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

Primary Purpose

Congenital Bleeding Disorder, Haemophilia B

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
nonacog beta pegol
Sponsored by
Novo Nordisk A/S
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Congenital Bleeding Disorder

Eligibility Criteria

0 Years - 6 Years (Child)MaleDoes not accept healthy volunteers

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

Sites / Locations

  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
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  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site
  • Novo Nordisk Investigational Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

50 EDs (exposure days)

Arm Description

Outcomes

Primary Outcome Measures

Incidence of inhibitory antibodies against coagulation factor IX (FIX)
Incidence of inhibitory antibodies against coagulation factor IX (FIX)
Incidence of inhibitory antibodies against coagulation factor IX (FIX)

Secondary Outcome Measures

Number and frequency of adverse events
Number and frequency of serious adverse events
Number and frequency of Medical Events of Special Interest
Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate)
Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor")
Number and frequency of adverse events
Number and frequency of adverse events
Number and frequency of serious adverse events
Number and frequency of serious adverse events
Number and frequency of Medical Events of Special Interest
Number and frequency of Medical Events of Special Interest
Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate)
Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate)
Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor")
Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor")

Full Information

First Posted
March 28, 2014
Last Updated
June 26, 2023
Sponsor
Novo Nordisk A/S
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1. Study Identification

Unique Protocol Identification Number
NCT02141074
Brief Title
Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B
Acronym
paradigm™6
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)
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Completed
Study Start Date
July 2, 2014 (Actual)
Primary Completion Date
October 27, 2022 (Actual)
Study Completion Date
October 27, 2022 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novo Nordisk A/S

4. Oversight

Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Congenital Bleeding Disorder, Haemophilia B

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
60 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
50 EDs (exposure days)
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
nonacog beta pegol
Intervention Description
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.
Primary Outcome Measure Information:
Title
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)
Title
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)
Title
Incidence of inhibitory antibodies against coagulation factor IX (FIX)
Time Frame
At end of trial (after approx. 100 months)
Secondary Outcome Measure Information:
Title
Number and frequency of adverse events
Time Frame
When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months)
Title
Number and frequency of serious adverse events
Time Frame
When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months)
Title
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)
Title
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)
Title
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)
Title
Number and frequency of adverse events
Time Frame
When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months)
Title
Number and frequency of adverse events
Time Frame
At end of trial (after approx. 100 months)
Title
Number and frequency of serious adverse events
Time Frame
When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months)
Title
Number and frequency of serious adverse events
Time Frame
At end of trial (after approx. 100 months)
Title
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)
Title
Number and frequency of Medical Events of Special Interest
Time Frame
At end of trial (after approx. 100 months)
Title
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)
Title
Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate)
Time Frame
At end of trial (after approx. 100 months)
Title
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)
Title
Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor")
Time Frame
At end of trial (after approx. 100 months)

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
0 Years
Maximum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Global Clinical Registry (GCR, 1452)
Organizational Affiliation
Novo Nordisk A/S
Official's Role
Study Director
Facility Information:
Facility Name
Novo Nordisk Investigational Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30912
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Macon
State/Province
Georgia
ZIP/Postal Code
31201
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Boise
State/Province
Idaho
ZIP/Postal Code
83712
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70118-5720
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68198-6828
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28204
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44106
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Dayton
State/Province
Ohio
ZIP/Postal Code
45404
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19134
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84113
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Algiers
ZIP/Postal Code
16000
Country
Algeria
Facility Name
Novo Nordisk Investigational Site
City
Setif
ZIP/Postal Code
19000
Country
Algeria
Facility Name
Novo Nordisk Investigational Site
City
Caba
ZIP/Postal Code
C1245AAM
Country
Argentina
Facility Name
Novo Nordisk Investigational Site
City
Cordoba
ZIP/Postal Code
X5000FAL
Country
Argentina
Facility Name
Novo Nordisk Investigational Site
City
South Brisbane
State/Province
Queensland
ZIP/Postal Code
4101
Country
Australia
Facility Name
Novo Nordisk Investigational Site
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3052
Country
Australia
Facility Name
Novo Nordisk Investigational Site
City
Graz
ZIP/Postal Code
8036
Country
Austria
Facility Name
Novo Nordisk Investigational Site
City
Innsbruck
ZIP/Postal Code
6020
Country
Austria
Facility Name
Novo Nordisk Investigational Site
City
Klagenfurt
ZIP/Postal Code
9020
Country
Austria
Facility Name
Novo Nordisk Investigational Site
City
Linz
ZIP/Postal Code
4020
Country
Austria
Facility Name
Novo Nordisk Investigational Site
City
Salzburg
ZIP/Postal Code
A 5020
Country
Austria
Facility Name
Novo Nordisk Investigational Site
City
St. Poelten
ZIP/Postal Code
A 3100
Country
Austria
Facility Name
Novo Nordisk Investigational Site
City
Wien
ZIP/Postal Code
A 1090
Country
Austria
Facility Name
Novo Nordisk Investigational Site
City
Hamilton
State/Province
Ontario
ZIP/Postal Code
L8N 3Z5
Country
Canada
Facility Name
Novo Nordisk Investigational Site
City
Clermont Ferrand
ZIP/Postal Code
63003
Country
France
Facility Name
Novo Nordisk Investigational Site
City
Kremlin-Bicêtre
ZIP/Postal Code
94270
Country
France
Facility Name
Novo Nordisk Investigational Site
City
Nantes Cedex 1
ZIP/Postal Code
44093
Country
France
Facility Name
Novo Nordisk Investigational Site
City
Tel-Hashomer
ZIP/Postal Code
52621
Country
Israel
Facility Name
Novo Nordisk Investigational Site
City
Firenze
ZIP/Postal Code
50134
Country
Italy
Facility Name
Novo Nordisk Investigational Site
City
Saitama
ZIP/Postal Code
330-8777
Country
Japan
Facility Name
Novo Nordisk Investigational Site
City
Georgetown, Penang
ZIP/Postal Code
10450
Country
Malaysia
Facility Name
Novo Nordisk Investigational Site
City
Klang, Selangor
ZIP/Postal Code
41200
Country
Malaysia
Facility Name
Novo Nordisk Investigational Site
City
Kuala Lumpur
ZIP/Postal Code
50400
Country
Malaysia
Facility Name
Novo Nordisk Investigational Site
City
Kuantan
ZIP/Postal Code
25100
Country
Malaysia
Facility Name
Novo Nordisk Investigational Site
City
Esplugues Llobregat
ZIP/Postal Code
08950
Country
Spain
Facility Name
Novo Nordisk Investigational Site
City
Madrid
ZIP/Postal Code
28046
Country
Spain
Facility Name
Novo Nordisk Investigational Site
City
Valencia
ZIP/Postal Code
46026
Country
Spain
Facility Name
Novo Nordisk Investigational Site
City
Changhua
ZIP/Postal Code
500
Country
Taiwan
Facility Name
Novo Nordisk Investigational Site
City
Kaohsiung
ZIP/Postal Code
807
Country
Taiwan
Facility Name
Novo Nordisk Investigational Site
City
Taichung
ZIP/Postal Code
40447
Country
Taiwan
Facility Name
Novo Nordisk Investigational Site
City
Taipei
ZIP/Postal Code
100
Country
Taiwan
Facility Name
Novo Nordisk Investigational Site
City
Bangkok
ZIP/Postal Code
10400
Country
Thailand
Facility Name
Novo Nordisk Investigational Site
City
Chiang Mai
ZIP/Postal Code
50200
Country
Thailand
Facility Name
Novo Nordisk Investigational Site
City
Birmingham
ZIP/Postal Code
B4 6NH
Country
United Kingdom
Facility Name
Novo Nordisk Investigational Site
City
Leicester
ZIP/Postal Code
LE1 5WW
Country
United Kingdom
Facility Name
Novo Nordisk Investigational Site
City
London
ZIP/Postal Code
SE1 7EH
Country
United Kingdom
Facility Name
Novo Nordisk Investigational Site
City
Oxford
ZIP/Postal Code
OX3 9DU
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
33134776
Citation
Chan AK, Alamelu J, Barnes C, Chuansumrit A, Garly ML, Meldgaard RM, Young G. Nonacog beta pegol (N9-GP) in hemophilia B: First report on safety and efficacy in previously untreated and minimally treated patients. Res Pract Thromb Haemost. 2020 Jul 29;4(7):1101-1113. doi: 10.1002/rth2.12412. eCollection 2020 Oct.
Results Reference
background
Links:
URL
http://novonordisk-trials.com
Description
Clinical Trials at Novo Nordisk

Learn more about this trial

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

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