Trial · NCT01333111
Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients
NCT01333111, Phase 3, Completed, Novo Nordisk A/S; results first posted 2017-07-28
3 sourcestrials-2026-11-12-0.5.0
Download JSON
Cite this record
ConductScience Library. Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients. Trials, record NCT01333111. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01333111
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients
- NCT number
- NCT01333111
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 74 participants, actual
- Lead sponsor
- Novo Nordisk A/S
- Conditions
- Congenital Bleeding Disorder, Haemophilia B
- Interventions
- nonacog beta pegol, nonacog beta pegol, nonacog beta pegol
- First posted
- 2011-04-11
- Last updated
- 2017-07-28
- Other registries' IDs
- eudract:2010-023069-24
- Results first posted
- 2017-07-28
Source ClinicalTrials.gov
Registered outcomes12
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Incidence of Inhibitory Antibodies Against Factor IX Defined as Titre Equal to or Above 0.6 BU (Bethesda Units) | 52 weeks after treatment start for patients on prophylaxis | Inhibitors were analysed with either the Nijmegen modified factor IX Bethesda assay or a heat/cold Nijmegen modified factor IX Bethesda assay. Number of subjects who developed inhibitory antibodies against factor IX are reported. |
| Primary | Incidence of Inhibitory Antibodies Against Factor IX Defined as Titre Equal to or Above 0.6 BU (Bethesda Units) | 28 weeks after treatment start on on-demand treatment | Inhibitors were analysed with either the Nijmegen modified factor IX Bethesda assay or a heat/cold Nijmegen modified factor IX Bethesda assay. Number of subjects who developed inhibitory antibodies against factor IX are reported. |
| Secondary | Haemostatic Effect of NNC-0156-0000-0009 When Used for Prophylaxis of Bleeding Episodes, Assessed as Success/Failure Based on a Four-point Scale for Haemostatic Response | 52 weeks after treatment start for patients on prophylaxis | Haemostatic response after treatment of a bleed with nonacog beta pegol was evaluated on a 4 point scale as excellent, good, moderate or poor. A success rate was calculated based on counting good or excellent as successes and poor and moderate as failures. * Excellent - abrupt pain relief and/or clear improvement in objective signs of bleeding within 8 hours after a single injection * 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 injection within 8 hours * Poor - no improvement, or worsening of symptoms within 8 hours after two injections. The success rate and 95% confidence interval (CI) are reported here. |
| Secondary | Haemostatic Effect of NNC-0156-0000-0009 When Used for Treatment of Bleeding Episodes, Assessed as Success/Failure Based on a Four-point Scale for Haemostatic Response | 28 weeks after treatment start on on-demand treatment | Haemostatic response after treatment of a bleed with nonacog beta pegol was evaluated on a 4 point scale as excellent, good, moderate or poor. A success rate was calculated based on counting good or excellent as successes and poor and moderate as failures. * Excellent - abrupt pain relief and/or clear improvement in objective signs of bleeding within 8 hours after a single injection * 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 injection within 8 hours * Poor - no improvement, or worsening of symptoms within 8 hours after two injections. The success rate and 95% confidence interval (CI) are reported here. |
| Secondary | Number of Bleeding Episodes Per Patient During Routine Prophylaxis | 52 weeks after treatment start for patients on prophylaxis | The number of bleeding episodes per patient during routine prophylaxis was assessed using the individual annualised bleeding rates (spontaneous and traumatic bleeding episodes per patient per year). |
| Secondary | Factor IX Trough Levels | 52 weeks after treatment start for patients on prophylaxis | The mean pre-dose factor IX levels was measured with the one-stage clotting assay during the trial. Lowest factor IX activity recorded during single-dose and steady state, immediately before next dose was given. The analysis was based on a mixed model on the log-transformed plasma factor IX activity with subject as a random effect. The estimated mean factor IX trough level was presented back-transformed to the natural scale. |
| Secondary | Incidence of Adverse Events (AEs) | at 56 weeks ±2 weeks for patients on prophylaxis | The incidence of adverse events were summarised by the rate of AEs (number of AEs per patient years of exposure \[PYE\]). Number of adverse events per PYE is number of adverse events /total time in trial. All adverse events reported are treatment emergent (any adverse events which occurred after trial product administration). |
| Secondary | Incidence of Adverse Events (AEs) | at 32 weeks ±2 weeks for patients on on-demand treatment | The incidence of adverse events were summarised by the rate of AEs (number of AEs per PYE). Number of adverse events per PYE is number of adverse events /total time in trial. All adverse events reported are treatment emergent (any adverse events which occurred after trial product administration). |
| Secondary | Incidence of Serious Adverse Events (SAEs) | at 56 weeks ±2 weeks for patients on prophylaxis | SAE was defined as an AE that resulted in any of the following: death, a life-threatening experience, in-subject hospitalization/prolongation of existing hospitalisation, persistent/significant disability/incapacity/congenital anomaly/birth defect. The incidence of SAEs were summarised by the rate of SAEs (number of SAEs per PYE). Number of SAEs per PYE is number of SAEs/total time in trial. All SAEs reported are treatment emergent (any serious adverse events which occurred after trial product administration). |
| Secondary | Incidence of Serious Adverse Events (SAEs) | at 32 weeks ±2 weeks for patients on on-demand treatment | SAE was defined as an AE that resulted in any of the following: death, a life-threatening experience, in-subject hospitalization/prolongation of existing hospitalisation, persistent/significant disability/incapacity/congenital anomaly/birth defect. The incidence of SAEs were summarised by the rate of SAEs (number of SAEs per PYE). Number of SAEs per PYE is number of SAEs/total time in trial. All SAEs reported are treatment emergent (any serious adverse events which occurred after trial product administration). |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 registered outcomesOutcomes12
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Incidence of Inhibitory Antibodies Against Factor IX Defined as Titre Equal to or Above 0.6 BU (Bethesda Units) | 52 weeks after treatment start for patients on prophylaxis | Full analysis set included all subjects exposed to nonacog beta pegol. Subjects in prophylaxis arm were included for this analysis. | Number of subjects | NUMBER |
| PRIMARY | Incidence of Inhibitory Antibodies Against Factor IX Defined as Titre Equal to or Above 0.6 BU (Bethesda Units) | 28 weeks after treatment start on on-demand treatment | Full analysis set included all subjects exposed to nonacog beta pegol. Subjects in on-demand arm were included for this analysis. | number of subjects | NUMBER |
| SECONDARY | Haemostatic Effect of NNC-0156-0000-0009 When Used for Prophylaxis of Bleeding Episodes, Assessed as Success/Failure Based on a Four-point Scale for Haemostatic Response | 52 weeks after treatment start for patients on prophylaxis | Full analysis set included all subjects exposed to nonacog beta pegol. Subjects in prophylaxis arm were included for this analysis. | percentage of bleeding episodes | NUMBER |
| SECONDARY | Haemostatic Effect of NNC-0156-0000-0009 When Used for Treatment of Bleeding Episodes, Assessed as Success/Failure Based on a Four-point Scale for Haemostatic Response | 28 weeks after treatment start on on-demand treatment | Full analysis set included all subjects exposed to nonacog beta pegol. Subjects in on-demand arm were included for this analysis. | percentage of bleeding episodes | NUMBER |
| SECONDARY | Number of Bleeding Episodes Per Patient During Routine Prophylaxis | 52 weeks after treatment start for patients on prophylaxis | Full analysis set included all subjects exposed to nonacog beta pegol. Subjects in prophylaxis arm were included for this analysis. | bleeds/patient/year | MEDIAN |
| SECONDARY | Factor IX Trough Levels | 52 weeks after treatment start for patients on prophylaxis | Full analysis set included all subjects exposed to nonacog beta pegol. Subjects in prophylaxis arm were included for this analysis. | U/mL | MEAN |
| SECONDARY | Incidence of Adverse Events (AEs) | at 56 weeks ±2 weeks for patients on prophylaxis | Safety analysis set included all subjects exposed to nonacog beta pegol. Subjects in prophylaxis arm were included for this analysis. | number of AEs per PYE | NUMBER |
| SECONDARY | Incidence of Adverse Events (AEs) | at 32 weeks ±2 weeks for patients on on-demand treatment | Safety analysis set included all subjects exposed to nonacog beta pegol.Subjects in on-demand arm were included for this analysis. | number of AEs per PYE | NUMBER |
| SECONDARY | Incidence of Serious Adverse Events (SAEs) | at 56 weeks ±2 weeks for patients on prophylaxis | Safety analysis set included all subjects exposed to nonacog beta pegol. Subjects in prophylaxis arm were included for this analysis. | number of SAEs per PYE | NUMBER |
| SECONDARY | Incidence of Serious Adverse Events (SAEs) | at 32 weeks ±2 weeks for patients on on-demand treatment | Safety analysis set included all subjects exposed to nonacog beta pegol. Subjects in on-demand arm were included for this analysis. | number of SAEs per PYE | NUMBER |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 outcomesResults by group19
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Incidence of Inhibitory Antibodies Against Factor IX Defined as Titre Equal to or Above 0.6 BU (Bethesda Units) | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | 0 | 0 to 0 | Number of subjects | |
| Incidence of Inhibitory Antibodies Against Factor IX Defined as Titre Equal to or Above 0.6 BU (Bethesda Units) | Prophylaxis, High Dose 40 U/kg (52 Weeks) | 0 | 0 to 0 | Number of subjects | |
| Incidence of Inhibitory Antibodies Against Factor IX Defined as Titre Equal to or Above 0.6 BU (Bethesda Units) | On-Demand (28 Weeks) | 0 | number of subjects | ||
| Haemostatic Effect of NNC-0156-0000-0009 When Used for Prophylaxis of Bleeding Episodes, Assessed as Success/Failure Based on a Four-point Scale for Haemostatic Response | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | 86.9 | 95% Confidence Interval 76.5 to 93.1 | percentage of bleeding episodes | |
| Haemostatic Effect of NNC-0156-0000-0009 When Used for Prophylaxis of Bleeding Episodes, Assessed as Success/Failure Based on a Four-point Scale for Haemostatic Response | Prophylaxis, High Dose 40 U/kg (52 Weeks) | 97.1 | 95% Confidence Interval 90.0 to 99.2 | percentage of bleeding episodes | |
| Haemostatic Effect of NNC-0156-0000-0009 When Used for Treatment of Bleeding Episodes, Assessed as Success/Failure Based on a Four-point Scale for Haemostatic Response | On-Demand (28 Weeks) | 95.1 | 95% Confidence Interval 87.0 to 98.2 | percentage of bleeding episodes | |
| Number of Bleeding Episodes Per Patient During Routine Prophylaxis | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | 2.93 | Inter-Quartile Range 5.41 | bleeds/patient/year | |
| Number of Bleeding Episodes Per Patient During Routine Prophylaxis | Prophylaxis, High Dose 40 U/kg (52 Weeks) | 1.04 | Inter-Quartile Range 7.41 | bleeds/patient/year | |
| Factor IX Trough Levels | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | 0.085 | 95% Confidence Interval 0.077 to 0.093 | U/mL | |
| Factor IX Trough Levels | Prophylaxis, High Dose 40 U/kg (52 Weeks) | 0.273 | 95% Confidence Interval 0.248 to 0.300 | U/mL |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | 30 | |
| Overall Study | STARTED | Prophylaxis, High Dose 40 U/kg (52 Weeks) | 29 | |
| Overall Study | STARTED | On-Demand (28 Weeks) | 15 | |
| Overall Study | COMPLETED | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | 28 | |
| Overall Study | COMPLETED | Prophylaxis, High Dose 40 U/kg (52 Weeks) | 26 | |
| Overall Study | COMPLETED | On-Demand (28 Weeks) | 13 | |
| Overall Study | NOT COMPLETED | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | 2 | |
| Overall Study | NOT COMPLETED | Prophylaxis, High Dose 40 U/kg (52 Weeks) | 3 | |
| Overall Study | NOT COMPLETED | On-Demand (28 Weeks) | 2 |
Source ClinicalTrials.gov
Adverse events126
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | 2 | 30 | 2 |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | Prophylaxis, High Dose 40 U/kg (52 Weeks) | 1 | 29 | 2 |
| other | Arthralgia | Musculoskeletal and connective tissue disorders | On-Demand (28 Weeks) | 1 | 15 | 2 |
| other | Attention deficit/hyperactivity disorder | Psychiatric disorders | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | 0 | 30 | 0 |
| other | Attention deficit/hyperactivity disorder | Psychiatric disorders | Prophylaxis, High Dose 40 U/kg (52 Weeks) | 0 | 29 | 0 |
| other | Attention deficit/hyperactivity disorder | Psychiatric disorders | On-Demand (28 Weeks) | 1 | 15 | 1 |
| other | Back pain | Musculoskeletal and connective tissue disorders | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | 2 | 30 | 2 |
| other | Back pain | Musculoskeletal and connective tissue disorders | Prophylaxis, High Dose 40 U/kg (52 Weeks) | 0 | 29 | 0 |
| other | Back pain | Musculoskeletal and connective tissue disorders | On-Demand (28 Weeks) | 0 | 15 | 0 |
| other | C-reactive protein increased | Investigations | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | 4 | 30 | 4 |
1 to 10 of 126Source ClinicalTrials.gov
See all 126 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | ||
| Total, all-cause mortality | Prophylaxis, High Dose 40 U/kg (52 Weeks) | ||
| Total, all-cause mortality | On-Demand (28 Weeks) | ||
| Total, other adverse events | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | 21 | 30 |
| Total, other adverse events | Prophylaxis, High Dose 40 U/kg (52 Weeks) | 19 | 29 |
| Total, other adverse events | On-Demand (28 Weeks) | 11 | 15 |
| Total, serious adverse events | Prophylaxis, Low Dose 10 U/kg (52 Weeks) | 1 | 30 |
| Total, serious adverse events | Prophylaxis, High Dose 40 U/kg (52 Weeks) | 3 | 29 |
| Total, serious adverse events | On-Demand (28 Weeks) | 0 | 15 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 25261199 | BACKGROUND |
Source PubMed
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01333111
GET https://conductscience.org/api/trials/v1/NCT01333111/events?page=2