A Phase 3 interventional study of turoctocog alfa pegol in Congenital Bleeding Disorder and Haemophilia A, sponsored by Novo Nordisk A/S. Completed at 96 sites in 23 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 turoctocog alfa pegol (N8-GP) in previously untreated patients (PUPs) with haemophilia A.
844 studies on the registry are indexed under Hemophilia A; 138 are open to participants now.
This study's enrollment of 124 is above the median of 30 across 499 interventional studies indexed under Hemophilia A.
Browse Hemophilia A 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: turoctocog alfa pegol
For intravenous (i.v.) injection. Frequency and dosage (20-75 U/kg) dependent on whether given as treatment for bleeding episode or as prophylaxis
Also known as: NNC 0129-0000-1003, N8-GP
Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII)
Number of participants with inhibitory antibodies against coagulation factor VIII (FVIII) was reported during the main and extension phase of the trial.
Time frame: From start of the treatment up to 7 years
Number of Adverse Events Including Serious Adverse Events and Medical Events of Special Interest
Number of adverse events including serious adverse events and medical events of special interest reported during the main and extension phase of the trial. An adverse event (AE) is any untoward medical occurrence in a patient administered a product, and which does not necessarily have a causal relationship with this treatment. Serious adverse event (SAE) is an experience that at any dose results in any of the following: Death, a life-threatening experience, in-patient hospitalisation or prolongation of existing hospitalisation, persistent or significant disability or incapacity, congenital anomaly or birth defect and important medical events that may not result in death, be life threatening or require hospitalisation. Medical event of special interest (MESI) is an event which, in the evaluation of safety, has a special focus.
Time frame: From start of the treatment up to 8.9 years
Number of Participants With Confirmed High Titre Inhibitors (Defined as Inhibitor Titre Above 5 Bethesda Units (BU)
Number of participants with confirmed high titre inhibitors (defined as inhibitor titre above 5 Bethesda Units (BU) was reported during the main and extension phase of the trial.
Time frame: From start of the treatment up to 8.9 years
Number of Breakthrough Bleeding Episodes During Prophylaxis With N8-GP (Annualised Bleeding Rate)
The number of bleeding episodes per year reported during the prophylactic treatment with N8-GP was reported.
Time frame: From start of the treatment up to 8.9 years
Haemostatic Effect of N8-GP in Treatment of Bleeding Episodes, Assessed by a Predefined 4-point Haemostatic Response Scale ("Excellent", "Good", "Moderate" and "None")
Haemostatic effect of turoctocog alfa pegol for treatment of bleeding episodes was assessed by 4-point response scale: none, moderate, good or excellent. Excellent: Abrupt pain relief and/or clear improvement in objective signs of bleeding within approximately 8 hours after a single injection; Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hours after a single injection, but possibly requiring more than one injection for complete resolution; Moderate: Probable or slight beneficial effect within approximately 8 hours after the first injection, but usually requiring more than one injection; None: No improvement, or worsening of symptoms. Number of bleeding episodes in each category is reported.
Time frame: From start of the treatment up to 8.9 years
Consumption of N8-GP for Prophylaxis (International Unit Per Kilogram (IU/Kg))
Consumption of N8-GP for prophylaxis is reported. Consumption used for treatment includes all doses given. Yearly consumption is only calculated for patients with a exposure time of at least 30 days. Average dose consumption in IU/kg during main and extension phase is reported.
Time frame: From start of the treatment up to 8.9 years
Consumption of N8-GP for Prophylaxis (Number of Injections)
Consumption of N8-GP for prophylaxis is reported. Consumption used for treatment includes all doses given. Yearly consumption is only calculated for patients with a exposure time of at least 30 days. Number of injections consumption per patient during main and extension phase is reported.
Time frame: From start of the treatment up to 8.9 years
Consumption of N8-GP for Treatment of Bleeding Episodes (International Unit Per Kilogram Per Bleed (IU/kg/Bleed))
Consumption of N8-GP for treatment of bleeding episodes (IU/kg/bleed) is reported. Average dose consumption in IU/kg/bleed during main and extension phase is reported.
Time frame: From start of the treatment up to 8.9 years
Consumption of N8-GP for Treatment of Bleeding Episodes (Number of Injections)
Consumption of N8-GP for treatment of bleeding episodes (number of injections) is reported. Number of average injections required for treatment of per bleed is reported.
Time frame: From start of the treatment up to 8.9 years
Total Consumption of N8-GP Per Patient (Prevention and Treatment of Bleeding Episodes) Annualised Value
Total consumption of N8-GP per patient (prevention and treatment of bleeding episodes) annualised value is presented. Consumption used for treatment includes all doses given. Yearly consumption is only calculated for patients with a exposure time of at least 30 days. Consumption used for treatment per year per patient (IU/kg/year/patient) is reported.
Time frame: From start of the treatment up to 8.9 years
Outcome of ITI, Assessed by a Predefined 4-point ITI Outcome Scale ("Success", "Partial Success", "Failure", "Other")
The outcome of ITI was evaluated by predefined 4-point outcome of ITI scale ('success', 'partial success', 'failure', 'other'). Success: An inhibitor titre less than (\<) 0.6 BU. A normalised FVIII recovery, defined as greater than or equal to (≥) 66 percentage (%) of expected incremental recovery. An N8-GP half-life (t½) ≥9 hours after a 72-hour treatment-free washout period. Partial success: Reduction in inhibitor titre to less than or equal to (≤) 5 BU. Clinical effect of N8-GP therapy as judged by the investigator. Failure: Failure to attain defined success or partial success within 24 months of uninterrupted ITI with N8-GP. Inhibitor decrease less than (\<) 20% after one year of ITI treatment. Other: Not fulfilling the above criteria. Number of participants in each category is reported.
Time frame: From start of the treatment up to 8.9 years
The trial was conducted in 14 countries as follows: Australia (2), Austria (2), Bulgaria (1), Canada (1), Germany (1), Greece (2), Israel (1), Italy (1), Japan (3), Malaysia (3), Spain (2), Taiwan (1), Thailand (3), United States (8).
| Milestone | Pre-prophylaxis | Prophylaxis | Immune Tolerance Induction (ITI) |
|---|---|---|---|
| Started | 55 | 0 | 0 |
| Completed | 44 | 0 | 0 |
| Not completed | 11 | 0 | 0 |
| Withdrew: Withdrawal by parent/guardian | 1 | 0 | 0 |
| Withdrew: Protocol violation | 1 | 0 | 0 |
| Withdrew: Adverse event | 3 | 0 | 0 |
| Withdrew: Withdrawal by subject | 2 | 0 | 0 |
| Withdrew: Lack of efficacy | 1 | 0 | 0 |
| Withdrew: Other | 3 | 0 | 0 |
| Milestone | Pre-prophylaxis | Prophylaxis | Immune Tolerance Induction (ITI) |
|---|---|---|---|
| Started | 0 | 69 | 0 |
| Completed | 0 | 55 | 0 |
| Not completed | 0 | 14 | 0 |
| Withdrew: Withdrawal by parent/guardian | 0 | 2 | 0 |
| Withdrew: Protocol violation | 0 | 1 | 0 |
| Withdrew: Adverse event | 0 | 4 | 0 |
| Withdrew: Lack of efficacy | 0 | 1 | 0 |
| Withdrew: Other | 0 | 1 | 0 |
| Withdrew: Switched to iti without completing main phase | 0 | 2 | 0 |
| Withdrew: Early site closure | 0 | 2 | 0 |
| Withdrew: Lost to follow-up | 0 | 1 | 0 |
| Milestone | Pre-prophylaxis | Prophylaxis | Immune Tolerance Induction (ITI) |
|---|---|---|---|
| Started | 0 | 0 | 8 |
| Completed | 0 | 0 | 4 |
| Not completed | 0 | 0 | 4 |
| Withdrew: Withdrawal by parent/guardian | 0 | 0 | 1 |
| Withdrew: Lack of efficacy | 0 | 0 | 1 |
| Withdrew: Undergone minor surgery | 0 | 0 | 2 |
| Milestone | Pre-prophylaxis | Prophylaxis | Immune Tolerance Induction (ITI) |
|---|---|---|---|
| Started | 0 | 55 | 0 |
| Completed | 0 | 49 | 0 |
| Not completed | 0 | 6 | 0 |
| Withdrew: Adverse event | 0 | 1 | 0 |
| Withdrew: Protocol violation | 0 | 1 | 0 |
| Withdrew: Withdrawal by parent/guardian | 0 | 1 | 0 |
| Withdrew: Other | 0 | 1 | 0 |
| Withdrew: Early site closure | 0 | 2 | 0 |
Number of participants with inhibitory antibodies against coagulation factor VIII (FVIII) was reported during the main and extension phase of the trial.
| Participants | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) | 11 | 10 |
Number of adverse events including serious adverse events and medical events of special interest reported during the main and extension phase of the trial. An adverse event (AE) is any untoward medical occurrence in a patient administered a product, and which does not necessarily have a causal relationship with this treatment. Serious adverse event (SAE) is an experience that at any dose results in any of the following: Death, a life-threatening experience, in-patient hospitalisation or prolongation of existing hospitalisation, persistent or significant disability or incapacity, congenital anomaly or birth defect and important medical events that may not result in death, be life threatening or require hospitalisation. Medical event of special interest (MESI) is an event which, in the evaluation of safety, has a special focus.
| Events | Pre-prophylaxis | Prophylaxis | Immune Tolerance Induction (ITI) |
|---|---|---|---|
| Adverse events | 116 | 644 | 32 |
| Serious adverse events | 24 | 56 | 3 |
| Medical events of special interest | 17 | 47 | 0 |
Number of participants with confirmed high titre inhibitors (defined as inhibitor titre above 5 Bethesda Units (BU) was reported during the main and extension phase of the trial.
| Participants | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| Number of Participants With Confirmed High Titre Inhibitors (Defined as Inhibitor Titre Above 5 Bethesda Units (BU) | 3 | 8 |
The number of bleeding episodes per year reported during the prophylactic treatment with N8-GP was reported.
| bleeds/patient/year | Prophylaxis |
|---|---|
| Number of Breakthrough Bleeding Episodes During Prophylaxis With N8-GP (Annualised Bleeding Rate) | 1.35 (0.60 to 3.50) |
Haemostatic effect of turoctocog alfa pegol for treatment of bleeding episodes was assessed by 4-point response scale: none, moderate, good or excellent. Excellent: Abrupt pain relief and/or clear improvement in objective signs of bleeding within approximately 8 hours after a single injection; Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hours after a single injection, but possibly requiring more than one injection for complete resolution; Moderate: Probable or slight beneficial effect within approximately 8 hours after the first injection, but usually requiring more than one injection; None: No improvement, or worsening of symptoms. Number of bleeding episodes in each category is reported.
| Bleeding episodes | Pre-prophylaxis | Prophylaxis | Immune Tolerance Induction (ITI) |
|---|---|---|---|
| Excellent | 69 | 227 | 2 |
| Good | 62 | 92 | 3 |
| Moderate | 18 | 18 | 3 |
| None | 3 | 6 | 0 |
| Missing | 8 | 2 | 0 |
Consumption of N8-GP for prophylaxis is reported. Consumption used for treatment includes all doses given. Yearly consumption is only calculated for patients with a exposure time of at least 30 days. Average dose consumption in IU/kg during main and extension phase is reported.
| IU/kg | Prophylaxis |
|---|---|
| Consumption of N8-GP for Prophylaxis (International Unit Per Kilogram (IU/Kg)) | 68.9 ± 7.6 |
Consumption of N8-GP for prophylaxis is reported. Consumption used for treatment includes all doses given. Yearly consumption is only calculated for patients with a exposure time of at least 30 days. Number of injections consumption per patient during main and extension phase is reported.
| Injections/patient | Prophylaxis |
|---|---|
| Consumption of N8-GP for Prophylaxis (Number of Injections) | 236.6 ± 208.2 |
Consumption of N8-GP for treatment of bleeding episodes (IU/kg/bleed) is reported. Average dose consumption in IU/kg/bleed during main and extension phase is reported.
| IU/kg/bleed | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| Consumption of N8-GP for Treatment of Bleeding Episodes (International Unit Per Kilogram Per Bleed (IU/kg/Bleed)) | 112.9 ± 113.4 | 92.1 ± 71.7 |
Consumption of N8-GP for treatment of bleeding episodes (number of injections) is reported. Number of average injections required for treatment of per bleed is reported.
| Injections/bleed | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| Consumption of N8-GP for Treatment of Bleeding Episodes (Number of Injections) | 1.6 ± 1.6 | 1.3 ± 1.1 |
Total consumption of N8-GP per patient (prevention and treatment of bleeding episodes) annualised value is presented. Consumption used for treatment includes all doses given. Yearly consumption is only calculated for patients with a exposure time of at least 30 days. Consumption used for treatment per year per patient (IU/kg/year/patient) is reported.
| IU/kg/year/patient | Pre-prophylaxis | Prophylaxis |
|---|---|---|
| Total Consumption of N8-GP Per Patient (Prevention and Treatment of Bleeding Episodes) Annualised Value | 2108.6 ± 2697.6 | 5394.9 ± 1887.9 |
The outcome of ITI was evaluated by predefined 4-point outcome of ITI scale ('success', 'partial success', 'failure', 'other'). Success: An inhibitor titre less than (\<) 0.6 BU. A normalised FVIII recovery, defined as greater than or equal to (≥) 66 percentage (%) of expected incremental recovery. An N8-GP half-life (t½) ≥9 hours after a 72-hour treatment-free washout period. Partial success: Reduction in inhibitor titre to less than or equal to (≤) 5 BU. Clinical effect of N8-GP therapy as judged by the investigator. Failure: Failure to attain defined success or partial success within 24 months of uninterrupted ITI with N8-GP. Inhibitor decrease less than (\<) 20% after one year of ITI treatment. Other: Not fulfilling the above criteria. Number of participants in each category is reported.
| Participants | Immune Tolerance Induction (ITI) |
|---|---|
| Success | 4 |
| Partial success | 2 |
| Failure | 2 |
Collected over From start of the treatment up to 8.9 years. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Pre-prophylaxis | 0/55 (0%) | 18/55 (32.7%) | 28/55 (50.9%) |
| Prophylaxis | 0/69 (0%) | 34/69 (49.3%) | 51/69 (73.9%) |
| Immune Tolerance Induction (ITI) | 0/8 (0%) | 1/8 (12.5%) | 7/8 (87.5%) |
| Event | Pre-prophylaxis | Prophylaxis | Immune Tolerance Induction (ITI) |
|---|---|---|---|
| Factor VIII inhibitionBlood and lymphatic system disorders | 10/55 | 12/69 | 0/8 |
| Device issueProduct Issues | 0/55 | 0/69 | 1/8 |
| Device related infectionInfections and infestations | 0/55 | 0/69 | 1/8 |
| Head injuryInjury, poisoning and procedural complications | 2/55 | 1/69 | 0/8 |
| Anti factor VIII antibody positiveInvestigations | 0/55 | 2/69 | 0/8 |
| PyrexiaGeneral disorders | 0/55 | 2/69 | 0/8 |
| BronchiolitisInfections and infestations | 1/55 | 0/69 | 0/8 |
| Cerebral haemorrhageNervous system disorders | 1/55 | 1/69 | 0/8 |
| FallInjury, poisoning and procedural complications | 1/55 | 0/69 | 0/8 |
| Febrile convulsionNervous system disorders | 1/55 | 0/69 | 0/8 |
| Event | Pre-prophylaxis | Prophylaxis | Immune Tolerance Induction (ITI) |
|---|---|---|---|
| PyrexiaGeneral disorders | 5/55 | 26/69 | 2/8 |
| NasopharyngitisInfections and infestations | 6/55 | 22/69 | 1/8 |
| Upper respiratory tract infectionInfections and infestations | 7/55 | 16/69 | 2/8 |
| CoughRespiratory, thoracic and mediastinal disorders | 3/55 | 9/69 | 0/8 |
| Ear infectionInfections and infestations | 3/55 | 9/69 | 0/8 |
| GastroenteritisInfections and infestations | 1/55 | 9/69 | 0/8 |
| AnaemiaBlood and lymphatic system disorders | 2/55 | 0/69 | 1/8 |
| Anaesthetic complicationInjury, poisoning and procedural complications | 0/55 | 0/69 | 1/8 |
| BronchitisInfections and infestations | 1/55 | 6/69 | 1/8 |
| CellulitisInfections and infestations | 0/55 | 1/69 | 1/8 |
All participants that started the study.
| Age, Continuous(Years) | All Participants |
|---|---|
| Mean | 10.2 ± 11.0 |
| Sex: Female, Male(Participants) | All Participants |
|---|---|
| Female | 0 |
| Male | 81 |
| Ethnicity (NIH/OMB)(Participants) | All Participants |
|---|---|
| Hispanic or Latino | 7 |
| Not Hispanic or Latino | 74 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | All Participants |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 24 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 2 |
| White | 49 |
| More than one race | 0 |
| Unknown or Not Reported | 6 |
PDFs hosted by the registry, ClinicalTrials.gov.
Ran from Jun 2014 to primary completion in Jun 2023, 107 months.
Results were first posted in Oct 2024, 1 year 4 months after primary completion.
Of 89 industry-sponsored phase 3 interventional studies under Hemophilia A, half reached primary completion within 32 months of starting (the median), counting those still running; a quarter within 18 and three quarters within 56. This study's 107 months is above that.
Counting every sponsor, half of 91 studies reached it within 33 months; a quarter within 20 and three quarters within 56.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Dec 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Turoctocog alfa pegol