Trial · NCT01467427

Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children With Haemophilia B.

NCT01467427, Phase 3, Completed, Novo Nordisk A/S; results first posted 2017-11-17

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children With Haemophilia B. Trials, record NCT01467427. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01467427

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children With Haemophilia B.
NCT number: NCT01467427
Status: Completed
Phases: Phase 3
Enrollment: 25 participants, actual
Lead sponsor: Novo Nordisk A/S
Conditions: Congenital Bleeding Disorder, Haemophilia B
Interventions: nonacog beta pegol
First posted: 2011-11-08
Last updated: 2025-12-23
Other registries' IDs: eudract:2011-000826-31
Results first posted: 2017-11-17

Source ClinicalTrials.gov

Registered outcomes29

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU)	From week 0 to week 52	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.
Primary	Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU)	From week 52 to End of trial (EOT) (approximately week 544)	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.
Secondary	Number of Bleeding Episodes During Prophylaxis	From week 0 to EOT (approximately week 544)	The number of bleeding episodes per participant during routine prophylaxis was assessed using the individual annualised bleeding rates (bleeding episodes per participant per year).
Secondary	Haemostatic Effect of N9-GP in Treatment of Bleeding Episodes by 4-point Categorical Scale for Haemostatic Response (Excellent, Good, Moderate and Poor)	From week 0 to EOT (approximately week 544)	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.
Secondary	Incremental Recovery at 30 Minutes (IR30min)	Week 0 (30 minutes after first exposure)	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.
Secondary	Trough Level (Single-dose )	Week 0 (one week after first exposure)	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).
Secondary	Terminal Half-life (t1/2)	Week 0 (30 minutes until one week after first exposure)	Terminal half life is presented at week 0, 30 minutes until one week after first exposure.
Secondary	Trough Level (Steady State)	From week 4 to EOT (approximately week 544)	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.
Secondary	Number of Adverse Events	Week 0 to EOT (approximately week 544)	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.
Secondary	Number of Serious Adverse Events (SAEs)	Week 0 to EOT (approximately week 544)	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.

1 to 10 of 29Source ClinicalTrials.gov

See all 29 registered outcomes

Outcomes29

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 Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU)	From week 0 to week 52	Safety analysis set (SAS) included all participants exposed to nonacog beta pegol.	Participants	COUNT_OF_PARTICIPANTS
PRIMARY	Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU)	From week 52 to End of trial (EOT) (approximately week 544)	Safety analysis set (SAS) included all participants exposed to nonacog beta pegol.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Number of Bleeding Episodes During Prophylaxis	From week 0 to EOT (approximately week 544)	Full analysis set (FAS) included all participants with efficacy data after exposure to nonacog beta pegol.	bleeds/participant/year	MEDIAN
SECONDARY	Haemostatic Effect of N9-GP in Treatment of Bleeding Episodes by 4-point Categorical Scale for Haemostatic Response (Excellent, Good, Moderate and Poor)	From week 0 to EOT (approximately week 544)	FAS included all participants with efficacy data after exposure to nonacog beta pegol. Here, overall number of participants analysed=participants who experienced bleeding episodes. Number analysed = participants analysed for specific category for this outcome measure.	percentage of bleeding episodes	NUMBER
SECONDARY	Incremental Recovery at 30 Minutes (IR30min)	Week 0 (30 minutes after first exposure)	FAS included all participants with efficacy data after exposure to nonacog beta pegol. Here, overall number of participants analysed = Participants who were evaluated for this parameter.	(U/mL)/(U/kg)	GEOMETRIC_MEAN
SECONDARY	Trough Level (Single-dose )	Week 0 (one week after first exposure)	FAS included all participants with efficacy data after exposure to nonacog beta pegol. Here, overall number of participants analysed = Participants who were evaluated for this parameter.	U/mL	GEOMETRIC_MEAN
SECONDARY	Terminal Half-life (t1/2)	Week 0 (30 minutes until one week after first exposure)	FAS included all participants with efficacy data after exposure to nonacog beta pegol.	hours	GEOMETRIC_MEAN
SECONDARY	Trough Level (Steady State)	From week 4 to EOT (approximately week 544)	FAS included all participants with efficacy data after exposure to nonacog beta pegol.~Age groups are based on actual age, therefore some participants are represented in multiple columns.	U/mL	MEAN
SECONDARY	Number of Adverse Events	Week 0 to EOT (approximately week 544)	SAS included all participants exposed to nonacog beta pegol. Here, overall number of participants analysed = participants who experienced bleeding episodes. Number analysed = participants analysed for specific category for this outcome measure.~Age groups are based on actual age, therefore some participants are represented in multiple columns.	Events	NUMBER
SECONDARY	Number of Serious Adverse Events (SAEs)	Week 0 to EOT (approximately week 544)	SAS included all participants exposed to nonacog beta pegol. Here, overall number of participants analysed = participants who experienced bleeding episodes. Number analysed = participants analysed for specific category for this outcome measure.~Age groups are based on actual age, therefore some participants are represented in multiple columns.	Events	NUMBER

1 to 10 of 29Source ClinicalTrials.gov

See all 29 outcomes

Results by group157

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 Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU)	Younger Children (0-6 Years)		0		Participants
Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU)	Older Children (7-12 Years)		0		Participants
Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU)	Younger Children (0-6 Years)		0		Participants
Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU)	Older Children (7-12 Years)		0		Participants
Number of Bleeding Episodes During Prophylaxis	Younger Children (0-6 Years)		0.33	Inter-Quartile Range 0.05 to 0.87	bleeds/participant/year
Number of Bleeding Episodes During Prophylaxis	Older Children (7-12 Years)		0.78	Inter-Quartile Range 0.35 to 1.73	bleeds/participant/year
Haemostatic Effect of N9-GP in Treatment of Bleeding Episodes by 4-point Categorical Scale for Haemostatic Response (Excellent, Good, Moderate and Poor)	Younger Children (0-6 Years)	Success	90.3		percentage of bleeding episodes
Haemostatic Effect of N9-GP in Treatment of Bleeding Episodes by 4-point Categorical Scale for Haemostatic Response (Excellent, Good, Moderate and Poor)	Older Children (7-12 Years)	Success	89.5		percentage of bleeding episodes
Haemostatic Effect of N9-GP in Treatment of Bleeding Episodes by 4-point Categorical Scale for Haemostatic Response (Excellent, Good, Moderate and Poor)	Younger Children (0-6 Years)	Failure	9.7		percentage of bleeding episodes
Haemostatic Effect of N9-GP in Treatment of Bleeding Episodes by 4-point Categorical Scale for Haemostatic Response (Excellent, Good, Moderate and Poor)	Older Children (7-12 Years)	Failure	10.5		percentage of bleeding episodes

1 to 10 of 157Source ClinicalTrials.gov

See all 157 results by group

Participant flow10

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Younger Children (0-6 Years)	12
Overall Study	STARTED	Older Children (7-12 Years)	13
Overall Study	Completed Main Phase	Younger Children (0-6 Years)	11
Overall Study	Completed Main Phase	Older Children (7-12 Years)	13
Overall Study	Entered Extension Phase	Younger Children (0-6 Years)	11
Overall Study	Entered Extension Phase	Older Children (7-12 Years)	11
Overall Study	COMPLETED	Younger Children (0-6 Years)	6
Overall Study	COMPLETED	Older Children (7-12 Years)	4
Overall Study	NOT COMPLETED	Younger Children (0-6 Years)	6
Overall Study	NOT COMPLETED	Older Children (7-12 Years)	9

Source ClinicalTrials.gov

Adverse events532

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	Abdominal discomfort	Gastrointestinal disorders	Younger Children (0 - 6 Years)	0	12	0
other	Abdominal discomfort	Gastrointestinal disorders	Older Children (7 - 12 Years)	2	23	2
other	Abdominal discomfort	Gastrointestinal disorders	Adolescents (13 - 17 Years)	0	14	0
other	Abdominal discomfort	Gastrointestinal disorders	Adults (18 - 70 Years)	0	6	0
other	Abdominal injury	Injury, poisoning and procedural complications	Younger Children (0 - 6 Years)	1	12	1
other	Abdominal injury	Injury, poisoning and procedural complications	Older Children (7 - 12 Years)	1	23	1
other	Abdominal injury	Injury, poisoning and procedural complications	Adolescents (13 - 17 Years)	0	14	0
other	Abdominal injury	Injury, poisoning and procedural complications	Adults (18 - 70 Years)	0	6	0
other	Abdominal pain	Gastrointestinal disorders	Younger Children (0 - 6 Years)	1	12	3
other	Abdominal pain	Gastrointestinal disorders	Older Children (7 - 12 Years)	1	23	1

1 to 10 of 532Source ClinicalTrials.gov

See all 532 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Younger Children (0 - 6 Years)	0	12
Total, all-cause mortality	Older Children (7 - 12 Years)	0	23
Total, all-cause mortality	Adolescents (13 - 17 Years)	0	14
Total, all-cause mortality	Adults (18 - 70 Years)	0	6
Total, other adverse events	Younger Children (0 - 6 Years)	11	12
Total, other adverse events	Older Children (7 - 12 Years)	20	23
Total, other adverse events	Adolescents (13 - 17 Years)	12	14
Total, other adverse events	Adults (18 - 70 Years)	4	6
Total, serious adverse events	Younger Children (0 - 6 Years)	2	12
Total, serious adverse events	Older Children (7 - 12 Years)	4	23

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers2

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as	Source
27174727	RESULT	PubMed
32369845	RESULT	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 comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Younger Children (0-6 Years) vs 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)	Trial comparisons
Younger Children (0-6 Years) vs 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)	Trial comparisons

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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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.

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Read this trial from software.

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