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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

Tracking Information
First Submitted Date  ICMJE June 30, 2011
First Posted Date  ICMJE July 18, 2011
Results First Submitted Date  ICMJE November 28, 2017
Results First Posted Date  ICMJE May 9, 2018
Last Update Posted Date May 9, 2018
Actual Study Start Date  ICMJE April 15, 2012
Actual Primary Completion Date March 30, 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 9, 2018)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: July 15, 2011)
Incidence of Inhibitory Antibodies Against FIX Defined as Titre Above or Equal to 0.6 BU (Bethesda Units) [ Time Frame: Every 3rd month the first year of trial, then every 6 months until trial completion - up to 4 years ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: April 9, 2018)
  • 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.
  • 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.
  • 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.
  • 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).
  • 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).
Original Secondary Outcome Measures  ICMJE
 (submitted: July 15, 2011)
  • 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 (excellent, good moderate, poor) [ Time Frame: Every 3rd month the first year of trial, then every 6 months until trial completion - up to 4 years ]
  • Number of Bleeding Episodes During Routine Prophylaxis [ Time Frame: Every 3rd month the first year of trial, then every 6 months until trial completion - up to 4 years ]
  • FIX Trough Levels [ Time Frame: Every 3rd month the first year of trial, then every 6 months until trial completion - up to 4 years ]
  • Incidence of Adverse Events (AEs) [ Time Frame: Every 3rd month the first year of trial, then every 6 months until trial completion - up to 4 years ]
  • Incidence of Serious Adverse Events (SAEs) [ Time Frame: Every 3rd month the first year of trial, then every 6 months until trial completion - up to 4 years ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE 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
Official Title  ICMJE Safety and Efficacy of NNC-0156-0000-0009 After Long-Term Exposure in Patients With Haemophilia B
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).

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Congenital Bleeding Disorder
  • Haemophilia B
Intervention  ICMJE
  • 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
  • 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
  • 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
Study Arms  ICMJE
  • Experimental: Prophylaxis, high dose (once weekly)
    Intervention: Drug: nonacog beta pegol
  • Experimental: Prophylaxis, low dose (once weekly)
    Intervention: Drug: nonacog beta pegol
  • Experimental: On-demand
    Intervention: Drug: nonacog beta pegol
  • Experimental: Prophylaxis, high dose (every second week)
    Intervention: Drug: nonacog beta pegol
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: October 17, 2013)
71
Original Estimated Enrollment  ICMJE
 (submitted: July 15, 2011)
70
Actual Study Completion Date  ICMJE March 30, 2014
Actual Primary Completion Date March 30, 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 13 Years to 70 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Austria,   France,   Germany,   Greece,   Italy,   Japan,   Macedonia, The Former Yugoslav Republic of,   Malaysia,   Netherlands,   Romania,   Russian Federation,   South Africa,   Spain,   Taiwan,   Thailand,   Turkey,   United Kingdom,   United States
Removed Location Countries Bulgaria,   Canada,   Hungary,   Puerto Rico
 
Administrative Information
NCT Number  ICMJE NCT01395810
Other Study ID Numbers  ICMJE NN7999-3775
2010-023072-17 ( EudraCT Number )
U1111-1121-5408 ( Other Identifier: WHO )
JapicCTI-121812 ( Registry Identifier: JAPIC )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Novo Nordisk A/S
Study Sponsor  ICMJE Novo Nordisk A/S
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
PRS Account Novo Nordisk A/S
Verification Date April 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP