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Safety and Efficacy of NNC-0156-0000-0009 After Long-Term Exposure in Patients With Haemophilia B: An Extension to Trials NN7999-3747 and NN7999-3773 (paradigm™ 4)

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ClinicalTrials.gov Identifier: NCT01395810
Recruitment Status : Completed
First Posted : July 18, 2011
Results First Posted : May 9, 2018
Last Update Posted : May 9, 2018
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

Study Description
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Brief Summary:

This trial is conducted in Asia, Europe, Japan, North America and South Africa. The aim is to evaluate the safety and efficacy of nonacog beta pegol (NNC-0156-0000-0009) after long-term exposure in patients with haemophilia B.

This trial is an extension to trials NN7999-3747 (NCT01333111/paradigm™ 2) and NN7999-3773 (NCT01386528/paradigm™ 3).


Condition or disease Intervention/treatment Phase
Congenital Bleeding Disorder Haemophilia B Drug: nonacog beta pegol Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 71 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Safety and Efficacy of NNC-0156-0000-0009 After Long-Term Exposure in Patients With Haemophilia B
Actual Study Start Date : April 15, 2012
Actual Primary Completion Date : March 30, 2014
Actual Study Completion Date : March 30, 2014

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Prophylaxis, high dose (once weekly) Drug: nonacog beta pegol
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 Name: NNC-0156-0000-0009
Experimental: Prophylaxis, low dose (once weekly) Drug: nonacog beta pegol
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 Name: NNC-0156-0000-0009
Experimental: On-demand Drug: nonacog beta pegol
One single dose administered intravenously (into the vein). Patients will treat themselves with either a low or a high dose dependent on the severity of the bleeding episode.
Other Name: NNC-0156-0000-0009
Experimental: Prophylaxis, high dose (every second week) Drug: nonacog beta pegol
One single dose administered intravenously (into the vein) every second week. Patients will receive instruction on how to treat any bleeding episode they may experience.
Other Name: NNC-0156-0000-0009


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of Inhibitory Antibodies Against FIX Defined as Titre Above or Equal to 0.6 BU (Bethesda Units) [ Time Frame: From Day 1 up to 2 years ]

    The primary endpoint was incidence of inhibitors against coagulation factor nine (FIX) defined as titre

    ≥0.6 Bethesda unit (BU). Number of subjects who developed inhibitors against FIX are reported.



Secondary Outcome Measures :
  1. Haemostatic Effect of Nonacog Beta Pegol When Used for Treatment of Bleeding Episodes, Assessed as Success/Failure Based on a Four-point Scale for Haemostatic Response (Excellent, Good, Moderate, Poor) [ Time Frame: From Day 1 up to 2 years ]
    The haemostatic effect was evaluated by a four-point scale where an "excellent" or "good" outcome translated into a successful treatment, and a "moderate" or "poor" outcome was considered a treatment failure. The values mentioned below do not include bleeds with missing response.

  2. Number of Bleeding Episodes During Routine Prophylaxis [ Time Frame: From Day 1 up to 2 years ]
    Annualized bleeding rate is the total number of bleeding episodes/total exposure time. It is analysed by a Poisson regression model with dose as a factor allowing for over-dispersion and using treatment duration as an offset. Median annualized bleeding rate is the median of individual annualized bleeding rates. Numbers are based on the treatment arm at the time of each bleed.

  3. FIX Trough Levels [ Time Frame: From Day 1 up to 2 years ]
    During the trial, the pre-dose FIX levels was measured with the one-stage clotting assay. Measurements taken at least 5 days and no more than 10 days after last dose as well as at least 14 days after last bleeding episode were included in this analysis. The mean FIX trough levels were estimated based on the mixed effects model on the log-transformed plasma concentration with subject as a random effect. The mean FIX trough level was presented back-transformed to the natural scale.

  4. Incidence of Adverse Events (AEs) [ Time Frame: From Day 1 up to 2 years ]
    AEs were summarized by frequency of events and frequency of patients with any event. Incidence of AEs was expressed as number of AEs per subject years of exposure (total number of events /total time in trial).

  5. Incidence of Serious Adverse Events (SAEs) [ Time Frame: From Day 1 up to 2 years ]
    AEs were summarized by frequency of events and frequency of patients with any event. Incidence of serious AEs was expressed as number of serious AEs per subject years of exposure (total number of events /total time in trial).


Eligibility Criteria
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Ages Eligible for Study:   13 Years to 70 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Previous participation in NN7999-3747 (NCT01333111) and/or NN7999-3773

Exclusion Criteria:

  • Known history of FIX inhibitors based on existing medical records, laboratory report reviews and patient and LAR (legal acceptable representative) interviews
  • Current FIX inhibitors above or equal to 0.6 BU (Bethesda Units)
  • 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)
  • Any disease (liver, kidney, inflammatory and mental disorders included) or condition which, according to the Investigator's (trial physician) judgement, could imply a potential hazard to the patient, interfere with trial participation, or interfere with trial outcome
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01395810


Locations
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Sponsors and Collaborators
Novo Nordisk A/S
Investigators
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Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
More Information
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Additional Information:

Publications of Results:

Other Publications:
Young G, Collins P, Tehranchi R, Chuansumrit A, Hanabusa H, Lentz SR, Mahlangu J, Mauser-Bunschoten E, Negrier C, Oldenburg J, Patiroglu T, Santagostino E, Zak M, Abdul Karim F. Safety and efficacy of nonacog beta pegol (N9-GP) for prophylaxis and treatment of bleeding episodes in previously-treated patients with hemophilia B: results from an extension trial. American Society of Hematology - 56th Annual Meeting (ASH) in San Francisco, CA, US

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Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT01395810    
Other Study ID Numbers: NN7999-3775
2010-023072-17 ( EudraCT Number )
U1111-1121-5408 ( Other Identifier: WHO )
JapicCTI-121812 ( Registry Identifier: JAPIC )
First Posted: July 18, 2011    Key Record Dates
Results First Posted: May 9, 2018
Last Update Posted: May 9, 2018
Last Verified: April 2018
Additional relevant MeSH terms:
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Hemostatic Disorders
Hemophilia A
Hemophilia B
Blood Coagulation Disorders
Blood Coagulation Disorders, Inherited
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Vascular Diseases
Cardiovascular Diseases