/60 • Number of events 3 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Gastrointestinal disorders
Diarrhoea
|
20.0%
12/60 • Number of events 25 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Ear infection
|
10.0%
6/60 • Number of events 10 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
8.3%
5/60 • Number of events 6 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Blood and lymphatic system disorders
Factor VIII inhibition
|
6.7%
4/60 • Number of events 4 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Gastroenteritis
|
13.3%
8/60 • Number of events 8 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Gastroenteritis viral
|
5.0%
3/60 • Number of events 4 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Hand-foot-and-mouth disease
|
6.7%
4/60 • Number of events 5 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Injury, poisoning and procedural complications
Head injury
|
8.3%
5/60 • Number of events 9 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Influenza
|
8.3%
5/60 • Number of events 5 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Metabolism and nutrition disorders
Iron deficiency
|
6.7%
4/60 • Number of events 4 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
13.3%
8/60 • Number of events 12 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
6.7%
4/60 • Number of events 6 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Nasopharyngitis
|
26.7%
16/60 • Number of events 36 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Otitis media
|
6.7%
4/60 • Number of events 4 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Otitis media acute
|
6.7%
4/60 • Number of events 7 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Pharyngitis
|
6.7%
4/60 • Number of events 4 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Pneumonia
|
8.3%
5/60 • Number of events 5 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
General disorders
Pyrexia
|
53.3%
32/60 • Number of events 85 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Skin and subcutaneous tissue disorders
Rash
|
8.3%
5/60 • Number of events 11 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Skin and subcutaneous tissue disorders
Rash papular
|
5.0%
3/60 • Number of events 3 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
11.7%
7/60 • Number of events 10 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Gastrointestinal disorders
Teething
|
5.0%
3/60 • Number of events 3 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Tonsillitis
|
5.0%
3/60 • Number of events 6 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Upper respiratory tract infection
|
25.0%
15/60 • Number of events 64 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Varicella
|
5.0%
3/60 • Number of events 3 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Viral infection
|
11.7%
7/60 • Number of events 9 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
6.7%
4/60 • Number of events 11 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
|
Gastrointestinal disorders
Vomiting
|
18.3%
11/60 • Number of events 19 • Adverse events from the first trial related activity after the participant had signed the informed consent (visit 1) until the end-of-trial visit (within 8 weeks of their last scheduled visit in the extension phase; 100th exposure day).
Reported adverse events were based on the safety analysis set including all dosed participants with data after dosing.
|
Additional Information
Clinical Reporting Anchor and Disclosure (1452)
Novo Nordisk A/S
Phone: (+1) 866-867-7178
Email: [email protected]
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