| Primary | Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU) | Inhibitors were analysed with either the Nijmegen modified factor IX Bethesda assay or a heat/cold Nijmegen modified factor IX Bethesda assay. Number of participants who developed inhibitory antibodies against factor IX are reported. | Safety analysis set (SAS) included all participants exposed to nonacog beta pegol. | Posted | | Count of Participants | | Participants | | From week 0 to week 52 | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Primary | Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU) | Inhibitors were analysed with either the Nijmegen modified factor IX Bethesda assay or a heat/cold Nijmegen modified factor IX Bethesda assay. Number of participants who developed inhibitory antibodies against factor IX are reported. | Safety analysis set (SAS) included all participants exposed to nonacog beta pegol. | Posted | | Count of Participants | | Participants | | From week 52 to End of trial (EOT) (approximately week 544) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | Number of Bleeding Episodes During Prophylaxis | The number of bleeding episodes per participant during routine prophylaxis was assessed using the individual annualised bleeding rates (bleeding episodes per participant per year). | Full analysis set (FAS) included all participants with efficacy data after exposure to nonacog beta pegol. | Posted | | Median | Inter-Quartile Range | bleeds/participant/year | | From week 0 to EOT (approximately week 544) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | Haemostatic Effect of N9-GP in Treatment of Bleeding Episodes by 4-point Categorical Scale for Haemostatic Response (Excellent, Good, Moderate and Poor) | Description of the haemostatic effect of nonacog beta pegol when used for treatment of bleeding episodes was measured and listed according to the four point scale for haemostatic response as below:
- Excellent - abrupt pain relief and/or clear improvement in objective signs of bleeding within 8 hours after a single infusion.
- Good - noticeable pain relief and/or improvement in signs of bleeding within 8 hours after a single injection.
- Moderate - probable or slight beneficial effect within the first 8 hours after the first injection but requiring more than one infusion within 8 hours.
- Poor - no improvement, or worsening of symptoms within 8 hours after two injections.
A success rate was calculated based on counting good or excellent as successes and poor and moderate as failures. | FAS included all participants with efficacy data after exposure to nonacog beta pegol. Here, overall number of participants analysed=participants who experienced bleeding episodes. Number analysed = participants analysed for specific category for this outcome measure. | Posted | | Number | | percentage of bleeding episodes | | From week 0 to EOT (approximately week 544) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | Incremental Recovery at 30 Minutes (IR30min) | The incremental recovery was calculated by dividing the baseline-subtracted factor IX activity Units per milliliter (U/mL) measured in plasma 30 min after dosing by the dose injected at time 0 expressed as units per kilogram (U/kg) body weight. | FAS included all participants with efficacy data after exposure to nonacog beta pegol. Here, overall number of participants analysed = Participants who were evaluated for this parameter. | Posted | | Geometric Mean | Geometric Coefficient of Variation | (U/mL)/(U/kg) | | Week 0 (30 minutes after first exposure) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | Trough Level (Single-dose ) | The mean pre-dose factor IX levels was measured with the one-stage clotting assay during the trial. Geometric mean of the lowest activity of factor IX recorded at week 0 (immediately before next dose was given). | FAS included all participants with efficacy data after exposure to nonacog beta pegol. Here, overall number of participants analysed = Participants who were evaluated for this parameter. | Posted | | Geometric Mean | Geometric Coefficient of Variation | U/mL | | Week 0 (one week after first exposure) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | Terminal Half-life (t1/2) | Terminal half life is presented at week 0, 30 minutes until one week after first exposure. | FAS included all participants with efficacy data after exposure to nonacog beta pegol. | Posted | | Geometric Mean | Geometric Coefficient of Variation | hours | | Week 0 (30 minutes until one week after first exposure) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | Trough Level (Steady State) | The mean pre-dose factor IX levels was measured with the one-stage clotting assay during the trial. The estimated mean of the lowest activity recorded immediately before next dose was given from week 4 to EOT approximately (week 544). Data is reported for specific age groups in which participants were a part of at any time from week 4 to EOT, not at specific time points assessed from week 4 to EOT. The analysis is based on a mixed model on the log-transformed plasma concentrations with participant as a random effect and the mean trough level is presented back-transformed to the natural scale. Considering the time frame, actual age groups (adolescents and adults) are added for this outcome measure. | FAS included all participants with efficacy data after exposure to nonacog beta pegol. Age groups are based on actual age, therefore some participants are represented in multiple columns. | Posted | | Mean | 95% Confidence Interval | U/mL | | From week 4 to EOT (approximately week 544) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) |
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| Secondary | Number of Adverse Events | An adverse event (AE) was any unfavourable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. Here, data is presented for all adverse events (serious adverse events and other adverse events) from week 0 to EOT approximately (week 544). Data is reported for specific Age Groups in which participants were a part of at any time from week 0 to EOT, not at specific time points assessed from week 0 to EOT (approximately week 544). Considering the time frame, actual age groups (adolescents and adults) are added for this outcome measure. | SAS included all participants exposed to nonacog beta pegol. Here, overall number of participants analysed = participants who experienced bleeding episodes. Number analysed = participants analysed for specific category for this outcome measure. Age groups are based on actual age, therefore some participants are represented in multiple columns. | Posted | | Number | | Events | | Week 0 to EOT (approximately week 544) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 |
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| Secondary | Number of Serious Adverse Events (SAEs) | A SAE was an experience that at any dose resulted in any of the following: death, a life-threatening experience a), In-patient hospitalisation or prolongation of existing hospitalisation b) a persistent or significant disability/incapacity c) a congenital anomaly/birth defect, Important medical events d) that did not result in death, were life-threatening a) or required hospitalization. Here, data is presented from week 0 to EOT (approximately week 544). Data is reported for specific Age Groups in which participants were a part of at any time from week 0 to EOT, not at specific time points assessed from week 0 to EOT (approximately week 544). Considering the time frame, actual age groups (adolescents and adults) are added for this outcome measure. | SAS included all participants exposed to nonacog beta pegol. Here, overall number of participants analysed = participants who experienced bleeding episodes. Number analysed = participants analysed for specific category for this outcome measure. Age groups are based on actual age, therefore some participants are represented in multiple columns. | Posted | | Number | | Events | | Week 0 to EOT (approximately week 544) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | Medical Events of Special Interest (MESI) | The following events were defined as MESIs: -Medication errors concerning trial products, -Administration of wrong drug,
- Wrong route of administration,
- Administration of a high dose with the intention to cause harm, e.g. suicide attempt,
- Administration of an accidental overdose: more than 20 % from the intended dose,
- Inhibitor formation against factor IX (FIX),
- Thromboembolic events,
- Anaphylactic reaction.
- Allergic reaction including, but not limited to, any acute immunoglobulin E (IgE) mediated reaction or delayed type hypersensitivity.
Here, data is presented from week 0 to EOT (approximately week 544). Data is reported for specific Age Groups in which participants were a part of at any time from week 0 to EOT, not at specific time points assessed from week 0 to EOT (approximately week 544). Considering the time frame, actual age groups (adolescents and adults) are added for this outcome measure. | SAS included all participants exposed to nonacog beta pegol. Here, overall number of participants analysed = participants who experienced bleeding episodes. Number analysed = participants analysed for specific category for this outcome measure. Age groups are based on actual age, therefore some participants are represented in multiple columns. | Posted | | Number | | Events | | Week 0 to EOT (approximately week 544) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | Development of Host Cell Protein (HCP) Antibodies | Participants were examined for the development of antibodies against HCP. Number of participants who developed antibodies against HCP is presented. | SAS included all participants exposed to nonacog beta pegol. | Posted | | Count of Participants | | Participants | | From week 0 to EOT (approximately week 544) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | FIX Consumption Described as Frequency of Dose/kg for Prophylaxis Use for the Treatment of Bleeding Episodes | Consumption of nonacog beta pegol for treatment of bleeding episodes International units per kilogram per year (IU/Kg/year) per participant is presented from week 0 to EOT approximately (week 544). Data is reported for specific Age Groups in which participants were a part of at any time from week 0 to EOT, not at specific time points assessed from week 0 to EOT (approximately week 544). Considering the time frame, actual age groups (adolescents and adults) are added for this outcome measure. | The FAS included all participants with efficacy data after exposure to nonacog beta pegol. Here, overall number of participants analysed = participants who experienced bleeding episodes. Number analysed = participants analysed for specific category for this outcome measure. Age groups are based on actual age, therefore some participants are represented in multiple columns. | Posted | | Mean | Standard Deviation | IU/kg/year | | From week 0 to EOT (approximately week 544) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) |
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| Secondary | FIX Consumption Described as Amount Consumed for the Treatment of Bleeding Episodes | Average dose of nonacog beta pegol for treatment of bleed from start to stop of bleed is presented from week 0 to EOT approximately (week 544) in international units per kilogram per bleed (IU/Kg/bleed). Data is reported for specific Age Groups in which participants were a part of at any time from week 0 to EOT, not at specific time points assessed from week 0 to EOT (approximately week 544). Considering the time frame, actual age groups (adolescents and adults) are added for this outcome measure. | The FAS included all participants with efficacy data after exposure to nonacog beta pegol. Here, overall number of participants analysed = participants who experienced bleeding episodes. Number analysed = participants analysed for specific category for this outcome measure. Age groups are based on actual age, therefore some participants are represented in multiple columns. | Posted | | Mean | Standard Deviation | IU/kg/bleed | | From week 0 to EOT (approximately week 544) | Bleeds | Bleeds | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) |
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| Secondary | Number of Doses of FIX Consumed for the Treatment of Bleeding Episodes | Number of doses of FIX consumed for the treatment of bleeding episodes is presented from week 0 to EOT approximately (week 544). Here, data of number of doses of FIX for the treatment of bleeding episodes is reported among all the participants in an arm. Data is reported for specific Age Groups in which participants were a part of at any time from week 0 to EOT, not at specific time points assessed from week 0 to EOT (approximately week 544). Considering the time frame, actual age groups (adolescents and adults) are added for this outcome measure. | The FAS included all participants with efficacy data after exposure to nonacog beta pegol. Here, overall number of participants analysed = participants who experienced bleeding episodes. Number analysed = participants analysed for specific category for this outcome measure. Age groups are based on actual age, therefore some participants are represented in multiple columns. | Posted | | Number | | Doses of FIX | | From week 0 to EOT (approximately week 544) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) |
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| Secondary | Area Under the Curve Activity Versus Time Profile From Time Zero to 168 Hours Post Dose (AUC(0-168)) | Area under the curve activity versus time profile from time zero to 168 hours post dose of nonacog beta pegol is presented. | The FAS included all participants with efficacy data after exposure to nonacog beta pegol. | Posted | | Mean | Standard Deviation | Units*hour per milliliter (U*h/mL) | | 0-168 hours post-dosing at week 0 | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | Clearance (CL) | Clearance of nonacog beta pegol after single dose is presented. | The FAS included all participants with efficacy data after exposure to nonacog beta pegol. | Posted | | Mean | Standard Deviation | Milliliters/hour/kilogram (mL/h/kg) | | 0-168 hours post-dosing at week 0 | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | Mean Residence Time (MRT) | Mean residence time (MRT) of nonacog beta pegol after single dose is presented. | The FAS included all participants with efficacy data after exposure to nonacog beta pegol. | Posted | | Mean | Standard Deviation | Hour (h) | | 0-168 hours post-dosing at week 0 | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | Volume of Distribution at Steady State (Vss) | Volume of distribution at steady state (Vss) of nonacog beta pegol is presented. | The FAS included all participants with efficacy data after exposure to nonacog beta pegol. | Posted | | Mean | Standard Deviation | Milliliters per kilogram (mL/kg) | | 0-168 hours post-dosing at week 0 | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | FIX Activity at 30 Minutes (C30min) (Single Dose) | FIX activity (international units per milliliter (IU/mL)) at 30 minutes after single dose is presented. | The FAS included all participants with efficacy data after exposure to nonacog beta pegol. Here, overall number of participants analyzed = participants with available data. | Posted | | Mean | Standard Deviation | IU/mL | | 30 min post-dosing at week 0 | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | FIX Activity at 30 Minutes (C30min) (Steady State) | Mean FIX activity at 30 minutes post-dosing from week 4 to EOT approximately (week 544) (C30min) (steady state) is presented. | The FAS included all participants with efficacy data after exposure to nonacog beta pegol. | Posted | | Geometric Mean | 95% Confidence Interval | IU/mL | | 30 min post-dosing from week 4 to EOT (approximately week 544) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | TNO-AZL Preschool Quality of Life (TAPQOL) | The Dutch institute of Prevention and Health and the Leiden University Hospital (TNO-AZL) preschool quality of life clustered into 12 multi-item scales is used to assess the health-related quality of life, such as children's motor, communication, emotions, and body structure. Suitable for children from 6 months to 6 years old (TAPQOL). Parents fill in according to the child's condition. Higher score (range 0-100) represents better outcome. The scale range for each of 12 multi-item scales was (0-100) with high score representing better outcome. In this study, the TAPQOL was assessed for children of age 0-3 years. | The FAS included all participants with efficacy data after exposure to nonacog beta pegol. Here, overall number of participants analysed = participants who experienced bleeding episodes. Number analysed = participants analysed for specific category for this outcome measure. | Posted | | Mean | Standard Deviation | Score on scale | | Screening (Week -6), week 52, week 176 approximately (visit 17) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-3 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | Number of Participants With Health Economic Impact of N9-GP Treatment Through Characterisation of General Hospitalisation | Number of participants with health economic impact of N9-GP treatment through characterisation of general hospitalisation is presented. Number of participants hospitalised for 0 & 1 day is presented. | The FAS included all participants with efficacy data after exposure to nonacog beta pegol. | Posted | | Count of Participants | | Participants | | From week 0 to EOT (approximately week 544) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | Health Economic Impact of N9-GP Treatment Through Characterisation of Intensive Care Hospitalisation | Health economic impact of N9-GP treatment is presented through number of intensive care hospitalization days. | The FAS included all participants with efficacy data after exposure to nonacog beta pegol. Here, overall number of participants analyzed = number of participants with available data. Number analysed = participants analysed for specific category for this outcome measure. | Posted | | Mean | Standard Deviation | Days | | From week 0 to EOT (approximately week 544) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | Health Economic Impact of N9-GP Treatment Through Characterisation of Bleedings Caused Missing School or Studies | Health economic impact of N9-GP treatment through number of days bleedings caused missing school or studies. Number of participants who missed school or studies for 0,1 and 2 days are presented. | The FAS included all participants with efficacy data after exposure to nonacog beta pegol. Here, overall number of participants analyzed = number of participants with available data. | Posted | | Count of Participants | | Participants | | From week 0 to EOT (approximately week 544) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | Health Economic Impact of N9-GP Treatment Through Characterisation of Number of Days Bleedings Caused Using of Mobility Aids | Health economic impact of N9-GP treatment through number of days the patient used mobility aids (wheelchair and/or crutches) is presented. | The FAS included all participants with efficacy data after exposure to nonacog beta pegol. Here, overall number of participants analyzed = number of participants with available data. | Posted | | Mean | Standard Deviation | Days | | From week 0 to EOT (approximately week 544) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | Health Economic Impact of N9-GP Treatment Through Characterisation of Number of Days Bleedings Caused Parents to Miss Work | Health economic impact of N9-GP treatment through number of days bleeding caused parents to miss work is presented from week 0 to EOT approximately (week 544). | The FAS included all participants with efficacy data after exposure to nonacog beta pegol. Here, overall number of participants analyzed = number of participants with available data. | Posted | | Mean | Standard Deviation | Days | | From week 0 to EOT (approximately week 544) | | | | ID | Title | Description |
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| OG000 | Younger Children (0-6 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | | OG001 | Older Children (7-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | Haemophilia-quality of Life (HAEMO-QOL) | Haemophilia quality of life clustered into 11 multi-item scale which is used to assess haemophilia related quality of life such as children's physical health, feeling, family, friends, sport, treatment, dealing with haemophilia, view of a patient. Here, HAEMO-QOL was assessed for the children of age group 8-12 years. The scale range for each of 11 multi-item scale was 0-100 with high scores indicating low quality of life. Multi-item scores and total score were calculated using the following formula: 1/4(Sum of answered items / number of answered items - 1) x 100. HAEMO-QOL scores range from a 0 to 100 scale where high scores (nearing 100) indicate a low quality of life rating. | FAS included all participants with efficacy data after exposure to nonacog beta pegol. Here, overall number of participants analysed=participants who experienced bleeding episodes. Number analysed = participants analysed for specific category for this outcome measure. | Posted | | Mean | Standard Deviation | Score on scale | | Screening (week -6), week 52, EOT (approximately week 544) | | | | ID | Title | Description |
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| OG000 | Younger Children (8-12 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. |
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| Secondary | Hemophilia Treatment Satisfaction (HEMO-SAT) | Haemophilia treatment satisfaction change from baseline (screening week -6) to week 176 (visit 17) is presented. The treatment satisfaction of a bleed with N8-GP was assessed using HEMO-SAT assessment tool which contains a questionnaire with 6 domains (Ease and convenience, efficacy, burden, specialist/nurses, centre/hospital, general satisfaction). The scale range for each 6 domains was (0-100) with lower score indicating higher treatment satisfaction. HEMO-SAT was assessed for the children of age group 4-7 years and 8-12 years. Domain score and total score were calculated using following formula - 1/4 (sum of answered items / Number of answered item - 1) x 100. HEMO-SAT scores range from 0-100, where low scores reflecting greater treatment satisfaction. | FAS included all participants with efficacy data after exposure to nonacog beta pegol. Here, overall number of participants analysed = participants who experienced bleeding episodes. Number analysed = participants analysed for specific category for this outcome measure. | Posted | | Mean | Standard Deviation | Score on scale | | Screening (Week -6), week 176 (visit 17) | | | | ID | Title | Description |
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| OG000 | Younger Children (4-7 Years) | Participants received nonacog beta pegol 40 U/kg I.V. once weekly for 52 weeks in main phase and extension phase till end of trial. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg I.V. Severe bleeding episodes were treated immediately with 80 U/kg. | |
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