This trial is conducted in Africa, Asia, Europe, Japan and North America. The aim of this trial is to evaluate the safety and efficacy, including pharmacokinetics (the rate at which the body eliminates the trial drug), of NNC-0156-0000-0009 (nonacog beta pegol) when used for treatment and prophylaxis of bleeding episodes in patients with haemophilia B.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
74
One single dose administered intravenously (into the vein) once weekly. Patients will receive instruction on how to treat any bleeding episode they may experience
One single dose administered intravenously (into the vein) once weekly. Patients will receive instruction on how to treat any bleeding episode they may experience
Patients will treat themselves with either a low or a high dose dependent on the severity of the bleeding episode
Incidence of Inhibitory Antibodies Against Factor IX Defined as Titre Equal to or Above 0.6 BU (Bethesda Units)
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.
Time frame: 52 weeks after treatment start for patients on prophylaxis
Incidence of Inhibitory Antibodies Against Factor IX Defined as Titre Equal to or Above 0.6 BU (Bethesda Units)
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.
Time frame: 28 weeks after treatment start on on-demand treatment
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
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.
Time frame: 52 weeks after treatment start for patients on prophylaxis
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
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Novo Nordisk Investigational Site
Los Angeles, California, United States
Novo Nordisk Investigational Site
Jacksonville, Florida, United States
Novo Nordisk Investigational Site
Augusta, Georgia, United States
Novo Nordisk Investigational Site
Iowa City, Iowa, United States
Novo Nordisk Investigational Site
Baltimore, Maryland, United States
Novo Nordisk Investigational Site
Minneapolis, Minnesota, United States
Novo Nordisk Investigational Site
Omaha, Nebraska, United States
Novo Nordisk Investigational Site
Newark, New Jersey, United States
Novo Nordisk Investigational Site
New York, New York, United States
Novo Nordisk Investigational Site
Syracuse, New York, United States
...and 33 more locations
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.
Time frame: 28 weeks after treatment start on on-demand treatment
Number of Bleeding Episodes Per Patient During Routine 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).
Time frame: 52 weeks after treatment start for patients on prophylaxis
Factor IX Trough Levels
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.
Time frame: 52 weeks after treatment start for patients on prophylaxis
Incidence of Adverse Events (AEs)
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).
Time frame: at 56 weeks ±2 weeks for patients on prophylaxis
Incidence of Adverse Events (AEs)
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).
Time frame: at 32 weeks ±2 weeks for patients on on-demand treatment
Incidence of Serious Adverse Events (SAEs)
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).
Time frame: at 56 weeks ±2 weeks for patients on prophylaxis
Incidence of Serious Adverse Events (SAEs)
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).
Time frame: at 32 weeks ±2 weeks for patients on on-demand treatment
Host Cell Proteins (HCP) Antibodies
Subjects who were positive for anti-Host Cell Protein (HCP) antibodies.
Time frame: 52 weeks after treatment start for patients on prophylaxis
Host Cell Proteins (HCP) Antibodies
Subjects who were positive for anti-HCP antibodies.
Time frame: 28 weeks after treatment start on on-demand treatment