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Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B (paradigm™6)

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ClinicalTrials.gov Identifier: NCT02141074
Recruitment Status : Completed
First Posted : May 19, 2014
Last Update Posted : March 20, 2023
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

Study Description
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Brief Summary:
This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of nonacog beta pegol (N9-GP) in previously untreated patients with Haemophilia B.

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)
Estimated Enrollment : 60 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label Single-arm Multicentre Non-controlled Phase 3 a Trial Investigating Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Patients With Haemophilia B (FIX Activity Below or Equal to 2 Percent)
Actual Study Start Date : July 2, 2014
Actual Primary Completion Date : October 27, 2022
Actual Study Completion Date : October 27, 2022

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Arms and Interventions
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Arm Intervention/treatment
Experimental: 50 EDs (exposure days) Drug: nonacog beta pegol
For intravenous (i.v.) injection. A single dose of 40 U/kg, unless the bleeding episode is severe in which case it should be treated with 80 U/kg.


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of inhibitory antibodies against coagulation factor IX (FIX) [ Time Frame: When minimum 20 previously untreated patients (PUPs) have reached at least 50 exposure days (EDs) (after approx. 48 months) ]
  2. Incidence of inhibitory antibodies against coagulation factor IX (FIX) [ Time Frame: When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months) ]
  3. Incidence of inhibitory antibodies against coagulation factor IX (FIX) [ Time Frame: At end of trial (after approx. 100 months) ]

Secondary Outcome Measures :
  1. Number and frequency of adverse events [ Time Frame: When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months) ]
  2. Number and frequency of serious adverse events [ Time Frame: When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months) ]
  3. Number and frequency of Medical Events of Special Interest [ Time Frame: When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months) ]
  4. Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate) [ Time Frame: When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months) ]
  5. Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor") [ Time Frame: When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months) ]
  6. Number and frequency of adverse events [ Time Frame: When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months) ]
  7. Number and frequency of adverse events [ Time Frame: At end of trial (after approx. 100 months) ]
  8. Number and frequency of serious adverse events [ Time Frame: When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months) ]
  9. Number and frequency of serious adverse events [ Time Frame: At end of trial (after approx. 100 months) ]
  10. Number and frequency of Medical Events of Special Interest [ Time Frame: When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months) ]
  11. Number and frequency of Medical Events of Special Interest [ Time Frame: At end of trial (after approx. 100 months) ]
  12. Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate) [ Time Frame: When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months) ]
  13. Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate) [ Time Frame: At end of trial (after approx. 100 months) ]
  14. Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor") [ Time Frame: When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months) ]
  15. Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor") [ Time Frame: At end of trial (after approx. 100 months) ]

Eligibility Criteria
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Ages Eligible for Study:   0 Years to 6 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial
  • Male, age below 6 years at the time of signing informed consent
  • Patients with the diagnosis of haemophilia B (FIX (coagulation factor IX) activity level below or equal to 2%) based on medical records or central laboratory results
  • Previously untreated or exposed to FIX containing products less than or equal to 3 exposure days (5 previous exposures to blood components is acceptable)

Exclusion Criteria:

  • Any history of FIX inhibitors (defined by medical records)
  • Known or suspected hypersensitivity to trial product or related products
  • Previous participation in this trial. Participation is defined as first dose administered of trial product
  • Receipt of any investigational medicinal product within 30 days before screening
  • Congenital or acquired coagulation disorder other than haemophilia B
  • Any chronic disorder or severe disease which, in the opinion of the Investigator, might jeopardise the patient's safety or compliance with the protocol
  • Patient's parent(s)/LAR(s) (legally acceptable representative) mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation
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): NCT02141074


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:

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Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT02141074    
Other Study ID Numbers: NN7999-3895
2012-004867-38 ( EudraCT Number )
U1111-1135-9557 ( Other Identifier: WHO )
JapicCTI-142611 ( Registry Identifier: JAPIC )
NL53683.091.15 ( Other Identifier: CCMO )
First Posted: May 19, 2014    Key Record Dates
Last Update Posted: March 20, 2023
Last Verified: March 2023
Additional relevant MeSH terms:
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Hemostatic Disorders
Hemophilia A
Hemophilia B
Blood Coagulation Disorders
Hemorrhage
Pathologic Processes
Blood Coagulation Disorders, Inherited
 Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Vascular Diseases
Cardiovascular Diseases