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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02141074




Registration number
NCT02141074
Ethics application status
Date submitted
28/03/2014
Date registered
19/05/2014
Date last updated
19/03/2020

Titles & IDs
Public title
Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B
Scientific 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)
Secondary ID [1] 0 0
2012-004867-38
Secondary ID [2] 0 0
NN7999-3895
Universal Trial Number (UTN)
Trial acronym
paradigmâ„¢6
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Congenital Bleeding Disorder 0 0
Haemophilia B 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - nonacog beta pegol

Experimental: 50 EDs (exposure days) -


Treatment: Drugs: 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.

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of inhibitory antibodies against coagulation factor IX (FIX)
Timepoint [1] 0 0
When minimum 20 previously untreated patients (PUPs) have reached at least 50 exposure days (EDs) (after approx. 48 months)
Primary outcome [2] 0 0
Incidence of inhibitory antibodies against coagulation factor IX (FIX)
Timepoint [2] 0 0
When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months)
Primary outcome [3] 0 0
Incidence of inhibitory antibodies against coagulation factor IX (FIX)
Timepoint [3] 0 0
At end of trial (after approx. 100 months)
Secondary outcome [1] 0 0
Number and frequency of adverse events
Timepoint [1] 0 0
When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months)
Secondary outcome [2] 0 0
Number and frequency of serious adverse events
Timepoint [2] 0 0
When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months)
Secondary outcome [3] 0 0
Number and frequency of Medical Events of Special Interest
Timepoint [3] 0 0
When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months)
Secondary outcome [4] 0 0
Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate)
Timepoint [4] 0 0
When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months)
Secondary outcome [5] 0 0
Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor")
Timepoint [5] 0 0
When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months)
Secondary outcome [6] 0 0
Number and frequency of adverse events
Timepoint [6] 0 0
When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months)
Secondary outcome [7] 0 0
Number and frequency of adverse events
Timepoint [7] 0 0
At end of trial (after approx. 100 months)
Secondary outcome [8] 0 0
Number and frequency of serious adverse events
Timepoint [8] 0 0
When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months)
Secondary outcome [9] 0 0
Number and frequency of serious adverse events
Timepoint [9] 0 0
At end of trial (after approx. 100 months)
Secondary outcome [10] 0 0
Number and frequency of Medical Events of Special Interest
Timepoint [10] 0 0
When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months)
Secondary outcome [11] 0 0
Number and frequency of Medical Events of Special Interest
Timepoint [11] 0 0
At end of trial (after approx. 100 months)
Secondary outcome [12] 0 0
Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate)
Timepoint [12] 0 0
When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months)
Secondary outcome [13] 0 0
Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate)
Timepoint [13] 0 0
At end of trial (after approx. 100 months)
Secondary outcome [14] 0 0
Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor")
Timepoint [14] 0 0
When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months)
Secondary outcome [15] 0 0
Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor")
Timepoint [15] 0 0
At end of trial (after approx. 100 months)

Eligibility
Key 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)
Minimum age
No limit
Maximum age
6 Years
Gender
Males
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
Novo Nordisk Investigational Site - South Brisbane
Recruitment hospital [2] 0 0
Novo Nordisk Investigational Site - Parkville
Recruitment postcode(s) [1] 0 0
4101 - South Brisbane
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Georgia
Country [3] 0 0
United States of America
State/province [3] 0 0
Idaho
Country [4] 0 0
United States of America
State/province [4] 0 0
Louisiana
Country [5] 0 0
United States of America
State/province [5] 0 0
Maryland
Country [6] 0 0
United States of America
State/province [6] 0 0
Nebraska
Country [7] 0 0
United States of America
State/province [7] 0 0
North Carolina
Country [8] 0 0
United States of America
State/province [8] 0 0
Ohio
Country [9] 0 0
United States of America
State/province [9] 0 0
Pennsylvania
Country [10] 0 0
United States of America
State/province [10] 0 0
Tennessee
Country [11] 0 0
United States of America
State/province [11] 0 0
Utah
Country [12] 0 0
Algeria
State/province [12] 0 0
Algiers
Country [13] 0 0
Algeria
State/province [13] 0 0
Setif
Country [14] 0 0
Argentina
State/province [14] 0 0
Caba
Country [15] 0 0
Argentina
State/province [15] 0 0
Cordoba
Country [16] 0 0
Austria
State/province [16] 0 0
Graz
Country [17] 0 0
Austria
State/province [17] 0 0
Innsbruck
Country [18] 0 0
Austria
State/province [18] 0 0
Klagenfurt
Country [19] 0 0
Austria
State/province [19] 0 0
Linz
Country [20] 0 0
Austria
State/province [20] 0 0
Salzburg
Country [21] 0 0
Austria
State/province [21] 0 0
St. Poelten
Country [22] 0 0
Austria
State/province [22] 0 0
Wien
Country [23] 0 0
Canada
State/province [23] 0 0
Ontario
Country [24] 0 0
France
State/province [24] 0 0
Clermont Ferrand
Country [25] 0 0
France
State/province [25] 0 0
Kremlin-Bicêtre
Country [26] 0 0
France
State/province [26] 0 0
Nantes Cedex 1
Country [27] 0 0
Germany
State/province [27] 0 0
Bonn
Country [28] 0 0
Germany
State/province [28] 0 0
Duisburg
Country [29] 0 0
Germany
State/province [29] 0 0
Hannover
Country [30] 0 0
Germany
State/province [30] 0 0
Mörfelden-Walldorf
Country [31] 0 0
Israel
State/province [31] 0 0
Tel-Hashomer
Country [32] 0 0
Italy
State/province [32] 0 0
Firenze
Country [33] 0 0
Japan
State/province [33] 0 0
Aichi
Country [34] 0 0
Japan
State/province [34] 0 0
Kanagawa
Country [35] 0 0
Japan
State/province [35] 0 0
Saitama
Country [36] 0 0
Japan
State/province [36] 0 0
Shizuoka
Country [37] 0 0
Japan
State/province [37] 0 0
Tokyo
Country [38] 0 0
Malaysia
State/province [38] 0 0
Georgetown, Penang
Country [39] 0 0
Malaysia
State/province [39] 0 0
Klang, Selangor
Country [40] 0 0
Malaysia
State/province [40] 0 0
Kuala Lumpur
Country [41] 0 0
Malaysia
State/province [41] 0 0
Kuantan
Country [42] 0 0
Netherlands
State/province [42] 0 0
Nijmegen
Country [43] 0 0
Spain
State/province [43] 0 0
Esplugues Llobregat
Country [44] 0 0
Spain
State/province [44] 0 0
Madrid
Country [45] 0 0
Spain
State/province [45] 0 0
Valencia
Country [46] 0 0
Taiwan
State/province [46] 0 0
Changhua
Country [47] 0 0
Taiwan
State/province [47] 0 0
Kaohsiung
Country [48] 0 0
Taiwan
State/province [48] 0 0
Taichung
Country [49] 0 0
Taiwan
State/province [49] 0 0
Taipei
Country [50] 0 0
Thailand
State/province [50] 0 0
Bangkok
Country [51] 0 0
Thailand
State/province [51] 0 0
Chiang Mai
Country [52] 0 0
United Kingdom
State/province [52] 0 0
Birmingham
Country [53] 0 0
United Kingdom
State/province [53] 0 0
Glasgow
Country [54] 0 0
United Kingdom
State/province [54] 0 0
Leicester
Country [55] 0 0
United Kingdom
State/province [55] 0 0
London
Country [56] 0 0
United Kingdom
State/province [56] 0 0
Manchester
Country [57] 0 0
United Kingdom
State/province [57] 0 0
Oxford

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Novo Nordisk A/S
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT02141074
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Global Clinical Registry (GCR, 1452)
Address 0 0
Novo Nordisk A/S
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Novo Nordisk
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
clinicaltrials@novonordisk.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT02141074