A Phase 3 interventional study of nonacog beta pegol in Congenital Bleeding Disorder and Haemophilia B, sponsored by Novo Nordisk A/S. Completed at 51 sites in 12 countries. Open to male participants aged 0 Years to 12 Years. Per ClinicalTrials.gov, last updated 2025-12-23.
Sponsored by Novo Nordisk A/S · Phase 3, Interventional, and Treatment
This trial is conducted in Asia, Europe and North America. The aim of the trial is to evaluate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC-0156-0000-0009 (nonacog beta pegol, N9-GP) in previously treated children with Haemophilia B.
271 studies on the registry are indexed under Hemophilia B; 51 are open to participants now.
This study's enrollment of 25 is close to the median of 25 across 156 interventional studies indexed under Hemophilia B.
Browse Hemophilia B studies →Novo Nordisk A/S is the lead sponsor of 1,370 studies on the registry; 102 are open to participants now.
Of its 198 completed or terminated interventional studies of FDA-regulated products, 94 (47%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Drug: nonacog beta pegol
A single dose of 40 U/kg will be administered intravenously, i.v. (into the vein) once weekly.
Also known as: NNC-0156-0000-0009
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.
Time frame: From week 0 to week 52
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.
Time frame: From week 52 to End of trial (EOT) (approximately week 544)
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).
Time frame: From week 0 to EOT (approximately week 544)
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: 1. Excellent - abrupt pain relief and/or clear improvement in objective signs of bleeding within 8 hours after a single infusion. 2. Good - noticeable pain relief and/or improvement in signs of bleeding within 8 hours after a single injection. 3. Moderate - probable or slight beneficial effect within the first 8 hours after the first injection but requiring more than one infusion within 8 hours. 4. 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.
Time frame: From week 0 to EOT (approximately week 544)
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.
Time frame: Week 0 (30 minutes after first exposure)
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).
Time frame: Week 0 (one week after first exposure)
Terminal Half-life (t1/2)
Terminal half life is presented at week 0, 30 minutes until one week after first exposure.
Time frame: Week 0 (30 minutes until one week after first exposure)
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.
Time frame: From week 4 to EOT (approximately week 544)
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.
Time frame: Week 0 to EOT (approximately week 544)
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.
Time frame: Week 0 to EOT (approximately week 544)
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.
Time frame: Week 0 to EOT (approximately week 544)
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.
Time frame: From week 0 to EOT (approximately week 544)
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.
Time frame: From week 0 to EOT (approximately week 544)
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.
Time frame: From week 0 to EOT (approximately week 544)
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.
Time frame: From week 0 to EOT (approximately week 544)
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.
Time frame: 0-168 hours post-dosing at week 0
Clearance (CL)
Clearance of nonacog beta pegol after single dose is presented.
Time frame: 0-168 hours post-dosing at week 0
Mean Residence Time (MRT)
Mean residence time (MRT) of nonacog beta pegol after single dose is presented.
Time frame: 0-168 hours post-dosing at week 0
Volume of Distribution at Steady State (Vss)
Volume of distribution at steady state (Vss) of nonacog beta pegol is presented.
Time frame: 0-168 hours post-dosing at week 0
FIX Activity at 30 Minutes (C30min) (Single Dose)
FIX activity (international units per milliliter (IU/mL)) at 30 minutes after single dose is presented.
Time frame: 30 min post-dosing at week 0
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.
Time frame: 30 min post-dosing from week 4 to EOT (approximately week 544)
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.
Time frame: Screening (Week -6), week 52, week 176 approximately (visit 17)
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.
Time frame: From week 0 to EOT (approximately week 544)
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.
Time frame: From week 0 to EOT (approximately week 544)
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.
Time frame: From week 0 to EOT (approximately week 544)
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.
Time frame: From week 0 to EOT (approximately week 544)
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).
Time frame: From week 0 to EOT (approximately week 544)
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.
Time frame: Screening (week -6), week 52, EOT (approximately week 544)
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.
Time frame: Screening (Week -6), week 176 (visit 17)
Of the 19 sites that screened participants, 17 sites enrolled participants. The trial was therefore conducted at 17 sites in 8 countries, as follows: Canada: 1 site; Germany: 1 site; Italy: 1 site; Japan: 3 sites; Malaysia: 1 site; Taiwan: 1 site; United Kingdom: 3 sites; United States: 6 sites
| Milestone | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| Started | 12 | 13 |
| Completed main phase | 11 | 13 |
| Entered extension phase | 11 | 11 |
| Completed | 6 | 4 |
| Not completed | 6 | 9 |
| Withdrew: Withdrawal by subject | 1 | 0 |
| Withdrew: Not continuing in extension phase | 0 | 2 |
| Withdrew: Withdrawal during extension phase,- non-compliance | 0 | 1 |
| Withdrew: Withdrawal during extension phase, withdrawal criteria | 0 | 2 |
| Withdrew: Withdrawal during extension phase, withdrawal of consent | 2 | 1 |
| Withdrew: Withdrawal during extension phase, other | 3 | 3 |
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.
| Participants | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU) | 0 | 0 |
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.
| Participants | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU) | 0 | 0 |
The number of bleeding episodes per participant during routine prophylaxis was assessed using the individual annualised bleeding rates (bleeding episodes per participant per year).
| bleeds/participant/year | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| Number of Bleeding Episodes During Prophylaxis | 0.33 (0.05 to 0.87) | 0.78 (0.35 to 1.73) |
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: 1. Excellent - abrupt pain relief and/or clear improvement in objective signs of bleeding within 8 hours after a single infusion. 2. Good - noticeable pain relief and/or improvement in signs of bleeding within 8 hours after a single injection. 3. Moderate - probable or slight beneficial effect within the first 8 hours after the first injection but requiring more than one infusion within 8 hours. 4. 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.
| percentage of bleeding episodes | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| Success | 90.3 | 89.5 |
| Failure | 9.7 | 10.5 |
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.
| (U/mL)/(U/kg) | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| Incremental Recovery at 30 Minutes (IR30min) | 0.015 ± 7.31 | 0.016 ± 16.18 |
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).
| U/mL | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| Trough Level (Single-dose ) | 0.084 ± 16.28 | 0.109 ± 18.89 |
Terminal half life is presented at week 0, 30 minutes until one week after first exposure.
| hours | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| Terminal Half-life (t1/2) | 69.576 ± 15.79 | 76.323 ± 25.48 |
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.
| U/mL | Younger Children (0-6 Years) | Older Children (7-12 Years) | Adolescents(13-17 Years) | Adult (18-70 Years) |
|---|---|---|---|---|
| Trough Level (Steady State) | 0.146 (0.127 to 0.166) | 0.193 (0.171 to 0.218) | 0.220 (0.192 to 0.252) | 0.316 (0.254 to 0.394) |
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.
| Events | Younger Children (0-6 Years) | Older Children (7-12 Years) | Adolescents(13-17 Years) | Adult (18-70 Years) |
|---|---|---|---|---|
| Number of Adverse Events | 252 | 342 | 81 | 10 |
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.
| Events | Younger Children (0-6 Years) | Older Children (7-12 Years) | Adolescents(13-17 Years) | Adult (18-70 Years) |
|---|---|---|---|---|
| Number of Serious Adverse Events (SAEs) | 3 | 5 | 1 | 0 |
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.
| Events | Younger Children (0-6 Years) | Older Children (7-12 Years) | Adolescents(13-17 Years) | Adult (18-70 Years) |
|---|---|---|---|---|
| Medical Events of Special Interest (MESI) | 1 | 3 | 5 | 1 |
Participants were examined for the development of antibodies against HCP. Number of participants who developed antibodies against HCP is presented.
| Participants | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| Yes | 1 | 0 |
| No | 11 | 13 |
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.
| IU/kg/year | Younger Children (0-6 Years) | Older Children (7-12 Years) | Adolescents(13-17 Years) | Adult (18-70 Years) |
|---|---|---|---|---|
| FIX Consumption Described as Frequency of Dose/kg for Prophylaxis Use for the Treatment of Bleeding Episodes | 2209.3 ± 79.3 | 2324.9 ± 83.5 | 2257.6 ± 121.1 | 1959.5 ± 533.4 |
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.
| IU/kg/bleed | Younger Children (0-6 Years) | Older Children (7-12 Years) | Adolescents(13-17 Years) | Adult (18-70 Years) |
|---|---|---|---|---|
| FIX Consumption Described as Amount Consumed for the Treatment of Bleeding Episodes | 53.6 ± 32.2 | 50.1 ± 19.4 | 84.3 ± 110.2 | 59.9 ± 32.7 |
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.
| Doses of FIX | Younger Children (0-6 Years) | Older Children (7-12 Years) | Adolescents(13-17 Years) | Adult (18-70 Years) |
|---|---|---|---|---|
| Number of Doses of FIX Consumed for the Treatment of Bleeding Episodes | 1768 | 4201 | 2740 | 747 |
Area under the curve activity versus time profile from time zero to 168 hours post dose of nonacog beta pegol is presented.
| Units*hour per milliliter (U*h/mL) | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| Area Under the Curve Activity Versus Time Profile From Time Zero to 168 Hours Post Dose (AUC(0-168)) | 37.764 ± 4.586 | 44.192 ± 7.300 |
Clearance of nonacog beta pegol after single dose is presented.
| Milliliters/hour/kilogram (mL/h/kg) | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| Clearance (CL) | 0.764 ± 0.102 | 0.664 ± 0.147 |
Mean residence time (MRT) of nonacog beta pegol after single dose is presented.
| Hour (h) | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| Mean Residence Time (MRT) | 96.332 ± 12.995 | 108.16 ± 29.937 |
Volume of distribution at steady state (Vss) of nonacog beta pegol is presented.
| Milliliters per kilogram (mL/kg) | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| Volume of Distribution at Steady State (Vss) | 73.046 ± 10.843 | 69.752 ± 15.253 |
FIX activity (international units per milliliter (IU/mL)) at 30 minutes after single dose is presented.
| IU/mL | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| FIX Activity at 30 Minutes (C30min) (Single Dose) | 0.544 ± 0.040 | 0.600 ± 0.074 |
Mean FIX activity at 30 minutes post-dosing from week 4 to EOT approximately (week 544) (C30min) (steady state) is presented.
| IU/mL | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| FIX Activity at 30 Minutes (C30min) (Steady State) | 0.174 (0.146 to 0.208) | 0.197 (0.166 to 0.234) |
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.
| Score on scale | Younger Children (0-3 Years) |
|---|---|
| Screening (week -6) : Sleeping problems | 80.4 ± 15.3 |
| Week 52: Sleeping problems | 76.0 ± 15.5 |
| Screening (week -6) : Appetite | 91.7 ± 11.8 |
| Week 52: Appetite | 90.3 ± 15.2 |
| Screening (week -6) : Lung problems | 100.0 ± 0.0 |
| Week 52: Lung problems | 93.2 ± 13.2 |
| Screening (week -6) : Stomach problems | 93.0 ± 12.1 |
| Week 52: Stomach problems | 90.3 ± 15.2 |
| Screening (week -6) : Skin problems | 88.1 ± 17.9 |
| Week 52: Skin problems | 90.3 ± 16.3 |
| Screening (week -6) : Motor functioning | 98.0 ± 4.9 |
| Week 52: Motor functioning | 98.0 ± 4.9 |
| Screening (week -6): Social functioning | 97.2 ± 6.9 |
| Week 52: Social functioning | 97.2 ± 6.9 |
| Screening (week -6) : Problem behavior | 58.1 ± 40.8 |
| Week 52: Problem behavior | 53.7 ± 29.6 |
| Screening (week -6) : Communication | 92.8 ± 14.8 |
| Week 52: Communication | 87.5 ± 14.2 |
| Screening (week -6) : Anxiety | 76.1 ± 16.1 |
| Week 52: Anxiety | 75.0 ± 22.9 |
| Screening (week -6) : Positive mood | 92.9 ± 18.9 |
| Week 52: Positive mood | 100.0 ± 0.0 |
| Screening (week -6) : Liveliness | 97.6 ± 6.4 |
| Week 52: Liveliness | 100.0 ± 0.0 |
| Approximately week 176: sleeping problem | 89.7 ± 15.0 |
| Approximately week 176: Appetite | 94.5 ± 10.1 |
| Approximately week 176: Lung problems | 97.2 ± 6.9 |
| Approximately week 176: Stomach problems | 83.2 ± 10.4 |
| Approximately week 176: Skin problems | 84.7 ± 20.0 |
| Approximately week 176: Motor functioning | 99.0 ± 2.4 |
| Approximately week 176: Social functioning | 86.2 ± 22.1 |
| Approximately week 176: Problem behavior | 58.3 ± 19.3 |
| Approximately week 176: Communication | 95.8 ± 10.2 |
| Approximately week 176: Anxiety | 80.2 ± 18.1 |
| Approximately week 176: Positive mood | 100.0 ± 0.0 |
| Approximately week 176: Liveliness | 94.3 ± 8.8 |
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.
| Participants | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| 0 Days | 3 | 5 |
| 1 Day | 1 | 0 |
Health economic impact of N9-GP treatment is presented through number of intensive care hospitalization days.
| Days | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| Health Economic Impact of N9-GP Treatment Through Characterisation of Intensive Care Hospitalisation | 0.0 ± 0.0 | 0.0 ± 0.0 |
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.
| Participants | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| 0 days | 9 | 11 |
| 1 day | 1 | 0 |
| 2 days | 1 | 2 |
Health economic impact of N9-GP treatment through number of days the patient used mobility aids (wheelchair and/or crutches) is presented.
| Days | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| Health Economic Impact of N9-GP Treatment Through Characterisation of Number of Days Bleedings Caused Using of Mobility Aids | 0.4 ± 0.7 | 1.0 ± 3.3 |
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).
| Days | Younger Children (0-6 Years) | Older Children (7-12 Years) |
|---|---|---|
| Health Economic Impact of N9-GP Treatment Through Characterisation of Number of Days Bleedings Caused Parents to Miss Work | 0.3 ± 0.6 | 0.5 ± 0.9 |
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.
| Score on scale | Younger Children (8-12 Years) |
|---|---|
| Screening (week -6) : physical health | 19.8 ± 19.1 |
| Week 52: physical health | 18.2 ± 17.0 |
| EOT approximately week 544: physical health | 7.0 ± 9.9 |
| Screening (week -6) : feeling | 15.5 ± 19.3 |
| Week 52: feeling | 12.8 ± 22.9 |
| EOT approximately week 544: feeling | 12.5 ± 17.7 |
| Screening (week -6) : view | 25.8 ± 18.6 |
| Week 52: view | 20.1 ± 22.5 |
| EOT approximately week 544: view | 23.5 ± 33.2 |
| Screening (week -6) : family | 25.0 ± 16.5 |
| Week 52: family | 16.3 ± 20.4 |
| EOT approximately week 544: family | 17.5 ± 24.7 |
| Screening (week -6): friends | 54.3 ± 23.0 |
| Week 52: friends | 49.0 ± 22.3 |
| EOT approximately week 544: friends | 43.5 ± 53.0 |
| Screening (week -6) : perceived support | 72.5 ± 26.1 |
| Week 52: perceived support | 71.5 ± 32 |
| EOT approximately week 544: perceived support | 34.5 ± 48.8 |
| Screening (week -6) : others | 14.3 ± 12.5 |
| Week 52: others | 14.5 ± 17.3 |
| EOT approximately week 544: others | 8.5 ± 12.0 |
| Screening (week -6) : sport | 20.8 ± 19.4 |
| Week 52: sport | 22.8 ± 18.7 |
| EOT approximately week 544: sport | 28.0 ± 26.9 |
| Screening (week -6) : dealing | 56.5 ± 27.4 |
| Week 52: dealing | 49.6 ± 22.0 |
| EOT approximately week 544: dealing | 41.5 ± 53.0 |
| Screening (week -6) : treatment | 34.7 ± 16.7 |
| Week 52: treatment | 25.1 ± 19.8 |
| EOT approximately week 544: treatment | 11.0 ± 0.0 |
| Screening (week -6) : total | 31.7 ± 10.1 |
| Week 52: total | 28.0 ± 12.5 |
| EOT approximately week 544: total | 21.5 ± 26.2 |
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.
| Score on scale | Younger Children (4-7 Years) | Older Children (8-12 Years) |
|---|---|---|
| Ease and convenience | -6.5 ± 19.4 | -13.4 ± 16.2 |
| Efficacy | -3.8 ± 21.3 | -14.5 ± 23.8 |
| Burden | -15.8 ± 16.0 | -8.5 ± 13.0 |
| Specialist/nurses | -1.8 ± 2.1 | -0.6 ± 2.1 |
| Centre/hospital | 5.0 ± 13.5 | -2.3 ± 6.1 |
| General satisfaction | -6.3 ± 12.5 | -3.5 ± 8.1 |
Collected over Week 0 to EOT (approximately week 544).. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Younger Children (0 - 6 Years) | 0/12 (0%) | 2/12 (16.7%) | 11/12 (91.7%) |
| Older Children (7 - 12 Years) | 0/23 (0%) | 4/23 (17.4%) | 20/23 (87%) |
| Adolescents (13 - 17 Years) | 0/14 (0%) | 1/14 (7.1%) | 12/14 (85.7%) |
| Adults (18 - 70 Years) | 0/6 (0%) | 0/6 (0%) | 4/6 (66.7%) |
| Event | Younger Children (0 - 6 Years) | Older Children (7 - 12 Years) | Adolescents (13 - 17 Years) | Adults (18 - 70 Years) |
|---|---|---|---|---|
| Catheter site infectionInfections and infestations | 1/12 | 0/23 | 0/14 | 0/6 |
| Tourette's disorderCongenital, familial and genetic disorders | 1/12 | 0/23 | 0/14 | 0/6 |
| Viral upper respiratory tract infectionInfections and infestations | 1/12 | 0/23 | 0/14 | 0/6 |
| HaemoptysisRespiratory, thoracic and mediastinal disorders | 0/12 | 0/23 | 1/14 | 0/6 |
| CryptorchismCongenital, familial and genetic disorders | 0/12 | 1/23 | 0/14 | 0/6 |
| Food poisoningGastrointestinal disorders | 0/12 | 1/23 | 0/14 | 0/6 |
| Gastroenteritis viralInfections and infestations | 0/12 | 1/23 | 0/14 | 0/6 |
| MigraineNervous system disorders | 0/12 | 1/23 | 0/14 | 0/6 |
| Radius fractureInjury, poisoning and procedural complications | 0/12 | 1/23 | 0/14 | 0/6 |
| Event | Younger Children (0 - 6 Years) | Older Children (7 - 12 Years) | Adolescents (13 - 17 Years) | Adults (18 - 70 Years) |
|---|---|---|---|---|
| CoughRespiratory, thoracic and mediastinal disorders | 10/12 | 5/23 | 1/14 | 1/6 |
| PyrexiaGeneral disorders | 7/12 | 6/23 | 2/14 | 0/6 |
| NasopharyngitisInfections and infestations | 5/12 | 4/23 | 2/14 | 0/6 |
| Skin abrasionInjury, poisoning and procedural complications | 4/12 | 3/23 | 0/14 | 0/6 |
| ContusionInjury, poisoning and procedural complications | 3/12 | 7/23 | 1/14 | 0/6 |
| Head injuryInjury, poisoning and procedural complications | 2/12 | 6/23 | 1/14 | 0/6 |
| Pain in extremityMusculoskeletal and connective tissue disorders | 1/12 | 6/23 | 2/14 | 0/6 |
| DiarrhoeaGastrointestinal disorders | 3/12 | 2/23 | 0/14 | 0/6 |
| Ear infectionInfections and infestations | 3/12 | 2/23 | 0/14 | 0/6 |
| FallInjury, poisoning and procedural complications | 3/12 | 4/23 | 0/14 | 0/6 |
| Age, Continuous(years) | Younger Children (0-6 Years) | Older Children (7-12 Years) | Total |
|---|---|---|---|
| Mean | 3.1 ± 1.7 | 9.6 ± 1.6 | 6.5 ± 3.7 |
| Sex: Female, Male(Participants) | Younger Children (0-6 Years) | Older Children (7-12 Years) | Total |
|---|---|---|---|
| Female | 0 | 0 | 0 |
| Male | 12 | 13 | 25 |
| Ethnicity (NIH/OMB)(Participants) | Younger Children (0-6 Years) | Older Children (7-12 Years) | Total |
|---|---|---|---|
| Hispanic or Latino | 0 | 2 | 2 |
| Not Hispanic or Latino | 12 | 11 | 23 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race/Ethnicity, Customized(Participants) | Younger Children (0-6 Years) | Older Children (7-12 Years) | Total |
|---|---|---|---|
| Race — Asian | 4 | 4 | 8 |
| Race — Black or African American | 0 | 1 | 1 |
| Race — White | 8 | 5 | 13 |
| Race — Other | 0 | 3 | 3 |
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