| Primary | Incidence of Inhibitory Antibodies Against Factor IX Defined as Titre Equal to or Above 0.6 BU (Bethesda Units) | Inhibitors were analysed with either the Nijmegen modified factor IX Bethesda assay or a heat/cold Nijmegen modified factor IX Bethesda assay. Number of subjects who developed inhibitory antibodies against factor IX are reported. | Full analysis set included all subjects exposed to nonacog beta pegol. Subjects in prophylaxis arm were included for this analysis. | Posted | | Number | | Number of subjects | | 52 weeks after treatment start for patients on prophylaxis | | | | ID | Title | Description |
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| OG000 | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | Subjects were randomised to either prophylaxis 10 U/kg or 40 U/kg arm. Subjects in this arm received 10 U/kg nonacog beta pegol dose once weekly (every 7th day ± 24 hours) for a period of 52 weeks. Nonacog beta pegol was given as an intravenous bolus injection with the maximum injection rate at 4 mL/min. Mild and moderate bleeding episodes were treated with 40 U/kg. Severe bleeding episodes were treated with 80 U/kg. | | OG001 | Prophylaxis, High Dose 40 U/kg (52 Weeks) | Subjects were randomised to either prophylaxis 10 U/kg or 40 U/kg arm. Subjects in this arm received 40 U/kg nonacog beta pegol dose once weekly (every 7th day ± 24 hours) for a period of 52 weeks. Nonacog beta pegol was given as an intravenous bolus injection with the maximum injection rate at 4 mL/min. Mild and moderate bleeding episodes were treated with 40 U/kg. Severe bleeding episodes were treated with 80 U/kg. |
| | | Title | Denominators | Categories |
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| | | Title | Measurements |
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| - OG0000(0 to 0)
- OG0010(0 to 0)
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| Primary | Incidence of Inhibitory Antibodies Against Factor IX Defined as Titre Equal to or Above 0.6 BU (Bethesda Units) | Inhibitors were analysed with either the Nijmegen modified factor IX Bethesda assay or a heat/cold Nijmegen modified factor IX Bethesda assay. Number of subjects who developed inhibitory antibodies against factor IX are reported. | Full analysis set included all subjects exposed to nonacog beta pegol. Subjects in on-demand arm were included for this analysis. | Posted | | Number | | number of subjects | | 28 weeks after treatment start on on-demand treatment | | | | ID | Title | Description |
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| OG000 | On-Demand (28 Weeks) | Subjects enrolled in this on-demand arm were treated for bleeding episodes on demand with nonacog beta pegol during a period of 28 weeks. Nonacog beta pegol was given as an intravenous bolus injection with the maximum injection rate at 4 mL/min. Mild and moderate bleeding episodes were treated with 40 U/kg. Severe bleeding episodes were treated with 80 U/kg. |
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| Secondary | Haemostatic Effect of NNC-0156-0000-0009 When Used for Prophylaxis of Bleeding Episodes, Assessed as Success/Failure Based on a Four-point Scale for Haemostatic Response | Haemostatic response after treatment of a bleed with nonacog beta pegol was evaluated on a 4 point scale as excellent, good, moderate or poor. A success rate was calculated based on counting good or excellent as successes and poor and moderate as failures.
- Excellent - abrupt pain relief and/or clear improvement in objective signs of bleeding within 8 hours after a single injection
- Good - noticeable pain relief and/or improvement in signs of bleeding within 8 hours after a single injection
- Moderate - probable or slight beneficial effect within the first 8 hours after the first injection but requiring more than one injection within 8 hours
- Poor - no improvement, or worsening of symptoms within 8 hours after two injections.
The success rate and 95% confidence interval (CI) are reported here. | Full analysis set included all subjects exposed to nonacog beta pegol. Subjects in prophylaxis arm were included for this analysis. | Posted | | Number | 95% Confidence Interval | percentage of bleeding episodes | | 52 weeks after treatment start for patients on prophylaxis | | | | ID | Title | Description |
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| OG000 | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | Subjects were randomised to either prophylaxis 10 U/kg or 40 U/kg arm. Subjects in this arm received 10 U/kg nonacog beta pegol dose once weekly (every 7th day ± 24 hours) for a period of 52 weeks. Nonacog beta pegol was given as an intravenous bolus injection with the maximum injection rate at 4 mL/min. Mild and moderate bleeding episodes were treated with 40 U/kg. Severe bleeding episodes were treated with 80 U/kg. |
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| Secondary | Haemostatic Effect of NNC-0156-0000-0009 When Used for Treatment of Bleeding Episodes, Assessed as Success/Failure Based on a Four-point Scale for Haemostatic Response | Haemostatic response after treatment of a bleed with nonacog beta pegol was evaluated on a 4 point scale as excellent, good, moderate or poor. A success rate was calculated based on counting good or excellent as successes and poor and moderate as failures.
- Excellent - abrupt pain relief and/or clear improvement in objective signs of bleeding within 8 hours after a single injection
- Good - noticeable pain relief and/or improvement in signs of bleeding within 8 hours after a single injection
- Moderate - probable or slight beneficial effect within the first 8 hours after the first injection but requiring more than one injection within 8 hours
- Poor - no improvement, or worsening of symptoms within 8 hours after two injections.
The success rate and 95% confidence interval (CI) are reported here. | Full analysis set included all subjects exposed to nonacog beta pegol. Subjects in on-demand arm were included for this analysis. | Posted | | Number | 95% Confidence Interval | percentage of bleeding episodes | | 28 weeks after treatment start on on-demand treatment | | | | ID | Title | Description |
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| OG000 | On-Demand (28 Weeks) | Subjects enrolled in this on-demand arm were treated for bleeding episodes on demand with nonacog beta pegol during a period of 28 weeks. Nonacog beta pegol was given as an intravenous bolus injection with the maximum injection rate at 4 mL/min. Mild and moderate bleeding episodes were treated with 40 U/kg. Severe bleeding episodes were treated with 80 U/kg. |
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| Secondary | Number of Bleeding Episodes Per Patient During Routine Prophylaxis | The number of bleeding episodes per patient during routine prophylaxis was assessed using the individual annualised bleeding rates (spontaneous and traumatic bleeding episodes per patient per year). | Full analysis set included all subjects exposed to nonacog beta pegol. Subjects in prophylaxis arm were included for this analysis. | Posted | | Median | Inter-Quartile Range | bleeds/patient/year | | 52 weeks after treatment start for patients on prophylaxis | | | | ID | Title | Description |
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| OG000 | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | Subjects were randomised to either prophylaxis 10 U/kg or 40 U/kg arm. Subjects in this arm received 10 U/kg nonacog beta pegol dose once weekly (every 7th day ± 24 hours) for a period of 52 weeks. Nonacog beta pegol was given as an intravenous bolus injection with the maximum injection rate at 4 mL/min. Mild and moderate bleeding episodes were treated with 40 U/kg. Severe bleeding episodes were treated with 80 U/kg. | | OG001 | Prophylaxis, High Dose 40 U/kg (52 Weeks) | Subjects were randomised to either prophylaxis 10 U/kg or 40 U/kg arm. Subjects in this arm received 40 U/kg nonacog beta pegol dose once weekly (every 7th day ± 24 hours) for a period of 52 weeks. Nonacog beta pegol was given as an intravenous bolus injection with the maximum injection rate at 4 mL/min. Mild and moderate bleeding episodes were treated with 40 U/kg. Severe bleeding episodes were treated with 80 U/kg. |
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| Secondary | Factor IX Trough Levels | The mean pre-dose factor IX levels was measured with the one-stage clotting assay during the trial. Lowest factor IX activity recorded during single-dose and steady state, immediately before next dose was given. The analysis was based on a mixed model on the log-transformed plasma factor IX activity with subject as a random effect. The estimated mean factor IX trough level was presented back-transformed to the natural scale. | Full analysis set included all subjects exposed to nonacog beta pegol. Subjects in prophylaxis arm were included for this analysis. | Posted | | Mean | 95% Confidence Interval | U/mL | | 52 weeks after treatment start for patients on prophylaxis | | | | ID | Title | Description |
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| OG000 | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | Subjects were randomised to either prophylaxis 10 U/kg or 40 U/kg arm. Subjects in this arm received 10 U/kg nonacog beta pegol dose once weekly (every 7th day ± 24 hours) for a period of 52 weeks. Nonacog beta pegol was given as an intravenous bolus injection with the maximum injection rate at 4 mL/min. Mild and moderate bleeding episodes were treated with 40 U/kg. Severe bleeding episodes were treated with 80 U/kg. | | OG001 | Prophylaxis, High Dose 40 U/kg (52 Weeks) | Subjects were randomised to either prophylaxis 10 U/kg or 40 U/kg arm. Subjects in this arm received 40 U/kg nonacog beta pegol dose once weekly (every 7th day ± 24 hours) for a period of 52 weeks. Nonacog beta pegol was given as an intravenous bolus injection with the maximum injection rate at 4 mL/min. Mild and moderate bleeding episodes were treated with 40 U/kg. Severe bleeding episodes were treated with 80 U/kg. |
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| Secondary | Incidence of Adverse Events (AEs) | The incidence of adverse events were summarised by the rate of AEs (number of AEs per patient years of exposure [PYE]). Number of adverse events per PYE is number of adverse events /total time in trial. All adverse events reported are treatment emergent (any adverse events which occurred after trial product administration). | Safety analysis set included all subjects exposed to nonacog beta pegol. Subjects in prophylaxis arm were included for this analysis. | Posted | | Number | | number of AEs per PYE | | at 56 weeks ±2 weeks for patients on prophylaxis | | | | ID | Title | Description |
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| OG000 | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | Subjects were randomised to either prophylaxis 10 U/kg or 40 U/kg arm. Subjects in this arm received 10 U/kg nonacog beta pegol dose once weekly (every 7th day ± 24 hours) for a period of 52 weeks. Nonacog beta pegol was given as an intravenous bolus injection with the maximum injection rate at 4 mL/min. Mild and moderate bleeding episodes were treated with 40 U/kg. Severe bleeding episodes were treated with 80 U/kg. | | OG001 | Prophylaxis, High Dose 40 U/kg (52 Weeks) | Subjects were randomised to either prophylaxis 10 U/kg or 40 U/kg arm. Subjects in this arm received 40 U/kg nonacog beta pegol dose once weekly (every 7th day ± 24 hours) for a period of 52 weeks. Nonacog beta pegol was given as an intravenous bolus injection with the maximum injection rate at 4 mL/min. Mild and moderate bleeding episodes were treated with 40 U/kg. Severe bleeding episodes were treated with 80 U/kg. |
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| Secondary | Incidence of Adverse Events (AEs) | The incidence of adverse events were summarised by the rate of AEs (number of AEs per PYE). Number of adverse events per PYE is number of adverse events /total time in trial. All adverse events reported are treatment emergent (any adverse events which occurred after trial product administration). | Safety analysis set included all subjects exposed to nonacog beta pegol.Subjects in on-demand arm were included for this analysis. | Posted | | Number | | number of AEs per PYE | | at 32 weeks ±2 weeks for patients on on-demand treatment | | | | ID | Title | Description |
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| OG000 | On-Demand (28 Weeks) | Subjects enrolled in this on-demand arm were treated for bleeding episodes on demand with nonacog beta pegol during a period of 28 weeks. Nonacog beta pegol was given as an intravenous bolus injection with the maximum injection rate at 4 mL/min. Mild and moderate bleeding episodes were treated with 40 U/kg. Severe bleeding episodes were treated with 80 U/kg. |
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| Secondary | Incidence of Serious Adverse Events (SAEs) | SAE was defined as an AE that resulted in any of the following: death, a life-threatening experience, in-subject hospitalization/prolongation of existing hospitalisation, persistent/significant disability/incapacity/congenital anomaly/birth defect. The incidence of SAEs were summarised by the rate of SAEs (number of SAEs per PYE). Number of SAEs per PYE is number of SAEs/total time in trial. All SAEs reported are treatment emergent (any serious adverse events which occurred after trial product administration). | Safety analysis set included all subjects exposed to nonacog beta pegol. Subjects in prophylaxis arm were included for this analysis. | Posted | | Number | | number of SAEs per PYE | | at 56 weeks ±2 weeks for patients on prophylaxis | | | | ID | Title | Description |
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| OG000 | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | Subjects were randomised to either prophylaxis 10 U/kg or 40 U/kg arm. Subjects in this arm received 10 U/kg nonacog beta pegol dose once weekly (every 7th day ± 24 hours) for a period of 52 weeks. Nonacog beta pegol was given as an intravenous bolus injection with the maximum injection rate at 4 mL/min. Mild and moderate bleeding episodes were treated with 40 U/kg. Severe bleeding episodes were treated with 80 U/kg. | | OG001 | Prophylaxis, High Dose 40 U/kg (52 Weeks) | Subjects were randomised to either prophylaxis 10 U/kg or 40 U/kg arm. Subjects in this arm received 40 U/kg nonacog beta pegol dose once weekly (every 7th day ± 24 hours) for a period of 52 weeks. Nonacog beta pegol was given as an intravenous bolus injection with the maximum injection rate at 4 mL/min. Mild and moderate bleeding episodes were treated with 40 U/kg. Severe bleeding episodes were treated with 80 U/kg. |
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| Secondary | Incidence of Serious Adverse Events (SAEs) | SAE was defined as an AE that resulted in any of the following: death, a life-threatening experience, in-subject hospitalization/prolongation of existing hospitalisation, persistent/significant disability/incapacity/congenital anomaly/birth defect. The incidence of SAEs were summarised by the rate of SAEs (number of SAEs per PYE). Number of SAEs per PYE is number of SAEs/total time in trial. All SAEs reported are treatment emergent (any serious adverse events which occurred after trial product administration). | Safety analysis set included all subjects exposed to nonacog beta pegol. Subjects in on-demand arm were included for this analysis. | Posted | | Number | | number of SAEs per PYE | | at 32 weeks ±2 weeks for patients on on-demand treatment | | | | ID | Title | Description |
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| OG000 | On-Demand (28 Weeks) | Subjects enrolled in this on-demand arm were treated for bleeding episodes on demand with nonacog beta pegol during a period of 28 weeks. Nonacog beta pegol was given as an intravenous bolus injection with the maximum injection rate at 4 mL/min. Mild and moderate bleeding episodes were treated with 40 U/kg. Severe bleeding episodes were treated with 80 U/kg. |
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| Secondary | Host Cell Proteins (HCP) Antibodies | Subjects who were positive for anti-Host Cell Protein (HCP) antibodies. | Safety analysis set included all subjects exposed to nonacog beta pegol. Subjects in prophylaxis arm were included for this analysis. | Posted | | Number | | number of subjects | | 52 weeks after treatment start for patients on prophylaxis | | | | ID | Title | Description |
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| OG000 | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | Subjects were randomised to either prophylaxis 10 U/kg or 40 U/kg arm. Subjects in this arm received 10 U/kg nonacog beta pegol dose once weekly (every 7th day ± 24 hours) for a period of 52 weeks. Nonacog beta pegol was given as an intravenous bolus injection with the maximum injection rate at 4 mL/min. Mild and moderate bleeding episodes were treated with 40 U/kg. Severe bleeding episodes were treated with 80 U/kg. | | OG001 | Prophylaxis, High Dose 40 U/kg (52 Weeks) | Subjects were randomised to either prophylaxis 10 U/kg or 40 U/kg arm. Subjects in this arm received 40 U/kg nonacog beta pegol dose once weekly (every 7th day ± 24 hours) for a period of 52 weeks. Nonacog beta pegol was given as an intravenous bolus injection with the maximum injection rate at 4 mL/min. Mild and moderate bleeding episodes were treated with 40 U/kg. Severe bleeding episodes were treated with 80 U/kg. |
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| Secondary | Host Cell Proteins (HCP) Antibodies | Subjects who were positive for anti-HCP antibodies. | Safety analysis set included all subjects exposed to nonacog beta pegol. Subjects in on-demand arm were included for this analysis. | Posted | | Number | | number of subjects | | 28 weeks after treatment start on on-demand treatment | | | | ID | Title | Description |
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| OG000 | On-Demand (28 Weeks) | Subjects enrolled in this on-demand arm were treated for bleeding episodes on demand with nonacog beta pegol during a period of 28 weeks. Nonacog beta pegol was given as an intravenous bolus injection with the maximum injection rate at 4 mL/min. Mild and moderate bleeding episodes were treated with 40 U/kg. Severe bleeding episodes were treated with 80 U/kg. |
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