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

Sponsor

Novo Nordisk A/S (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT02141074

Collaborator

(none)

Enrollment

Locations

Arm

99.9

Anticipated Duration (Months)

0.9

Patients Per Site

Patients Per Site Per Month

Study Details

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.

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

Study Design

Study Type:

Interventional

Anticipated 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 :

Jul 2, 2014

Anticipated Primary Completion Date :

Oct 30, 2022

Anticipated Study Completion Date :

Oct 30, 2022

Arms and Interventions

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.

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

Primary Outcome Measures

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

Secondary Outcome Measures

Number and frequency of adverse events [When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months)]
Number and frequency of serious adverse events [When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months)]
Number and frequency of Medical Events of Special Interest [When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months)]
Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate) [When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months)]
Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor") [When minimum 20 PUPs have reached at least 50 EDs (after approx. 48 months)]
Number and frequency of adverse events [When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months)]
Number and frequency of adverse events [At end of trial (after approx. 100 months)]
Number and frequency of serious adverse events [When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months)]
Number and frequency of serious adverse events [At end of trial (after approx. 100 months)]
Number and frequency of Medical Events of Special Interest [When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months)]
Number and frequency of Medical Events of Special Interest [At end of trial (after approx. 100 months)]
Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate) [When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months)]
Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate) [At end of trial (after approx. 100 months)]
Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor") [When minimum 40 PUPs have reached at least 100 EDs (after approx. 82 months)]
Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor") [At end of trial (after approx. 100 months)]

Eligibility Criteria

Criteria

Ages Eligible for Study:

0 Years to 6 Years

Sexes Eligible for Study:

Male

Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Novo Nordisk Investigational Site	Los Angeles	California	United States	90027
2	Novo Nordisk Investigational Site	Augusta	Georgia	United States	30912
3	Novo Nordisk Investigational Site	Macon	Georgia	United States	31201
4	Novo Nordisk Investigational Site	Boise	Idaho	United States	83712
5	Novo Nordisk Investigational Site	New Orleans	Louisiana	United States	70118-5720
6	Novo Nordisk Investigational Site	Baltimore	Maryland	United States	21205
7	Novo Nordisk Investigational Site	Omaha	Nebraska	United States	68198-6828
8	Novo Nordisk Investigational Site	Charlotte	North Carolina	United States	28204
9	Novo Nordisk Investigational Site	Cleveland	Ohio	United States	44106
10	Novo Nordisk Investigational Site	Dayton	Ohio	United States	45404
11	Novo Nordisk Investigational Site	Philadelphia	Pennsylvania	United States	19134
12	Novo Nordisk Investigational Site	Nashville	Tennessee	United States	37232
13	Novo Nordisk Investigational Site	Salt Lake City	Utah	United States	84113
14	Novo Nordisk Investigational Site	Algiers		Algeria	16000
15	Novo Nordisk Investigational Site	Setif		Algeria	19000
16	Novo Nordisk Investigational Site	Caba		Argentina	C1245AAM
17	Novo Nordisk Investigational Site	Cordoba		Argentina	X5000FAL
18	Novo Nordisk Investigational Site	South Brisbane	Queensland	Australia	4101
19	Novo Nordisk Investigational Site	Parkville	Victoria	Australia	3052
20	Novo Nordisk Investigational Site	Graz		Austria	8036
21	Novo Nordisk Investigational Site	Innsbruck		Austria	6020
22	Novo Nordisk Investigational Site	Klagenfurt		Austria	9020
23	Novo Nordisk Investigational Site	Linz		Austria	4020
24	Novo Nordisk Investigational Site	Salzburg		Austria	A 5020
25	Novo Nordisk Investigational Site	St. Poelten		Austria	A 3100
26	Novo Nordisk Investigational Site	Wien		Austria	A 1090
27	Novo Nordisk Investigational Site	Hamilton	Ontario	Canada	L8N 3Z5
28	Novo Nordisk Investigational Site	Toronto	Ontario	Canada	M5G 1X8
29	Novo Nordisk Investigational Site	Clermont Ferrand		France	63003
30	Novo Nordisk Investigational Site	Kremlin-Bicêtre		France	94270
31	Novo Nordisk Investigational Site	Nantes Cedex 1		France	44093
32	Novo Nordisk Investigational Site	Bonn		Germany	53127
33	Novo Nordisk Investigational Site	Duisburg		Germany	47051
34	Novo Nordisk Investigational Site	Hannover		Germany	30159
35	Novo Nordisk Investigational Site	Mörfelden-Walldorf		Germany	64546
36	Novo Nordisk Investigational Site	Tel-Hashomer		Israel	52621
37	Novo Nordisk Investigational Site	Firenze		Italy	50134
38	Novo Nordisk Investigational Site	Aichi		Japan	466-8560
39	Novo Nordisk Investigational Site	Kanagawa		Japan	232-8555
40	Novo Nordisk Investigational Site	Saitama		Japan	330-8777
41	Novo Nordisk Investigational Site	Shizuoka		Japan	420-8660
42	Novo Nordisk Investigational Site	Tokyo		Japan	157-8535
43	Novo Nordisk Investigational Site	Tokyo		Japan	167-0035
44	Novo Nordisk Investigational Site	Georgetown, Penang		Malaysia	10450
45	Novo Nordisk Investigational Site	Klang, Selangor		Malaysia	41200
46	Novo Nordisk Investigational Site	Kuala Lumpur		Malaysia	50400
47	Novo Nordisk Investigational Site	Kuantan		Malaysia	25100
48	Novo Nordisk Investigational Site	Nijmegen		Netherlands	6525 GA
49	Novo Nordisk Investigational Site	Esplugues Llobregat		Spain	08950
50	Novo Nordisk Investigational Site	Madrid		Spain	28046
51	Novo Nordisk Investigational Site	Valencia		Spain	46026
52	Novo Nordisk Investigational Site	Changhua		Taiwan	500
53	Novo Nordisk Investigational Site	Kaohsiung		Taiwan	807
54	Novo Nordisk Investigational Site	Taichung		Taiwan	40447
55	Novo Nordisk Investigational Site	Taipei		Taiwan	100
56	Novo Nordisk Investigational Site	Bangkok		Thailand	10330
57	Novo Nordisk Investigational Site	Bangkok		Thailand	10400
58	Novo Nordisk Investigational Site	Chiang Mai		Thailand	50200
59	Novo Nordisk Investigational Site	Birmingham		United Kingdom	B4 6NH
60	Novo Nordisk Investigational Site	Glasgow		United Kingdom	G51 4TF
61	Novo Nordisk Investigational Site	Leicester		United Kingdom	LE1 5WW
62	Novo Nordisk Investigational Site	London		United Kingdom	SE1 7EH
63	Novo Nordisk Investigational Site	Manchester		United Kingdom	M13 9WL
64	Novo Nordisk Investigational Site	Oxford		United Kingdom	OX3 9DU