| Primary | The Incidence Rate of FVIII-inhibitors ≥0.6 BU: After Approximately 19 Months | All participants with neutralizing antibodies were included in the numerator and any participant with a minimum 50 exposure days plus any participant with inhibitory inhibitors was included in the denominator. A positive inhibitor test was defined as ≥0.6 bethesda unit (BU). Estimates are based on exact calculations for a binomial distribution. For the calculation of the 'inhibitor rate' the nominator included all participants with neutralising antibodies while the denominator included all participants with a minimum of 50 exposures plus any participant with less than 50 exposures but with neutralising inhibitors. | Results are based on the safety analysis set (SAS). SAS comprised all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Number | | Inhibitor rate | | After approximately 19 months | | | | ID | Title | Description |
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| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants received one single bolus dose of 50 U/kg of body weight (BW) of N8-GP administered intravenously (IV) every 4th day (96 hours) or twice weekly (investigator's discretion). The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
| | | Title | Denominators | Categories |
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| Primary | Annualised Bleeding Rate in the Prophylaxis Arm: After Approximately 19 Months | Annualised bleeding rate (ABR) is the number of bleeding episodes per year. This was assessed only for the prophylaxis treatment with N8-GP. | Results were based on the full analysis set (FAS) which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Median | Inter-Quartile Range | Bleeds per participant per year | | After approximately 19 months | | | | ID | Title | Description |
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| OG000 | N8-GP Prophylaxis | Participants from both the N8-GP 50 U/kg Q4D and N8-GP 75 U/kg Q7D groups were included in this arm. |
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| Primary | The Incidence Rate of FVIII-inhibitors ≥0.6 BU: After Approximately 25 Months | All participants with neutralizing antibodies were included in the numerator and any participant with a minimum 50 exposure days plus any participant with inhibitory inhibitors was included in the denominator. A positive inhibitor test was defined as ≥0.6 bethesda unit (BU). Estimates are based on exact calculations for a binomial distribution. For the calculation of the 'inhibitor rate' the nominator included all participants with neutralising antibodies while the denominator included all participants with a minimum of 50 exposures plus any participant with less than 50 exposures but with neutralising inhibitors. | Results are based on the SAS. SAS comprised all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Number | | Inhibitor rate | | After approximately 25 months | | | | ID | Title | Description |
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| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 |
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| Primary | Annualised Bleeding Rate in the Prophylaxis Arm: After Approximately 25 Months | ABR is the number of bleeding episodes per year. This was assessed only for the prophylaxis treatment with N8-GP. | Results are based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Median | Inter-Quartile Range | Bleeds per participant per year | | After approximately 25 months | | | | ID | Title | Description |
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| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 75 U/kg Prophylaxis Q7D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 3+ bleeding episodes during the last 6 months before entering the extension phase, were randomised to receive one single bolus dose of 75 U/kg BW of N8-GP. Participants received one single bolus dose of 75 U/kg BW of N8-GP administered IV every 7th day (Q7D). Based on the bleeding pattern, the investigator could change the dosing frequency from Q7D to Q4D, but not vice versa. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Primary | Incidence Rate of FVIII-inhibitors ≥0.6 BU: At Approximately 80 Months | All participants with neutralizing antibodies were included in the numerator and any participant with a minimum 50 exposure days plus any participant with inhibitory inhibitors was included in the denominator. A positive inhibitor test was defined as ≥0.6 bethesda unit (BU). Estimates are based on exact calculations for a binomial distribution. For the calculation of the 'inhibitor rate' the nominator included all participants with neutralising antibodies while the denominator included all participants with a minimum of 50 exposures plus any participant with less than 50 exposures but with neutralising inhibitors. | Results are based on the SAS. SAS comprised all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Number | | Inhibitor rate | | At approximately 80 months | | | | ID | Title | Description |
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| OG000 | N8-GP Prophylaxis | Participants from both the N8-GP 50 U/kg Q4D and N8-GP 75 U/kg Q7D groups were included in this arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
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| Primary | Annualised Bleeding Rate in the Prophylaxis Arm: After Approximately 80 Months | Annualised bleeding rate (ABR) is the number of bleeding episodes per year reported during the prophylactic treatment with N8-GP. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Median | Inter-Quartile Range | Bleeds per participant per year | | After approximately 80 months | | | | ID | Title | Description |
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| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 75 U/kg Prophylaxis Q7D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 3+ bleeding episodes during the last 6 months before entering the extension phase, were randomised to receive one single bolus dose of 75 U/kg BW of N8-GP. Participants received one single bolus dose of 75 U/kg BW of N8-GP administered IV every 7th day (Q7D). Based on the bleeding pattern, the investigator could change the dosing frequency from Q7D to Q4D, but not vice versa. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Haemostatic Effect of N8-GP When Used for Treatment of Bleeds, Assessed on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate and None): After Approximately 19 Months | Haemostatic effect of N8-GP for treatment of bleeding episodes was assessed by 4-point response scale: none, moderate, good or excellent. Evaluation during trial was done by participant and/or parent(s)/caregiver within approximately 8 hours after a single injection as follows: Excellent: Abrupt pain relief and/or clear improvement in objective signs of bleeding within approximately 8 hrs after a single injection; Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hrs after a single injection, but possibly requiring more than one injection for complete resolution; Moderate: Probable or slight beneficial effect within approximately 8 hours after the first injection, but usually requiring more than one injection; None: No improvement, or worsening of symptoms. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of bleeds analysed. | Posted | | Count of Units | | Bleeding episodes | | After approximately 19 months | Bleeding episodes | Bleeding episodes | | ID | Title | Description |
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| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Haemostatic Effect of N8-GP When Used for Treatment of Bleeds, Assessed on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate and None): After Approximately 25 Months | Haemostatic effect of N8-GP for treatment of bleeding episodes was assessed by 4-point response scale: none, moderate, good or excellent. Evaluation during trial was done by participant and/or parent(s)/caregiver within approximately 8 hours after a single injection as follows: Excellent: Abrupt pain relief and/or clear improvement in objective signs of bleeding within approximately 8 hrs after a single injection; Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hrs after a single injection, but possibly requiring more than one injection for complete resolution; Moderate: Probable or slight beneficial effect within approximately 8 hours after the first injection, but usually requiring more than one injection; None: No improvement, or worsening of symptoms. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of bleeds analysed. | Posted | | Count of Units | | Bleeding episodes | | After approximately 25 months | Bleeding episodes | Bleeding episodes | | ID | Title | Description |
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| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Haemostatic Effect of N8-GP When Used for Treatment of Bleeds, Assessed on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate and None): After Approximately 80 Months | Haemostatic effect of N8-GP for treatment of bleeding episodes was assessed by 4-point response scale: none, moderate, good or excellent. Evaluation during trial was done by participant and/or parent(s)/caregiver within approximately 8 hours after a single injection as follows: Excellent: Abrupt pain relief and/or clear improvement in objective signs of bleeding within approximately 8 hrs after a single injection; Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hrs after a single injection, but possibly requiring more than one injection for complete resolution; Moderate: Probable or slight beneficial effect within approximately 8 hours after the first injection, but usually requiring more than one injection; None: No improvement, or worsening of symptoms. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of bleeds analysed. | Posted | | Count of Units | | Bleeding episodes | | After approximately 80 months | Bleeding episodes | Bleeding episodes | | ID | Title | Description |
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| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Consumption of N8-GP Per Bleeding Episode (Number of Infusions): After Approximately 19 Months | The mean number of infusions of N8-GP used for treatment of a bleed from start to stop of a bleed was reported. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of infusions in respective arm. | Posted | | Mean | Standard Deviation | Number of infusions | | After approximately 19 months | Infusions | Infusions | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
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| Secondary | Consumption of N8-GP Per Bleeding Episode (Number of Infusions): After Approximately 25 Months | The mean number of infusions of N8-GP used for treatment of a bleed from start to stop of a bleed was reported. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of infusions in respective arm. | Posted | | Mean | Standard Deviation | Number of infusions | | After approximately 25 months | Infusions | Infusions | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 75 U/kg Prophylaxis Q7D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 3+ bleeding episodes during the last 6 months before entering the extension phase, were randomised to receive one single bolus dose of 75 U/kg BW of N8-GP. Participants received one single bolus dose of 75 U/kg BW of N8-GP administered IV every 7th day (Q7D). Based on the bleeding pattern, the investigator could change the dosing frequency from Q7D to Q4D, but not vice versa. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Consumption of N8-GP Per Bleeding Episode (Number of Infusions): After Approximately 80 Months | The mean number of infusions of N8-GP used for treatment of a bleed from start to stop of a bleed was reported. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of infusions in respective arm. | Posted | | Mean | Standard Deviation | Number of infusions | | After approximately 80 months | Infusions | Infusions | | ID | Title | Description |
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| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 75 U/kg Prophylaxis Q7D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 3+ bleeding episodes during the last 6 months before entering the extension phase, were randomised to receive one single bolus dose of 75 U/kg BW of N8-GP. Participants received one single bolus dose of 75 U/kg BW of N8-GP administered IV every 7th day (Q7D). Based on the bleeding pattern, the investigator could change the dosing frequency from Q7D to Q4D, but not vice versa. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Consumption of N8-GP Per Bleeding Episode (U/kg): After Approximately 19 Months | The mean consumption of N8-GP (U/kg) used for treatment of a bleed from start to stop of a bleed was reported. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of bleeds analysed | Posted | | Mean | Standard Deviation | U/kg per bleed | | After approximately 19 months | Bleeding episodes | Bleeding episodes | | ID | Title | Description |
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| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
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| Secondary | Consumption of N8-GP Per Bleeding Episode (U/kg): After Approximately 25 Months | The mean consumption of N8-GP (U/kg) used for treatment of a bleed from start to stop of a bleed was reported. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of bleeds analysed. | Posted | | Mean | Standard Deviation | U/kg per bleed | | After approximately 25 months | Bleeding episodes | Bleeding episodes | | ID | Title | Description |
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| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 75 U/kg Prophylaxis Q7D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 3+ bleeding episodes during the last 6 months before entering the extension phase, were randomised to receive one single bolus dose of 75 U/kg BW of N8-GP. Participants received one single bolus dose of 75 U/kg BW of N8-GP administered IV every 7th day (Q7D). Based on the bleeding pattern, the investigator could change the dosing frequency from Q7D to Q4D, but not vice versa. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Consumption of N8-GP Per Bleeding Episode (U/kg): After Approximately 80 Months | The mean consumption of N8-GP (U/kg) used for treatment of a bleed from start to stop of a bleed was reported. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of bleeds analysed. | Posted | | Mean | Standard Deviation | U/kg per bleed | | After approximately 80 months | Bleeding episodes | Bleeding episodes | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 75 U/kg Prophylaxis Q7D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 3+ bleeding episodes during the last 6 months before entering the extension phase, were randomised to receive one single bolus dose of 75 U/kg BW of N8-GP. Participants received one single bolus dose of 75 U/kg BW of N8-GP administered IV every 7th day (Q7D). Based on the bleeding pattern, the investigator could change the dosing frequency from Q7D to Q4D, but not vice versa. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Consumption of N8-GP (Number of Infusions) During Prophylaxis and On-demand Treatment: After Approximately 19 Months | Number of infusions are presented as average dose used during propphylaxis and on-demand treatment. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of infusions used during prophylaxis and on-demand treatment. | Posted | | Mean | Standard Deviation | U/kg | | After approximately 19 months | Infusions | Infusions | | ID | Title | Description |
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| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
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| Secondary | Consumption of N8-GP (Number of Infusions) During Prophylaxis and On-demand Treatment: After Approximately 25 Months | Number of infusions are presented as average dose used during prophylaxis and on-demand treatment. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of infusions used during prophylaxis and on-demand treatment. | Posted | | Mean | Standard Deviation | U/kg | | After approximately 25 months | Infusions | Infusions | | ID | Title | Description |
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| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 75 U/kg Prophylaxis Q7D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 3+ bleeding episodes during the last 6 months before entering the extension phase, were randomised to receive one single bolus dose of 75 U/kg BW of N8-GP. Participants received one single bolus dose of 75 U/kg BW of N8-GP administered IV every 7th day (Q7D). Based on the bleeding pattern, the investigator could change the dosing frequency from Q7D to Q4D, but not vice versa. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Consumption of N8-GP (Number of Infusions) During Prophylaxis and On-demand Treatment: After Approximately 80 Months | Number of infusions are presented as average dose used during prophylaxis and on-demand treatment. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of infusions used during prophylaxis and on-demand treatment. | Posted | | Mean | Standard Deviation | U/kg | | After approximately 80 months | Infusions | Infusions | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 75 U/kg Prophylaxis Q7D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 3+ bleeding episodes during the last 6 months before entering the extension phase, were randomised to receive one single bolus dose of 75 U/kg BW of N8-GP. Participants received one single bolus dose of 75 U/kg BW of N8-GP administered IV every 7th day (Q7D). Based on the bleeding pattern, the investigator could change the dosing frequency from Q7D to Q4D, but not vice versa. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Consumption of N8-GP (U/kg Per Month) During Prophylaxis and On-demand Treatment: After Approximately 19 Months | The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per month per participant) was reported. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | U/kg per month | | After approximately 19 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
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| Secondary | Consumption of N8-GP (U/kg Per Month) During Prophylaxis and On-demand Treatment: After Approximately 25 Months | The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per month per participant) was reported. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | U/kg per month | | After approximately 25 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 75 U/kg Prophylaxis Q7D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 3+ bleeding episodes during the last 6 months before entering the extension phase, were randomised to receive one single bolus dose of 75 U/kg BW of N8-GP. Participants received one single bolus dose of 75 U/kg BW of N8-GP administered IV every 7th day (Q7D). Based on the bleeding pattern, the investigator could change the dosing frequency from Q7D to Q4D, but not vice versa. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Consumption of N8-GP (U/kg Per Month) During Prophylaxis and On-demand Treatment: After Approximately 80 Months | The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per month per participant) was reported. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | U/kg per month | | After approximately 80 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 75 U/kg Prophylaxis Q7D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 3+ bleeding episodes during the last 6 months before entering the extension phase, were randomised to receive one single bolus dose of 75 U/kg BW of N8-GP. Participants received one single bolus dose of 75 U/kg BW of N8-GP administered IV every 7th day (Q7D). Based on the bleeding pattern, the investigator could change the dosing frequency from Q7D to Q4D, but not vice versa. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Consumption of N8-GP (U/kg Per Year) During Prophylaxis and On-demand Treatment: After Approximately 19 Months | The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per year per participant) was reported. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | U/kg per year | | After approximately 19 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
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| Secondary | Consumption of N8-GP (U/kg Per Year) During Prophylaxis and On-demand Treatment: After Approximately 25 Months | The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per year per participant) was reported. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | U/kg per year | | After approximately 25 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 75 U/kg Prophylaxis Q7D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 3+ bleeding episodes during the last 6 months before entering the extension phase, were randomised to receive one single bolus dose of 75 U/kg BW of N8-GP. Participants received one single bolus dose of 75 U/kg BW of N8-GP administered IV every 7th day (Q7D). Based on the bleeding pattern, the investigator could change the dosing frequency from Q7D to Q4D, but not vice versa. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Consumption of N8-GP (U/kg Per Year) During Prophylaxis and On-demand Treatment: After Approximately 80 Months | The mean consumption of N8-GP used for treatment of a bleed from start to stop of a bleed (per year per participant) was reported. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | U/kg per year | | After approximately 80 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 75 U/kg Prophylaxis Q7D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 3+ bleeding episodes during the last 6 months before entering the extension phase, were randomised to receive one single bolus dose of 75 U/kg BW of N8-GP. Participants received one single bolus dose of 75 U/kg BW of N8-GP administered IV every 7th day (Q7D). Based on the bleeding pattern, the investigator could change the dosing frequency from Q7D to Q4D, but not vice versa. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Haemostatic Effect as Measured by Recovery and Trough Levels FVIII:C (in All Patients Receiving Prophylaxis Treatment): After Approximately 19 Months | Recovery and trough levels of FVIII:C was reported for all participants at Visit 3 (Week 4) and end of main phase (approx. 19 months). The data was reported for all participants who received prophylaxis treatment. Chromogenic assay was performed with N8-GP product specific standard (PSS) as a calibrator. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Geometric Mean | Geometric Coefficient of Variation | U/mL | | After approximately 19 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Haemostatic Effect as Measured by Recovery and Trough Levels FVIII:C (in All Patients Receiving Prophylaxis Treatment): After Approximately 25 Months | Recovery and trough levels of FVIII:C was reported for all participants at the end of extension phase 1 study (approx. 25 months). The data was reported for all participants who received prophylaxis treatment. Chromogenic assay was performed with N8-GP product specific standard (PSS) as a calibrator. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Geometric Mean | Geometric Coefficient of Variation | U/mL | | After approximately 25 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 75 U/kg Prophylaxis Q7D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 3+ bleeding episodes during the last 6 months before entering the extension phase, were randomised to receive one single bolus dose of 75 U/kg BW of N8-GP. Participants received one single bolus dose of 75 U/kg BW of N8-GP administered IV every 7th day (Q7D). Based on the bleeding pattern, the investigator could change the dosing frequency from Q7D to Q4D, but not vice versa. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Haemostatic Effect as Measured by Recovery and Trough Levels FVIII:C (in All Patients Receiving Prophylaxis Treatment): After Approximately 80 Months | Since patients were allowed to change prophylaxis regimen at any time during the extension phase part 2, and since the visit intervals were different for the 2 prophylaxis treatment regimens (Q4D and Q7D), FVIII activity data are reported only as incremental recovery at this timepoint. | Data were not collected for this endpoint. | Posted | | | | | | After approximately 80 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 75 U/kg Prophylaxis Q7D | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
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| Secondary | Patient Reported Outcomes - Change in HAEMO-QOL Total Scores (Patients 13-16 Years Old) After Approx 19 and 25 Months | Reported results are change from baseline (Month 0) measured at end of main phase (approx Month 19) and change from Month 19 at end of Extension 1 (approx Month 25) of the study. The HAEMO-QOL assessment included questions on physical health, feeling, view of yourself, family, friends, perceived support, other persons, sports and school, dealing with haemophilia, treatment, future, and relationships. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data. No participants from the 13-16 years age group received on-demand treatment. Thus on-demand treatment group is not applicable for this endpoint. | Posted | | Mean | Standard Deviation | Scores on a scale | | After approx 19 and 25 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants from the N8-GP 50 U/kg Q4D group were included in this arm. |
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| Secondary | Patient Reported Outcomes - Change in HAEMO-QOL Total Scores (Patients 13-16 Years Old): After Approx 80 Months | Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. The HAEMO-QOL assessment included questions on physical health, feeling, view of yourself, family, friends, perceived support, other persons, sports and school, dealing with haemophilia, treatment, future, and relationships. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. Overall number of units analysed = Maximum no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = maximum number of participants contributed to the analysis for each time point. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. No participants from the 13-16 years age group received on-demand treatment. Thus on-demand treatment group is not applicable for this endpoint. | Posted | | Mean | Standard Deviation | Scores on a scale | | 2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs | Number of questionnaires | Number of questionnaires | | ID | Title | Description |
|---|
| OG000 | N8-GP Prophylaxis | Participants from both the 50 U/kg Q4D and the 75 U/kg Q7D prophylaxis arms were included in this arm. |
| |
| Secondary | Patient Reported Outcomes - Change in HAEMO-QOL Total Scores (Parents of Patients 13-16 Years Old): After Approx 19 and 25 Months | Reported results are change from baseline (Month 0) measured at end of main phase (Month 19) and change from Month 19 to end of Extension 1 (Month 25) of the study. The questionnaire was completed by parents of the patients in the 13-16 years old age bracket. The HAEMO-QOL assessment included questions on physical health, feeling, view of yourself, family, friends, perceived support, other persons, sports and school, dealing with haemophilia, treatment, future, and relationships. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data. No participants from the 13-16 years age group received on-demand treatment. Thus on-demand treatment group is not applicable for this endpoint. | Posted | | Mean | Standard Deviation | Scores on a scale | | After approx 19 and 25 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants from the N8-GP 50 U/kg Q4D group were included in this arm. |
| |
| Secondary | Patient Reported Outcomes - Change in HAEMO-QOL Total Scores (Parents of Patients 13-16 Years Old): After Approx 80 Months | Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. The questionnaire was completed by parents of the patients in the 13-16 years old age bracket. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. The HAEMO-QOL assessment included questions on physical health, feeling, view of yourself, family, friends, perceived support, other persons, sports and school, dealing with haemophilia, treatment, future, and relationships. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. Overall number of units analysed = Maximum no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = maximum number of participants contributed to the analysis for each time point. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. No participants from the 13-16 years age group received on-demand treatment. Thus on-demand treatment group is not applicable for this endpoint. | Posted | | Mean | Standard Deviation | Scores on a scale | | 2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs | Number of questionnaires | Number of questionnaires | | ID | Title | Description |
|---|
| OG000 | N8-GP Prophylaxis | Participants from both the 50 U/kg Q4D and the 75 U/kg Q7D prophylaxis arms were included in this arm. |
| |
| Secondary | Patient Reported Outcomes - Change in HAEM-A-QOL (>=17 Years) Total Scores: After Approx 19 and 25 Months | Reported results are change from baseline (Month 0) measured at end of main phase (Month 19) and change from Month 19 to end of Extension 1 (Month 25) of the study. The HAEM-A-QOL (for adults (>=17 years)) assessment included questions on questions on physical health, feeling, view of yourself, sports and leisure, work and school, dealing with haemophilia, treatment, future, family planning, and partnership and sexuality. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | Scores on a scale | | After approx 19 and 25 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 |
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| Secondary | Patient Reported Outcomes - Change in HAEM-A-QOL (>=17 Years) Total Scores: After Approximately 80 Months | Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. Overall number of units analysed = Maximum no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = maximum number of participants contributed to the analysis for each time point. The HAEM-A-QOL (for adults (>=17 years)) assessment included questions on physical health, feeling, view of yourself, sports and leisure, work and school, dealing with haemophilia, treatment, future, family planning, and partnership and sexuality. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. Scores range for each question was 0-100, with a lower score indicating better quality of life related to haemophilia. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | Scores on a scale | | 2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs | Number of questionnaires | Number of questionnaires | | ID | Title | Description |
|---|
| OG000 | N8-GP Prophylaxis | Participants from both the 50 U/kg Q4D and the 75 U/kg Q7D prophylaxis arms were included in this arm. | | OG001 |
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| Secondary | Patient Reported Outcomes - Change in HEMO-SAT (Patients) Scores: After Approx 19 and 25 Months | Reported results are change from baseline (Month 0) measured at end of main phase (Month 19) and change from Month 19 to end of Extension 1 (Month 25) of the study. The HEMO-SAT (Hematology-satisfaction) assessment included questions on treatment aspects including Ease and convenience, efficacy, burden, specialist/nurses, centre/hospital, general satisfaction (reported by patients). 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. A decrease in the score would mean improvement. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | Scores on a scale | | After approx 19 and 25 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Patient Reported Outcomes - Change in HEMO-SAT (Patients) Scores: After Approximately 80 Months | Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. Overall number of units analysed = Maximum no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = maximum number of participants contributed to the analysis for each time point. The HEMO-SAT assessment included questions on treatment aspects including Ease and convenience, efficacy, burden, specialist/nurses, centre/hospital, general satisfaction (reported by patients). 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. A decrease in the score would mean improvement. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. | Posted | | Mean | Standard Deviation | Scores on a scale | | 1-<2 yrs, 2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs | Number of questionnaires | Number of questionnaires | | ID | Title | Description |
|---|
| OG000 | N8-GP Prophylaxis | Participants from both the 50 U/kg Q4D and the 75 U/kg Q7D prophylaxis arms were included in this arm. | | OG001 | N8-GP 20-75 U/kg On-demand |
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| Secondary | Patient Reported Outcomes - Change in HEMO-SAT Scores (Parents): After Approx 19 and 25 Months | The summary of change was based on individual changes observed at visit 13 (approximately 19 months) from visit 2a pre-dose (Month 0). The HEMO-SAT assessment included questions on treatment aspects including Ease and convenience, efficacy, burden, specialist/nurses, centre/hospital, general satisfaction (reported by parents). 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. A decrease in the score would mean improvement. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | Scores on a scale | | After approx 19 and 25 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
| |
| Secondary | Patient Reported Outcomes - Change in HEMO-SAT (Parents) Scores: After Approximately 80 Months | Reported results are from Visit 1 (Month 0), and change from visit 1 upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. Overall number of units analysed = Max. no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = max. number of participants contributed to the analysis for each time point. The HEMO-SAT assessment included questions on treatment aspects including Ease and convenience, efficacy, burden, specialist/nurses, centre/hospital, 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. A decrease in the score would mean improvement. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. | Posted | | Mean | Standard Deviation | Scores on a scale | | 2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs | Number of questionnaires | Number of questionnaires | | ID | Title | Description |
|---|
| OG000 | N8-GP Prophylaxis | Participants from both the 50 U/kg Q4D and the 75 U/kg Q7D prophylaxis arms were included in this arm. |
| |
| Secondary | Patient Reported Outcomes - Change in EQ-5D-VAS Scores: After Approx 19 and 25 Months | Reported results are change from baseline (Month 0) measured at end of main phase (Month 19) and change from Month 19 to end of Extension 1 (Month 25) of the study. The European quality of life visual analogue scale (EQ5D-VAS) records the patient's self-rated health on a vertical visual analogue scale, where the endpoints are labelled 'The best health you can imagine' and 'The worst health you can imagine'. The VAS can be used as a quantitative measure of health outcome that reflect the patient's own judgement. EQ-5D-VAS: range 0 to 100. A higher score indicates better self reported health status. A positive change indicates an improvement. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | Scores on a scale | | After approx 19 and 25 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
|
| Secondary | Patient Reported Outcomes - Change in EQ-5D-VAS Scores: After Approximately 80 Months | Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It was possible that a participant could answer more than one questionnaire in a single time interval. Overall number of units analysed = Maximum no of questionnaires answered by participants for this endpoint. Overall number of participants analysed = maximum number of participants contributed to the analysis for each time point. The EQ5D-VAS records the patient's self-rated health on a vertical visual analogue scale, where the endpoints are labelled 'The best health you can imagine' and 'The worst health you can imagine'. The VAS can be used as a quantitative measure of health outcome that reflect the patient's own judgement. EQ-5D-VAS: range 0 to 100. A higher score indicates better self reported health status. A positive change indicates an improvement. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. | Posted | | Mean | Standard Deviation | Scores on a scale | | 1-<2 yrs, 2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs | Number of questionnaires | Number of questionnaires | | ID | Title | Description |
|---|
| OG000 | N8-GP Prophylaxis | Participants from both the 50 U/kg Q4D and the 75 U/kg Q7D prophylaxis arms were included in this arm. | | OG001 | N8-GP 20-75 U/kg On-demand | |
|
| Secondary | Patient Reported Outcomes - Change in European Quality of Life Utility Index: After Approx 19 and 25 Months | Reported results are change from baseline (Month 0) measured at end of main phase (Month 19) and change from Month 19 to end of Extension 1 (Month 25) of the study. The European quality of life utility index comprises five dimensions: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 3 levels where 1 indicates better health state (no problems) and 3 indicates worst health state (confined to bed). Scoring formula developed by EuroQol Group assigns a utility value for each domain in the profile. Score is transformed and results in a total score range -0.594 to 1.000; a positive change indicates an improvement. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | Scores on a scale | | After approx 19 and 25 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Patient Reported Outcomes - Change in European Quality of Life Utility Index Scores: After Approximately 80 Months | Reported results are change from visit 1 (Month 0) upto end of Extension phase 2 (Month 80) of the study. Time intervals are assigned based on time after first dose. It is possible that a patient answers more than one questionnaire in a single time interval. Overall units analysed = Max no. of questionnaires answered by participants for this endpoint. Overall no. of participants analysed = max no. of participants analysed at each time point. This utility index has 5 dimensions: mobility, self-care, usual activities, pain/discomfort & anxiety/depression. Each dimension has 3 levels where 1 indicates better health state (no problems) and 3 indicates worst health state (confined to bed). Scoring formula developed by EuroQol Group assigns a utility value for each domain in the profile. Score is transformed and results in a total score range -0.594 to 1.000; a positive change indicates an improvement. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. | Posted | | Mean | Standard Deviation | Scores on a scale | | 1-<2 yrs, 2-<3 yrs, 3-<4 yrs, 4-<5 yrs, 5-<6 yrs and 6-<7 yrs | Number of questionnaires | Number of questionnaires | | ID | Title | Description |
|---|
| OG000 | N8-GP Prophylaxis | Participants from both the 50 U/kg Q4D and the 75 U/kg Q7D prophylaxis arms were included in this arm. | | OG001 | N8-GP 20-75 U/kg On-demand | |
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| Secondary | Number of Hospital Admissions During the Trial | The number of hospital admissions that took place in the study were reported. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Count of Units | | Number of hospital admissions | | After approx 19, 25 and 80 months | Number of hospital admissions | Number of hospital admissions | | ID | Title | Description |
|---|
| OG000 | N8-GP Prophylaxis | Participants from both the N8-GP 50 U/kg Q4D and N8-GP 75 U/kg Q7D groups were included in this arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
| |
| Secondary | Number of Days at the Hospital During the Trial | The mean number of days that participants spent at the hospital during the study were reported. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | Days | | After approx 19, 25 and 80 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP Prophylaxis | Participants from both the N8-GP 50 U/kg Q4D and N8-GP 75 U/kg Q7D groups were included in this arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
| |
| Secondary | Number of Admissions to the Emergency Room (ER) During the Trial | The number of admissions to the ER that took place in the study were reported for each group. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Count of Units | | ER admissions | | After approx 19, 25 and 80 months | ER admissions | ER admissions | | ID | Title | Description |
|---|
| OG000 | N8-GP Prophylaxis | Participants from both the N8-GP 50 U/kg Q4D and N8-GP 75 U/kg Q7D groups were included in this arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
| |
| Secondary | Number of Days Missing School or Work | The mean number of days that participants missed to go to school or work were reported. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | Days | | Approx 19, 25 and 80 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP Prophylaxis | Participants from both the N8-GP 50 U/kg Q4D and N8-GP 75 U/kg Q7D groups were included in this arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
| |
| Secondary | Number of Days Using Mobility Aid | The mean number of days that participants used any aids for mobility during the study were reported. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | Days | | Approx 19, 25 and 80 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP Prophylaxis | Participants from both the N8-GP 50 U/kg Q4D and N8-GP 75 U/kg Q7D groups were included in this arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
| |
| Secondary | Number of Participants Using Pain Medication | The number of participants using pain medication during the main plus extension phase 1 of the study (approximately 25 months) and during extension phase 2(approximately 80 months) were reported. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Count of Participants | | Participants | | After approx 25 and 80 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP Prophylaxis | Participants in this arm included participants from both the Q4D and the Q7D groups | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
| |
| Secondary | Number of Bleeds Using Pain Medication | The mean number of bleeds using pain medication in the main phase of the study (approximately 19 months) were reported. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | Number of bleeds | | After approx 19 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D: | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
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| Secondary | Number of Adverse Events Reported During the Trial Period: After Approximately 19 Months | All presented adverse events (AEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration. | Results are based on the SAS. SAS comprised all participants exposed to N8-GP in this trial. | Posted | | Number | | Number of adverse events | | After approx 19 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
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| Secondary | Number of Adverse Events Reported During the Trial Period: After Approximately 25 Months | All presented adverse events (AEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration. | Results are based on the SAS. SAS comprised all participants exposed to N8-GP in this trial. | Posted | | Number | | Number of adverse events | | After approx. 25 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 75 U/kg Prophylaxis Q7D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 3+ bleeding episodes during the last 6 months before entering the extension phase, were randomised to receive one single bolus dose of 75 U/kg BW of N8-GP. Participants received one single bolus dose of 75 U/kg BW of N8-GP administered IV every 7th day (Q7D). Based on the bleeding pattern, the investigator could change the dosing frequency from Q7D to Q4D, but not vice versa. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Number of Adverse Events Reported During the Trial Period: After Approximately 80 Months | The number of adverse events observed during the study after approximately 80 months was reported. | Results are based on the SAS. SAS comprised all participants exposed to N8-GP in this trial. | Posted | | Number | | Number of adverse events | | After approximately 80 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 75 U/kg Prophylaxis Q7D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 3+ bleeding episodes during the last 6 months before entering the extension phase, were randomised to receive one single bolus dose of 75 U/kg BW of N8-GP. Participants received one single bolus dose of 75 U/kg BW of N8-GP administered IV every 7th day (Q7D). Based on the bleeding pattern, the investigator could change the dosing frequency from Q7D to Q4D, but not vice versa. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Number of Serious Adverse Events Reported During the Trial Period: After Approximately 19 Months | All presented serious adverse events (SAEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration. | Results are based on the SAS. SAS comprised all participants exposed to N8-GP in this trial. | Posted | | Number | | Number of serious adverse events | | After approximately 19 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
|
| Secondary | Number of Serious Adverse Events Reported During the Trial Period: After Approximately 25 Months | All presented serious adverse events (SAEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration. | Results are based on the SAS. SAS comprised all participants exposed to N8-GP in this trial. | Posted | | Number | | Number of serious adverse events | | After approximately 25 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 75 U/kg Prophylaxis Q7D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 3+ bleeding episodes during the last 6 months before entering the extension phase, were randomised to receive one single bolus dose of 75 U/kg BW of N8-GP. Participants received one single bolus dose of 75 U/kg BW of N8-GP administered IV every 7th day (Q7D). Based on the bleeding pattern, the investigator could change the dosing frequency from Q7D to Q4D, but not vice versa. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
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| Secondary | Number of Serious Adverse Events Reported During the Trial Period: After Approximately 80 Months | All presented serious adverse events (SAEs) are treatment-emergent. A treatment-emergent adverse event was defined as an event with onset after first N8-GP administration. | Results are based on the SAS. SAS comprised all participants exposed to N8-GP in this trial. | Posted | | Number | | Number of serious adverse events | | After approximately 80 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 75 U/kg Prophylaxis Q7D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 3+ bleeding episodes during the last 6 months before entering the extension phase, were randomised to receive one single bolus dose of 75 U/kg BW of N8-GP. Participants received one single bolus dose of 75 U/kg BW of N8-GP administered IV every 7th day (Q7D). Based on the bleeding pattern, the investigator could change the dosing frequency from Q7D to Q4D, but not vice versa. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. |
|
| Secondary | Change in Blood Pressure: After Approximately 19 Months | The mean change in the systolic and diastolic blood pressure values of participants was reported. The summary of change was based on individual changes observed at visit 13 (approximately 19 months) from visit 2a pre-dose (Month 0). | Results were based on the SAS. SAS comprised all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | mmHg | | After approximately 19 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
|
| Secondary | Change in Blood Pressure: After Approximately 25 Months | The mean change in the systolic and diastolic blood pressure values of participants was reported. The summary of change was based on individual changes observed at visit 17 (approximately month 25) from visit 13 (approximately month 19). | Results were based on the SAS. SAS comprised all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | mmHg | | After approximately 19 and 25 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
|
| Secondary | Change in Blood Pressure: After Approximately 80 Months | Change in the blood pressure was not calculated in the extension phase 2 of the study since participants were allowed to change from Q4D to Q7D and vice versa. | Data were not collected for this outcome measure. | Posted | | | | | | After approximately 80 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
| |
| Secondary | Change in Pulse: After Approximately 19 Months | The mean change in the pulse values of participants was reported. The summary of change was based on individual changes observed at visit 13 (approximately 19 months) from visit 2a pre-dose (Month 0). | Results were based on the SAS. SAS comprised all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | Beats per min | | After approximately 19 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants in this arm received one single bolus dose of 50 U/kg BW of N8-GP administered intravenously (IV) every 4th day (96 hours) or twice weekly (investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
| |
| Secondary | Change in Pulse: After Approximately 25 Months | The mean change in the pulse values of participants was reported. The summary of change was based on individual changes observed at visit 17 (approximately month 25) from visit 13 (approximately month 19). | Results were based on the SAS. SAS comprised all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | Beats per min | | After approximately 25 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
|
| Secondary | Change in Pulse: After Approximately 80 Months | Change in pulse was not calculated in the extension phase 2 of the study since participants were allowed to change from Q4D to Q7D and vice versa. | Data were not collected for this outcome measure. | Posted | | | | | | After approximately 80 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
| |
| Secondary | Change in Body Temperature: After Approximately 19 Months | The mean change in the body temperature values of participants was reported. The summary of change was based on individual changes observed at visit 13 (approximately 19 months) from visit 2a pre-dose (Month 0). | Results were based on the SAS. SAS comprised all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | Degree celcius | | After approximately 19 months | | | | ID | Title | Description |
|---|
| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
|
| Secondary | Change in Body Temperature: After Approximately 25 Months | The mean change in the body temperature (C) values of participants was reported. The summary of change was based on individual changes observed at visit 17 (approximately month 25) from visit 13 (approximately month 19). | Results were based on the SAS. SAS comprised all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | Degree celcius | | After approximately 25 months | | | | ID | Title | Description |
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| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
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| Secondary | Change in Body Temperature: After Approximately 80 Months | Change in body temperature was not calculated in the extension phase 2 of the study since participants were allowed to change from Q4D to Q7D and vice versa. | Data were not collected for this outcome measure. | Posted | | | | | | After approximately 80 months | | | | ID | Title | Description |
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| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5 |
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| Secondary | Change in Respiratory Rate: After Approximately 19 Months | The mean change in the respiratory rate values of participants was reported. The summary of change was based on individual changes observed at visit 13 (approximately 19 months) from visit 2a pre-dose (Month 0). | Results were based on the SAS. SAS comprised all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | Breaths/min | | After approximately 19 months | | | | ID | Title | Description |
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| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
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| Secondary | Change in Respiratory Rate: After Approximately 25 Months | The mean change in the respiratory rate (breaths/min) values of participants was reported. The summary of change was based on individual changes observed at visit 17 (approximately month 25) from visit 13 (approximately month 19). | Results were based on the SAS. SAS comprised all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Mean | Standard Deviation | Breaths/min | | After approximately 25 months | | | | ID | Title | Description |
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| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants in this arm received one single bolus dose of 50 U/kg BW of N8-GP administered intravenously (IV) every 4th day (96 hours) or twice weekly (investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
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| Secondary | Change in Respiratory Rate: After Approximately 80 Months | Change in respiratory rate was not calculated in the extension phase 2 of the study since participants were allowed to change from Q4D to Q7D and vice versa. | Data were not collected for this outcome measure. | Posted | | | | | | After approximately 80 months | | | | ID | Title | Description |
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| OG000 | N8-GP 50 U/kg Prophylaxis Q4D | Participants who were on prophylaxis treatment with N8-GP in the main phase and had 0-2 bleeding episodes during the last 6 months before entering the extension phase, were included in this arm. Participants received one single bolus dose of 50 U/kg BW of N8-GP administered IV every 4th day (96 hours) or twice weekly (at investigator's discretion). The dose was adjusted to ensure a trough level of >1% FVIII:C activity in this arm. The dose was based on phase 1 data from the NN7088-3776 trial in order to ensure a trough level of >1% FVIII:C activity in the majority of participants in the prophylaxis arm. | | OG001 | N8-GP 20-75 U/kg On-demand | Participants in this arm received treatment with N8-GP in case of a bleeding episode. All bleeds were to be treated with doses between 20-75 U/kg BW according to the severity and location of the bleeding episode. The dosage (N8-GP units) was calculated by multiplying the participant's weight in kilograms by the desired factor level multiplied by 0.5. |
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| Secondary | FVIII Activity 30 Min Post -Injection (C30min) | FVIII plasma activity was measured after 30 mins of injection. This was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Geometric Mean | Geometric Coefficient of Variation | IU/mL | | Week 0, week 28 | | | | ID | Title | Description |
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| OG000 | Pharmacokinetics Assessment Arm | Patients participating in the pharmacokinetic assessments received a single i.v. bolus injection of 50 U/kg BW of N8-GP at Visit 2a (Week 0) and at Visit 7 (Week 28). The PK sessions were 4 days long and with multiple site visits. After completion of the PK sessions the patients continued treatment in the prophylaxis arm with the next visit in line. Patients must not have received current FVIII product for at least 4 days prior to the PK session at Visit 2a (Week 0) and N8-GP for at least 7 days prior to the PK session at Visit 7 (Week 28). |
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| Secondary | Incremental Recovery (Single Dose and Steady State) | Incremental recovery was defined as the dose-normalised activity recorded 30 min after end of injection. This was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Geometric Mean | Geometric Coefficient of Variation | [(U/mL)/(IU/kg)] | | Week 0, week 28 | | | | ID | Title | Description |
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| OG000 | Pharmacokinetics Assessment Arm | Patients participating in the pharmacokinetic assessments received a single i.v. bolus injection of 50 U/kg BW of N8-GP at Visit 2a (Week 0) and at Visit 7 (Week 28). The PK sessions were 4 days long and with multiple site visits. After completion of the PK sessions the patients continued treatment in the prophylaxis arm with the next visit in line. Patients must not have received current FVIII product for at least 4 days prior to the PK session at Visit 2a (Week 0) and N8-GP for at least 7 days prior to the PK session at Visit 7 (Week 28). |
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| Secondary | Trough Level (Single Dose and Steady State) | Trough level was defined as the plasma FVIII activity recorded immediately before next dose is given. This was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Geometric Mean | Geometric Coefficient of Variation | IU/mL | | Week 0, week 28 | | | | ID | Title | Description |
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| OG000 | Pharmacokinetics Assessment Arm | Patients participating in the pharmacokinetic assessments received a single i.v. bolus injection of 50 U/kg BW of N8-GP at Visit 2a (Week 0) and at Visit 7 (Week 28). The PK sessions were 4 days long and with multiple site visits. After completion of the PK sessions the patients continued treatment in the prophylaxis arm with the next visit in line. Patients must not have received current FVIII product for at least 4 days prior to the PK session at Visit 2a (Week 0) and N8-GP for at least 7 days prior to the PK session at Visit 7 (Week 28). |
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| Secondary | Area Under the Curve (AUC0-inf) | Area under the plasma activity versus time profile from time zero to infinity (AUC0-inf) was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. It is the measure of total plasma exposure. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Geometric Mean | Geometric Coefficient of Variation | IU*h/mL | | Pre dose and 30 min, 1, 4, 12, 24, 48, 72 and 96 hours post dose at week 0 and week 28 | | | | ID | Title | Description |
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| OG000 | Pharmacokinetics Assessment Arm | Patients participating in the pharmacokinetic assessments received a single i.v. bolus injection of 50 U/kg BW of N8-GP at Visit 2a (Week 0) and at Visit 7 (Week 28). The PK sessions were 4 days long and with multiple site visits. After completion of the PK sessions the patients continued treatment in the prophylaxis arm with the next visit in line. Patients must not have received current FVIII product for at least 4 days prior to the PK session at Visit 2a (Week 0) and N8-GP for at least 7 days prior to the PK session at Visit 7 (Week 28). |
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| Secondary | Area Under the Curve (AUC0-t) | Area under the plasma activity versus time profile from time zero to the last measurable activity (AUC0-t) was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Geometric Mean | Geometric Coefficient of Variation | IU*h/mL | | Pre dose and 30 min, 1, 4, 12, 24, 48, 72 and 96 hours post dose at week 0 and week 28 | | | | ID | Title | Description |
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| OG000 | Pharmacokinetics Assessment Arm | Patients participating in the pharmacokinetic assessments received a single i.v. bolus injection of 50 U/kg BW of N8-GP at Visit 2a (Week 0) and at Visit 7 (Week 28). The PK sessions were 4 days long and with multiple site visits. After completion of the PK sessions the patients continued treatment in the prophylaxis arm with the next visit in line. Patients must not have received current FVIII product for at least 4 days prior to the PK session at Visit 2a (Week 0) and N8-GP for at least 7 days prior to the PK session at Visit 7 (Week 28). |
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| Secondary | Terminal Half Life (t1/2) | t½ = ln(2) / λz, where λz is the terminal elimination rate constant. The terminal elimination rate constant was estimated using linear regression on the terminal part of the log(activity) versus time profile. This was measured at Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Geometric Mean | Geometric Coefficient of Variation | Hour (h) | | Pre dose and 30 min, 1, 4, 12, 24, 48, 72 and 96 hours post dose at week 0 and week 28 | | | | ID | Title | Description |
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| OG000 | Pharmacokinetics Assessment Arm | Patients participating in the pharmacokinetic assessments received a single i.v. bolus injection of 50 U/kg BW of N8-GP at Visit 2a (Week 0) and at Visit 7 (Week 28). The PK sessions were 4 days long and with multiple site visits. After completion of the PK sessions the patients continued treatment in the prophylaxis arm with the next visit in line. Patients must not have received current FVIII product for at least 4 days prior to the PK session at Visit 2a (Week 0) and N8-GP for at least 7 days prior to the PK session at Visit 7 (Week 28). |
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| Secondary | Clearance (CL) | Total plasma clearance (CL) of drug after intravenous administration was reported. Clearance was calculated using the formula CL= Dose / AUC(0-inf). This was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Geometric Mean | Geometric Coefficient of Variation | mL/h/kg | | Pre dose and 30 min, 1, 4, 12, 24, 48, 72 and 96 hours post dose at week 0 and week 28 | | | | ID | Title | Description |
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| OG000 | Pharmacokinetics Assessment Arm | Patients participating in the pharmacokinetic assessments received a single i.v. bolus injection of 50 U/kg BW of N8-GP at Visit 2a (Week 0) and at Visit 7 (Week 28). The PK sessions were 4 days long and with multiple site visits. After completion of the PK sessions the patients continued treatment in the prophylaxis arm with the next visit in line. Patients must not have received current FVIII product for at least 4 days prior to the PK session at Visit 2a (Week 0) and N8-GP for at least 7 days prior to the PK session at Visit 7 (Week 28). |
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| Secondary | Mean Residence Time (MRT) | MRT = AUMC/AUC(0-inf), where AUMC is the area under the first moment curve, i.e. the area under the curve t∙C(t), calculated with the same method as AUC(0-inf) (linear trapezoidal method + extrapolated area). This was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Geometric Mean | Geometric Coefficient of Variation | Hour (h) | | Pre dose and 30 min, 1, 4, 12, 24, 48, 72 and 96 hours post dose at week 0 and week 28 | | | | ID | Title | Description |
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| OG000 | Pharmacokinetics Assessment Arm | Patients participating in the pharmacokinetic assessments received a single i.v. bolus injection of 50 U/kg BW of N8-GP at Visit 2a (Week 0) and at Visit 7 (Week 28). The PK sessions were 4 days long and with multiple site visits. After completion of the PK sessions the patients continued treatment in the prophylaxis arm with the next visit in line. Patients must not have received current FVIII product for at least 4 days prior to the PK session at Visit 2a (Week 0) and N8-GP for at least 7 days prior to the PK session at Visit 7 (Week 28). |
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| Secondary | Volume of Distribution at Steady State (Vss) | Apparent volume of distribution at steady state is a product of the mean residence time and clearance and was calculated using the formula - Vss = CL x MRT. This was measured at two time points Visit 2a (Week 0) and visit 7 (Week 28) during the Main Phase of the study. Chromogenic assay was performed with normal human plasma (NHP) as a calibrator. | Results were based on the FAS which included all participants exposed to N8-GP in this trial. Number analysed = Number of participants with available data for respective arm. | Posted | | Geometric Mean | Geometric Coefficient of Variation | mL/kg | | Pre dose and 30 min, 1, 4, 12, 24, 48, 72 and 96 hours post dose at week 0 and week 28 | | | | ID | Title | Description |
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| OG000 | Pharmacokinetics Assessment Arm | Patients participating in the pharmacokinetic assessments received a single i.v. bolus injection of 50 U/kg BW of N8-GP at Visit 2a (Week 0) and at Visit 7 (Week 28). The PK sessions were 4 days long and with multiple site visits. After completion of the PK sessions the patients continued treatment in the prophylaxis arm with the next visit in line. Patients must not have received current FVIII product for at least 4 days prior to the PK session at Visit 2a (Week 0) and N8-GP for at least 7 days prior to the PK session at Visit 7 (Week 28). |
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