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Completed

NCT Number: NCT01333111

Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients

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.

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Key information

Conditions

Congenital Bleeding Disorder Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Haemophilia B Hematologic Diseases Hemic and Lymphatic Diseases Hemophilia B Hemorrhagic Disorders

Age range

13 year–70 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

Novo Nordisk Investigational Site, Edmonton, Alberta, Canada

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Male patients with moderately severe or severe congenital haemophilia B with a factor IX activity of 2% or below according to medical records
  • History of at least 150 exposure days to other factor IX products
  • Patients currently treated on-demand with at least 6 bleeding episodes during the last 12 months or at least 3 bleeding episodes during the last 6 months, or patients currently on prophylaxis

Exclusion criteria

  • Known history of factor IX inhibitors based on existing medical records, laboratory report reviews and patient and legally acceptable representative (LAR) interviews
  • HIV (Human immunodeficiency virus) positive, with a viral load equal to or above 400,000 copies/mL and/or CD4+ lymphocyte count equal to or below 200/microL
  • Congenital or acquired coagulation disorders other than haemophilia B
  • Previous arterial thrombotic events (e.g. myocardial infarction and intracranial thrombosis) or previous deep venous thrombosis or pulmonary embolism (as defined by available medical records)
  • Immune modulating or chemotherapeutic medication

Treatment and study plan

Nonacog beta pegol

Drug

One single dose administered intravenously (into the vein) once weekly. Patients will receive instruction on how to treat any bleeding episode they may experience

Other names: NNC-0156-0000-0009

Primary outcomes

  1. Incidence of Inhibitory Antibodies Against Factor IX Defined as Titre Equal to or Above 0.6 BU (Bethesda Units)

    Time frame: 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.

  2. Incidence of Inhibitory Antibodies Against Factor IX Defined as Titre Equal to or Above 0.6 BU (Bethesda Units)

    Time frame: 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 outcomes

  1. 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

    Time frame: 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.

  2. 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

    Time frame: 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.

  3. Number of Bleeding Episodes Per Patient During Routine Prophylaxis

    Time frame: 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).

  4. Factor IX Trough Levels

    Time frame: 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.

  5. Incidence of Adverse Events (AEs)

    Time frame: 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).

  6. Incidence of Adverse Events (AEs)

    Time frame: 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).

  7. Incidence of Serious Adverse Events (SAEs)

    Time frame: 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).

  8. Incidence of Serious Adverse Events (SAEs)

    Time frame: 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).

  9. Host Cell Proteins (HCP) Antibodies

    Time frame: 52 weeks after treatment start for patients on prophylaxis

    Subjects who were positive for anti-Host Cell Protein (HCP) antibodies.

  10. Host Cell Proteins (HCP) Antibodies

    Time frame: 28 weeks after treatment start on on-demand treatment

    Subjects who were positive for anti-HCP antibodies.

Sponsors and collaborators

Lead sponsor

Novo Nordisk A/S

Industry

Registry information

Official study title

A Multi-centre, Single-blind Trial Evaluating Safety and Efficacy, Including Pharmacokinetics, of NNC-0156-0000-0009 When Used for Treatment and Prophylaxis of Bleeding Episodes in Patients With Haemophilia B

Acronym: paradigm™ 2

Important dates

Study start
2011
Primary completion
2013
Study completion
2013
First posted
Apr 11, 2011
Registry last updated
Jul 28, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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