A Phase 3 interventional study of nonacog beta pegol in Congenital Bleeding Disorder and Haemophilia B, sponsored by Novo Nordisk A/S. Completed at 93 sites in 24 countries. Open to male participants aged 0 Years to 6 Years. Per ClinicalTrials.gov, last updated 2025-12-23.
Sponsored by Novo Nordisk A/S · Phase 3, Interventional, and Treatment
This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of nonacog beta pegol (N9-GP) in previously untreated patients with Haemophilia B.
292 studies on the registry are indexed under Hemophilia B; 51 are open to participants now.
This study's enrollment of 54 is above the median of 26 across 171 interventional studies indexed under Hemophilia B.
Browse Hemophilia B studies →Novo Nordisk A/S is the lead sponsor of 1,404 studies on the registry; 101 are open to participants now.
Of its 205 completed or terminated interventional studies of FDA-regulated products, 100 (49%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Drug: nonacog beta pegol
For intravenous (i.v.) injection. A single dose of 40 U/kg, unless the bleeding episode is severe in which case it should be treated with 80 U/kg.
Number of Participants With Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) (50 Exposure Days)
Number of participants with incidence of inihibitory antibodies against FIX after 50 ED is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.
Time frame: When minimum 20 previously untreated patients (PUPs) have reached at least 50 exposure days (ED) (up to 156 weeks)
Number of Participants With Incidence of Inhibitory Antibodies Against FIX (100 ED)
Number of participants with incidence of inihibitory antibodies against FIX after 100 ED is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.
Time frame: When minimum 40 PUPs have reached at least 100 ED (up to 208 weeks)
Number of Participants With Incidence of Inhibitory Antibodies Against FIX (At End of Trial)
Number of participants with incidence of inihibitory antibodies against FIX at end of trial is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.
Time frame: At end of trial (up to 434 weeks)
Number of Adverse Events
Number of adverse events after 50 ED, after 100 ED, and at end of trial is presented. An adverse event was defined as any untoward medical occurrence in a participant who was administered a product, and which does not necessarily have a causal relationship with this treatment. All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment.
Time frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
Frequency of Adverse Events
Frequency of adverse events after 50 ED, after 100 ED, and at end of trial is presented. Frequency of adverse events was expressed as number of adverse events per participant years of exposure (total number of events /total time in trial). An adverse event was defined as any untoward medical occurrence in a participant who was administered a product, and which does not necessarily have a causal relationship with this treatment. All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment.
Time frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
Number of Serious Adverse Events
Number of serious adverse events after 50 ED, after 100 ED, and at end of trial is presented. A serious adverse event was an experience that at any dose resulted in: death; life-threatening experience; in-patient hospitalisation or prolongation of existing hospitalisation; a persistent or significant disability/incapacity; congenital anomaly/birth defect; important medical events that may not result in death, be life-threatening/require hospitalisation could be considered a serious adverse event based upon appropriate medical judgement.
Time frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
Frequency of Serious Adverse Events
Frequency of serious adverse events after 50 ED, after 100 ED, and at end of trial is presented. Frequency of serious adverse events was expressed as number of serious adverse events per participant years of exposure (total number of events /total time in trial). A serious adverse event was an experience that at any dose resulted in: death; life-threatening experience; in-patient hospitalisation or prolongation of existing hospitalisation; a persistent or significant disability/incapacity; congenital anomaly/birth defect; important medical events that may not result in death, be life-threatening/require hospitalisation could be considered a serious adverse event based upon appropriate medical judgement.
Time frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
Number of Medical Events of Special Interest
Number of medical events of special interest after 50 ED, after 100 ED, and at end of trial is presented. A medical event of special interest (MESI) was an event that, in the evaluation of safety, has a special focus. A MESI was an adverse event (serious or non-serious adverse event) that fulfils one or more of the following MESI criteria: 1. Medication errors concerning the trial product; 2. Inhibitor formation against FIX; 3. Thromboembolic events; 4. Anaphylactic reaction; 5. Allergic reaction including, but not limited to, any acute immunoglobulin E mediated reaction of delayed-type hypersensitivity (clinical signs may include various types of skin rashes) that does not meet the definition of anaphylaxis; 6. CNS-related adverse events including, but not limited to, any learning and behavioral deficits; 7. Renal adverse events including new onset of renal disorder or renal impairment or acute and chronic renal failure.
Time frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
Frequency of Medical Events of Special Interest
Frequency of medical events of special interest after 50 ED, after 100 ED, and at end of trial is presented. It was expressed as number of MESI per participant years of exposure (total number of events/total time in trial). A MESI was an event that, in the evaluation of safety, has a special focus. A MESI was an adverse event that fulfils one or more of the MESI criteria: 1. Medication errors concerning the trial product; 2. Inhibitor formation against FIX; 3. Thromboembolic events; 4. Anaphylactic reaction; 5. Allergic reaction including, but not limited to, any acute immunoglobulin E mediated reaction of delayed-type hypersensitivity (clinical signs may include various types of skin rashes) that does not meet the definition of anaphylaxis; 6. CNS-related adverse events including, but not limited to, any learning and behavioral deficits; 7. Renal adverse events including new onset of renal disorder or renal impairment or acute and chronic renal failure.
Time frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
Number of Breakthrough Bleeding Episodes During Prophylaxis (Annualised Bleeding Rate)
Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate) after 50 ED, after 100 ED, and at end of trial is presented. Annualised bleeding rate is the number of bleeding episodes per year.
Time frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
Haemostatic Effect of Nonacog Beta Pegol in Treatment of Bleeding Episodes by 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "Poor")
Haemostatic effect of nonacog beta pegol in treatment of bleeding episodes by 4-point haemostatic response scale after 50 ED, after 100 ED, and at end of trial is presented. The haemostatic response after treatment of a bleed with nonacog beta pegol was evaluated on a 4-point scale as excellent, good, moderate or poor. Excellent: abrupt pain relief and/or clear improvement in objective signs of bleeding; Good: noticeable pain relief and/or improvement in signs of bleeding; Moderate: probable or slight beneficial effect after the first injection; Poor: no improvement or worsening of symptoms. If the haemostatic response was rated as excellent or good, the treatment of the bleed was considered a success. If the haemostatic response was rated as moderate or poor, the treatment was considered a failure.
Time frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
Incremental Recovery at 30 Minutes (IR30min)
Incremental recovery 30 minutes post dosing (IR30min) after 50 ED, after 100 ED is presented. IR30min was defined as the rise in FIX activity per international units per kilogram (IU/kg) administered and was recorded 30 minutes after the end of nonacog beta pegol injection. It was calculated as the baseline adjusted FIX activity recorded 30 minutes after ended nonacog beta pegol injection divided by the administered dose (IU/kg body weight). It was recorded as international units per milliliter (IU/mL)/international units per kilogram (IU/kg).
Time frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks)
FIX Activity at 30 Minutes (C30min)
FIX Activity 30 minutes post dosing (C30min) after 50 ED, after 100 ED is presented. The FIX activity was measured by one-stage clotting assay - a modified activated partial thromboplastin time (aPTT) assay.
Time frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks)
FIX Trough Levels
FIX trough levels after 50 ED, after 100 ED, and at end of trial is presented. FIX trough level was defined as the activity recorded immediately before nonacog beta pegol injection was given and was measured by one-stage clotting assay - a modified activated partial thromboplastin time (aPTT) assay. The analysis is based on a mixed model on the log-transformed plasma concentrations with participant as a random effect. The mean trough level is presented back-transformed to the natural scale.
Time frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
Amount of Drug Administered to Treat a Bleeding Episode
Amount of nonacog beta pegol administered (average dose of nonacog beta pegol) to treat a bleeding episode after 50 ED, after 100 ED, and at end of trial is presented as international units per kilogram per bleed (IU/kg/bleed).
Time frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
Number of Injections Needed to Treat a Bleeding Episode
The mean number of injections needed to treat a bleeding episode is presented.
Time frame: When minimum 20 PUPs have reached at least 50 ED (up to 156 weeks); when minimum 40 PUPs have reached at least 100 ED (up to 208 weeks); at end of trial (up to 434 weeks)
The trial was conducted at 29 sites in 11 countries as follows (number of sites that screened participants/ number of sites that randomised participants): Australia (1/1); Austria (2/2); Canada (1/1); Israel (1/1); Japan (1/1); Malaysia (4/4); Spain (3/3); Taiwan (2/2); Thailand (2/2); United Kingdom (3/3); United States (9/9).
| Milestone | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| Started | 34 | 0 |
| Completed | 31 | 0 |
| Not completed | 3 | 0 |
| Withdrew: Adverse event | 3 | 0 |
| Milestone | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| Started | 0 | 51 |
| Completed | 0 | 41 |
| Not completed | 0 | 10 |
| Withdrew: Withdrawal by parent/guardian | 0 | 2 |
| Withdrew: Adverse event | 0 | 4 |
| Withdrew: Withdrawn due to closure of trial | 0 | 3 |
| Withdrew: Withdrawal by subject | 0 | 1 |
Number of participants with incidence of inihibitory antibodies against FIX after 50 ED is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.
| Participants | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| Number of Participants With Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) (50 Exposure Days) | 2 | 0 |
Number of participants with incidence of inihibitory antibodies against FIX after 100 ED is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.
| Participants | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| Number of Participants With Incidence of Inhibitory Antibodies Against FIX (100 ED) | 2 | 2 |
Number of participants with incidence of inihibitory antibodies against FIX at end of trial is presented and defined as an inhibitory antibody titre greater than equal to 0.6 Bethesda unit (BU) at two consecutive tests performed at the central laboratory and also tested positive for nonacog beta pegol binding antibodies.
| Participants | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| Number of Participants With Incidence of Inhibitory Antibodies Against FIX (At End of Trial) | 2 | 2 |
Number of adverse events after 50 ED, after 100 ED, and at end of trial is presented. An adverse event was defined as any untoward medical occurrence in a participant who was administered a product, and which does not necessarily have a causal relationship with this treatment. All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment.
| Events | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| 50 ED | 86 | 291 |
| 100 ED | 131 | 610 |
| End of trial (Week 434) | 134 | 794 |
Frequency of adverse events after 50 ED, after 100 ED, and at end of trial is presented. Frequency of adverse events was expressed as number of adverse events per participant years of exposure (total number of events /total time in trial). An adverse event was defined as any untoward medical occurrence in a participant who was administered a product, and which does not necessarily have a causal relationship with this treatment. All presented adverse events are treatment emergent adverse events, defined as an event that occured while the participant was on treatment in the period from first dosing with nonacog beta pegol to the end of trial/discontinuation of treatment.
| Events per participant years of exposure | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| 50 ED | 5.59 | 5.87 |
| 100 ED | 5.92 | 5.08 |
| End of trial (Week 434) | 5.52 | 4.10 |
Number of serious adverse events after 50 ED, after 100 ED, and at end of trial is presented. A serious adverse event was an experience that at any dose resulted in: death; life-threatening experience; in-patient hospitalisation or prolongation of existing hospitalisation; a persistent or significant disability/incapacity; congenital anomaly/birth defect; important medical events that may not result in death, be life-threatening/require hospitalisation could be considered a serious adverse event based upon appropriate medical judgement.
| Events | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| 50 ED | 9 | 14 |
| 100 ED | 13 | 27 |
| End of trial (Week 434) | 14 | 30 |
Frequency of serious adverse events after 50 ED, after 100 ED, and at end of trial is presented. Frequency of serious adverse events was expressed as number of serious adverse events per participant years of exposure (total number of events /total time in trial). A serious adverse event was an experience that at any dose resulted in: death; life-threatening experience; in-patient hospitalisation or prolongation of existing hospitalisation; a persistent or significant disability/incapacity; congenital anomaly/birth defect; important medical events that may not result in death, be life-threatening/require hospitalisation could be considered a serious adverse event based upon appropriate medical judgement.
| Events per participant years of exposure | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| 50 ED | 0.59 | 0.28 |
| 100 ED | 0.59 | 0.22 |
| End of trial (Week 434) | 0.58 | 0.15 |
Number of medical events of special interest after 50 ED, after 100 ED, and at end of trial is presented. A medical event of special interest (MESI) was an event that, in the evaluation of safety, has a special focus. A MESI was an adverse event (serious or non-serious adverse event) that fulfils one or more of the following MESI criteria: 1. Medication errors concerning the trial product; 2. Inhibitor formation against FIX; 3. Thromboembolic events; 4. Anaphylactic reaction; 5. Allergic reaction including, but not limited to, any acute immunoglobulin E mediated reaction of delayed-type hypersensitivity (clinical signs may include various types of skin rashes) that does not meet the definition of anaphylaxis; 6. CNS-related adverse events including, but not limited to, any learning and behavioral deficits; 7. Renal adverse events including new onset of renal disorder or renal impairment or acute and chronic renal failure.
| Events | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| 50 ED | 5 | 9 |
| 100 ED | 6 | 31 |
| End of trial (Week 434) | 6 | 38 |
Frequency of medical events of special interest after 50 ED, after 100 ED, and at end of trial is presented. It was expressed as number of MESI per participant years of exposure (total number of events/total time in trial). A MESI was an event that, in the evaluation of safety, has a special focus. A MESI was an adverse event that fulfils one or more of the MESI criteria: 1. Medication errors concerning the trial product; 2. Inhibitor formation against FIX; 3. Thromboembolic events; 4. Anaphylactic reaction; 5. Allergic reaction including, but not limited to, any acute immunoglobulin E mediated reaction of delayed-type hypersensitivity (clinical signs may include various types of skin rashes) that does not meet the definition of anaphylaxis; 6. CNS-related adverse events including, but not limited to, any learning and behavioral deficits; 7. Renal adverse events including new onset of renal disorder or renal impairment or acute and chronic renal failure.
| Events per participant years of exposure | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| 50 ED | 0.33 | 0.18 |
| 100 ED | 0.27 | 0.26 |
| End of trial (Week 434) | 0.25 | 0.20 |
Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate) after 50 ED, after 100 ED, and at end of trial is presented. Annualised bleeding rate is the number of bleeding episodes per year.
| bleeds/participant/year | Prophylaxis |
|---|---|
| 50 ED | 0.00 (0.00 to 2.60) |
| 100 ED | 0.25 (0.00 to 17.39) |
| End of trial (Week 434) | 0.33 (0.00 to 17.39) |
Haemostatic effect of nonacog beta pegol in treatment of bleeding episodes by 4-point haemostatic response scale after 50 ED, after 100 ED, and at end of trial is presented. The haemostatic response after treatment of a bleed with nonacog beta pegol was evaluated on a 4-point scale as excellent, good, moderate or poor. Excellent: abrupt pain relief and/or clear improvement in objective signs of bleeding; Good: noticeable pain relief and/or improvement in signs of bleeding; Moderate: probable or slight beneficial effect after the first injection; Poor: no improvement or worsening of symptoms. If the haemostatic response was rated as excellent or good, the treatment of the bleed was considered a success. If the haemostatic response was rated as moderate or poor, the treatment was considered a failure.
| Number of bleeds treated | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| 50 ED Excellent | 23 | 9 |
| 50 ED Good | 8 | 5 |
| 50 ED Moderate | 2 | 1 |
| 50 ED Poor | 0 | 0 |
| 100 ED Excellent | 44 | 44 |
| 100 ED Good | 17 | 30 |
| 100 ED Moderate | 2 | 3 |
| 100 ED Poor | 0 | 0 |
| End of trial (Week 434) Excellent | 45 | 86 |
| End of trial (Week 434) Good | 18 | 45 |
| End of trial (Week 434) Moderate | 2 | 4 |
| End of trial (Week 434) Poor | 0 | 0 |
Incremental recovery 30 minutes post dosing (IR30min) after 50 ED, after 100 ED is presented. IR30min was defined as the rise in FIX activity per international units per kilogram (IU/kg) administered and was recorded 30 minutes after the end of nonacog beta pegol injection. It was calculated as the baseline adjusted FIX activity recorded 30 minutes after ended nonacog beta pegol injection divided by the administered dose (IU/kg body weight). It was recorded as international units per milliliter (IU/mL)/international units per kilogram (IU/kg).
| (IU/mL)/(IU/kg) | Prophylaxis |
|---|---|
| 50 ED | 0.012 ± 13.9 |
| 100 ED | 0.014 ± 20.1 |
FIX Activity 30 minutes post dosing (C30min) after 50 ED, after 100 ED is presented. The FIX activity was measured by one-stage clotting assay - a modified activated partial thromboplastin time (aPTT) assay.
| IU/mL | Prophylaxis |
|---|---|
| 50 ED | 0.652 ± 39.5 |
| 100 ED | 0.824 ± 21.6 |
FIX trough levels after 50 ED, after 100 ED, and at end of trial is presented. FIX trough level was defined as the activity recorded immediately before nonacog beta pegol injection was given and was measured by one-stage clotting assay - a modified activated partial thromboplastin time (aPTT) assay. The analysis is based on a mixed model on the log-transformed plasma concentrations with participant as a random effect. The mean trough level is presented back-transformed to the natural scale.
| IU/mL | Prophylaxis |
|---|---|
| 50 ED | 0.150 (0.141 to 0.160) |
| 100 ED | 0.156 (0.144 to 0.170) |
| End of trial (Week 434) | 0.167 (0.155 to 0.179) |
Amount of nonacog beta pegol administered (average dose of nonacog beta pegol) to treat a bleeding episode after 50 ED, after 100 ED, and at end of trial is presented as international units per kilogram per bleed (IU/kg/bleed).
| IU/kg/bleed | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| 50 ED | 55.0 ± 28.7 | 48.8 ± 16.9 |
| 100 ED | 47.7 ± 17.6 | 58.5 ± 70.0 |
| End of trial (Week 434) | 47.5 ± 17.4 | 54.0 ± 53.5 |
The mean number of injections needed to treat a bleeding episode is presented.
| Injections per bleed | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| 50 ED | 1.3 ± 0.7 | 1.0 ± 0.0 |
| 100 ED | 1.1 ± 0.4 | 1.3 ± 1.5 |
| End of trial (Week 434) | 1.1 ± 0.4 | 1.2 ± 1.2 |
Collected over Up to Week 434. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Pre-prophylaxis | 1/34 (2.9%) | 9/34 (26.5%) | 22/34 (64.7%) |
| Prophylaxis | 1/51 (2%) | 19/51 (37.3%) | 46/51 (90.2%) |
| Event | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| PneumoniaInfections and infestations | 0/34 | 4/51 |
| Factor IX inhibitionBlood and lymphatic system disorders | 2/34 | 2/51 |
| Haemorrhage intracranialNervous system disorders | 2/34 | 1/51 |
| Dental cariesGastrointestinal disorders | 0/34 | 2/51 |
| Anaphylactic reactionImmune system disorders | 1/34 | 0/51 |
| BronchiolitisInfections and infestations | 1/34 | 0/51 |
| BronchitisInfections and infestations | 1/34 | 0/51 |
| FallInjury, poisoning and procedural complications | 1/34 | 0/51 |
| Gastroenteritis salmonellaInfections and infestations | 1/34 | 0/51 |
| Gastroenteritis viralInfections and infestations | 1/34 | 0/51 |
| Event | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| PyrexiaGeneral disorders | 11/34 | 27/51 |
| NasopharyngitisInfections and infestations | 5/34 | 23/51 |
| Upper respiratory tract infectionInfections and infestations | 5/34 | 19/51 |
| CoughRespiratory, thoracic and mediastinal disorders | 4/34 | 17/51 |
| DiarrhoeaGastrointestinal disorders | 3/34 | 11/51 |
| VomitingGastrointestinal disorders | 2/34 | 11/51 |
| Ear infectionInfections and infestations | 3/34 | 9/51 |
| RhinorrhoeaRespiratory, thoracic and mediastinal disorders | 3/34 | 9/51 |
| FallInjury, poisoning and procedural complications | 1/34 | 8/51 |
| Head injuryInjury, poisoning and procedural complications | 0/34 | 7/51 |
Full analysis set: all participants exposed to study drug. Participants switched from pre-prophylaxis treatment to prophylaxis treatment. Other participants directly started on prophylaxis treatment at visit 1. Pre-prophylaxis and Prophylaxis arms are not mutually exclusive so the data in this section is provided for all participants that started the study.
| Age, Continuous(years) | Nonacog Beta Pegol |
|---|---|
| Mean | 0.8 ± 1.1 |
| Sex: Female, Male(Participants) | Nonacog Beta Pegol |
|---|---|
| Female | 0 |
| Male | 54 |
| Ethnicity (NIH/OMB)(Participants) | Nonacog Beta Pegol |
|---|---|
| Hispanic or Latino | 2 |
| Not Hispanic or Latino | 52 |
| Unknown or Not Reported | 0 |
| Race/Ethnicity, Customized(Participants) | Nonacog Beta Pegol |
|---|---|
| Asian | 19 |
| Black or African American | 7 |
| White | 25 |
| Other | 3 |
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Studies of the drug tested here: Nonacog beta pegol