pathfinder™6: Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A

Sponsor

Novo Nordisk A/S (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT02137850

Collaborator

(none)

125

Enrollment

Locations

Arm

107.7

Anticipated Duration (Months)

1.8

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 turoctocog alfa pegol (N8-GP) in previously untreated patients (PUPs) with haemophilia A.

Condition or Disease	Intervention/Treatment	Phase
Congenital Bleeding Disorder Haemophilia A	Drug: turoctocog alfa pegol	Phase 3

Study Design

Study Type:

Interventional

Anticipated Enrollment :

125 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 3a Trial Investigating Safety and Efficacy of N8-GP in Prophylaxis and Treatment of Bleeding Episodes in Previously Untreated Paediatric Patients With Severe Haemophilia A

Actual Study Start Date :

Jun 26, 2014

Anticipated Primary Completion Date :

Jun 17, 2023

Anticipated Study Completion Date :

Jun 17, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: 50 EDs (exposure days)	Drug: turoctocog alfa pegol For intravenous (i.v.) injection. Frequency and dosage (20-75 U/kg) dependent on whether given as treatment for bleeding episode or as prophylaxis Other Names: NNC 0129-0000-1003 N8-GP

Arm

Intervention/Treatment

Experimental: 50 EDs (exposure days)

Drug: turoctocog alfa pegol

For intravenous (i.v.) injection. Frequency and dosage (20-75 U/kg) dependent on whether given as treatment for bleeding episode or as prophylaxis

Other Names:

NNC 0129-0000-1003

N8-GP

Outcome Measures

Primary Outcome Measures

Incidence of inhibitory antibodies against coagulation factor VIII (FVIII) [When the first 50 PUP have reached at least 50 exposure dates. (Expected to reach between 6 - 18 months)]
Incidence of inhibitory antibodies against coagulation factor VIII (FVIII) [At the end of the trial. End of trial will be up to 4 years after the patient has reached 100 exposure dates. (Expected to reach between 12 - 60 months)]

Secondary Outcome Measures

Frequency of adverse events including serious adverse events and medical events of special interest [When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days]
Incidence of confirmed high titre inhibitors (defined as inhibitor titre above 5 Bethesda Units (BU) [When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days]
Number of breakthrough bleeding episodes during prophylaxis with turoctocog alfa pegol (N8-GP) (annualised bleeding rate) [When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days]
Haemostatic effect of N8-GP in treatment of bleeding episodes, assessed by a predefined 4-point haemostatic response scale ("excellent", "good", "moderate" and "none") [When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days]
Consumption of N8-GP for prophylaxis (number of injections and U/Kg per month and per year) [When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days]
Consumption of N8-GP for treatment of bleeding episodes (number of injections and U/Kg required per bleeding episode) [When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days]
Total consumption of N8-GP per patient (prevention and treatment of bleeding episodes) per month and annualised value [When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days]
Outcome of immune tolerance induction (ITI), assessed by a predefined 4-point ITI outcome scale ("success", "partial success", "failure", "other") [When the first 50 PUPs have reached at least 50 exposure days, and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days]

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A 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 of age at the time of signing informed consent
Diagnosis of severe haemophilia A (FVIII activity level 1%) based on medical records or central laboratory results
No prior use of purified clotting factor products (5 previous exposures to blood components is acceptable)

Exclusion Criteria:

Any history of FVIII inhibitor (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 A
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')/legally acceptable representative (LAR(s')) 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	Phoenix	Arizona	United States	85016-7710
2	Novo Nordisk Investigational Site	Long Beach	California	United States	90806
3	Novo Nordisk Investigational Site	Los Angeles	California	United States	90027
4	Novo Nordisk Investigational Site	Torrance	California	United States	90502-2004
5	Novo Nordisk Investigational Site	Tampa	Florida	United States	33607
6	Novo Nordisk Investigational Site	Boise	Idaho	United States	83712
7	Novo Nordisk Investigational Site	Chicago	Illinois	United States	60612
8	Novo Nordisk Investigational Site	Iowa City	Iowa	United States	52242
9	Novo Nordisk Investigational Site	New Orleans	Louisiana	United States	70112
10	Novo Nordisk Investigational Site	New Orleans	Louisiana	United States	70118-5720
11	Novo Nordisk Investigational Site	Omaha	Nebraska	United States	68198-6828
12	Novo Nordisk Investigational Site	Las Vegas	Nevada	United States	89113
13	Novo Nordisk Investigational Site	New Hyde Park	New York	United States	11042
14	Novo Nordisk Investigational Site	Charlotte	North Carolina	United States	28203
15	Novo Nordisk Investigational Site	Charlotte	North Carolina	United States	28204
16	Novo Nordisk Investigational Site	Dayton	Ohio	United States	45404
17	Novo Nordisk Investigational Site	Oklahoma City	Oklahoma	United States	73104
18	Novo Nordisk Investigational Site	Philadelphia	Pennsylvania	United States	19134
19	Novo Nordisk Investigational Site	Nashville	Tennessee	United States	37232
20	Novo Nordisk Investigational Site	Salt Lake City	Utah	United States	84113
21	Novo Nordisk Investigational Site	Norfolk	Virginia	United States	23507
22	Novo Nordisk Investigational Site	Algiers		Algeria	16000
23	Novo Nordisk Investigational Site	Setif		Algeria	19000
24	Novo Nordisk Investigational Site	Cordoba		Argentina	X5000FAL
25	Novo Nordisk Investigational Site	South Brisbane	Queensland	Australia	4101
26	Novo Nordisk Investigational Site	Parkville	Victoria	Australia	3052
27	Novo Nordisk Investigational Site	Graz		Austria	8036
28	Novo Nordisk Investigational Site	Innsbruck		Austria	6020
29	Novo Nordisk Investigational Site	Klagenfurt		Austria	9020
30	Novo Nordisk Investigational Site	Linz		Austria	4020
31	Novo Nordisk Investigational Site	Salzburg		Austria	A 5020
32	Novo Nordisk Investigational Site	St. Poelten		Austria	A 3100
33	Novo Nordisk Investigational Site	Wien		Austria	A 1090
34	Novo Nordisk Investigational Site	Plovdiv		Bulgaria	4002
35	Novo Nordisk Investigational Site	Hamilton	Ontario	Canada	L8N 3Z5
36	Novo Nordisk Investigational Site	Bron Cedex		France	69677
37	Novo Nordisk Investigational Site	Nantes Cedex 1		France	44093
38	Novo Nordisk Investigational Site	Paris		France	75015
39	Novo Nordisk Investigational Site	Duisburg		Germany	47051
40	Novo Nordisk Investigational Site	Hannover		Germany	30159
41	Novo Nordisk Investigational Site	Homburg/Saar		Germany	66424
42	Novo Nordisk Investigational Site	Mörfelden-Walldorf		Germany	64546
43	Novo Nordisk Investigational Site	Athens		Greece	GR-11527
44	Novo Nordisk Investigational Site	Thessaloniki		Greece	GR 54642
45	Novo Nordisk Investigational Site	Tel-Hashomer		Israel	52621
46	Novo Nordisk Investigational Site	Firenze		Italy	50134
47	Novo Nordisk Investigational Site	Firenze		Italy	50139
48	Novo Nordisk Investigational Site	Torino		Italy	10126
49	Novo Nordisk Investigational Site	Aichi		Japan	466-8560
50	Novo Nordisk Investigational Site	Hyogo		Japan	654-0047
51	Novo Nordisk Investigational Site	Kyoto		Japan	602-8566
52	Novo Nordisk Investigational Site	Saitama		Japan	330-8777
53	Novo Nordisk Investigational Site	Shizuoka		Japan	420-8660
54	Novo Nordisk Investigational Site	Tokyo		Japan	157-8535
55	Novo Nordisk Investigational Site	Georgetown, Penang		Malaysia	10450
56	Novo Nordisk Investigational Site	Kuala Lumpur		Malaysia	50300
57	Novo Nordisk Investigational Site	Kuala Lumpur		Malaysia	50400
58	Novo Nordisk Investigational Site	Timisoara	Timis	Romania	300011
59	Novo Nordisk Investigational Site	Bucharest		Romania	022328
60	Novo Nordisk Investigational Site	Constanta		Romania	900591
61	Novo Nordisk Investigational Site	A Coruña		Spain	15006
62	Novo Nordisk Investigational Site	Esplugues Llobregat		Spain	08950
63	Novo Nordisk Investigational Site	Madrid		Spain	28046
64	Novo Nordisk Investigational Site	Changhua		Taiwan	500
65	Novo Nordisk Investigational Site	Kaohsiung		Taiwan	807
66	Novo Nordisk Investigational Site	Taichung		Taiwan	40447
67	Novo Nordisk Investigational Site	Taipei		Taiwan	100
68	Novo Nordisk Investigational Site	Bangkok		Thailand	10400
69	Novo Nordisk Investigational Site	Chiang Mai		Thailand	50200
70	Novo Nordisk Investigational Site	Ubon Ratchathani		Thailand	34000