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

Primary Purpose

Congenital Bleeding Disorder, Haemophilia A

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
turoctocog alfa 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 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

Sites / Locations

  • 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
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  • 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

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 VIII (FVIII)
Incidence of inhibitory antibodies against coagulation factor VIII (FVIII)

Secondary Outcome Measures

Frequency of adverse events including serious adverse events and medical events of special interest
Incidence of confirmed high titre inhibitors (defined as inhibitor titre above 5 Bethesda Units (BU)
Number of breakthrough bleeding episodes during prophylaxis with turoctocog alfa pegol (N8-GP) (annualised bleeding rate)
Haemostatic effect of N8-GP in treatment of bleeding episodes, assessed by a predefined 4-point haemostatic response scale ("excellent", "good", "moderate" and "none")
Consumption of N8-GP for prophylaxis (number of injections and U/Kg per month and per year)
Consumption of N8-GP for treatment of bleeding episodes (number of injections and U/Kg required per bleeding episode)
Total consumption of N8-GP per patient (prevention and treatment of bleeding episodes) per month and annualised value
Outcome of immune tolerance induction (ITI), assessed by a predefined 4-point ITI outcome scale ("success", "partial success", "failure", "other")

Full Information

First Posted
March 28, 2014
Last Updated
June 10, 2023
Sponsor
Novo Nordisk A/S
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1. Study Identification

Unique Protocol Identification Number
NCT02137850
Brief Title
Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A
Acronym
pathfinder™6
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
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
June 26, 2014 (Actual)
Primary Completion Date
June 17, 2023 (Anticipated)
Study Completion Date
June 17, 2023 (Anticipated)

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

6. Conditions and Keywords

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

7. Study Design

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

8. Arms, Groups, and Interventions

Arm Title
50 EDs (exposure days)
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
turoctocog alfa pegol
Other Intervention Name(s)
NNC 0129-0000-1003, N8-GP
Intervention Description
For intravenous (i.v.) injection. Frequency and dosage (20-75 U/kg) dependent on whether given as treatment for bleeding episode or as prophylaxis
Primary Outcome Measure Information:
Title
Incidence of inhibitory antibodies against coagulation factor VIII (FVIII)
Time Frame
When the first 50 PUP have reached at least 50 exposure dates. (Expected to reach between 6 - 18 months)
Title
Incidence of inhibitory antibodies against coagulation factor VIII (FVIII)
Time Frame
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 Measure Information:
Title
Frequency of adverse events including serious adverse events and medical events of special interest
Time Frame
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
Title
Incidence of confirmed high titre inhibitors (defined as inhibitor titre above 5 Bethesda Units (BU)
Time Frame
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
Title
Number of breakthrough bleeding episodes during prophylaxis with turoctocog alfa pegol (N8-GP) (annualised bleeding rate)
Time Frame
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
Title
Haemostatic effect of N8-GP in treatment of bleeding episodes, assessed by a predefined 4-point haemostatic response scale ("excellent", "good", "moderate" and "none")
Time Frame
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
Title
Consumption of N8-GP for prophylaxis (number of injections and U/Kg per month and per year)
Time Frame
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
Title
Consumption of N8-GP for treatment of bleeding episodes (number of injections and U/Kg required per bleeding episode)
Time Frame
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
Title
Total consumption of N8-GP per patient (prevention and treatment of bleeding episodes) per month and annualised value
Time Frame
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
Title
Outcome of immune tolerance induction (ITI), assessed by a predefined 4-point ITI outcome scale ("success", "partial success", "failure", "other")
Time Frame
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

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 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
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
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016-7710
Country
United States
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
Torrance
State/Province
California
ZIP/Postal Code
90502-2004
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
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
New Hyde Park
State/Province
New York
ZIP/Postal Code
11042
Country
United States
Facility Name
Novo Nordisk Investigational Site
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28203
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
Oklahoma City
State/Province
Oklahoma
ZIP/Postal Code
73104
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
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
Plovdiv
ZIP/Postal Code
4002
Country
Bulgaria
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
Bron Cedex
ZIP/Postal Code
69677
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
Paris
ZIP/Postal Code
75015
Country
France
Facility Name
Novo Nordisk Investigational Site
City
Hannover
ZIP/Postal Code
30159
Country
Germany
Facility Name
Novo Nordisk Investigational Site
City
Homburg/Saar
ZIP/Postal Code
66424
Country
Germany
Facility Name
Novo Nordisk Investigational Site
City
Athens
ZIP/Postal Code
GR-11527
Country
Greece
Facility Name
Novo Nordisk Investigational Site
City
Thessaloniki
ZIP/Postal Code
GR 54642
Country
Greece
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
Firenze
ZIP/Postal Code
50139
Country
Italy
Facility Name
Novo Nordisk Investigational Site
City
Torino
ZIP/Postal Code
10126
Country
Italy
Facility Name
Novo Nordisk Investigational Site
City
Aichi
ZIP/Postal Code
466-8560
Country
Japan
Facility Name
Novo Nordisk Investigational Site
City
Hyogo
ZIP/Postal Code
654-0047
Country
Japan
Facility Name
Novo Nordisk Investigational Site
City
Kyoto
ZIP/Postal Code
602-8566
Country
Japan
Facility Name
Novo Nordisk Investigational Site
City
Saitama
ZIP/Postal Code
330-8777
Country
Japan
Facility Name
Novo Nordisk Investigational Site
City
Shizuoka
ZIP/Postal Code
420-8660
Country
Japan
Facility Name
Novo Nordisk Investigational Site
City
Tokyo
ZIP/Postal Code
157-8535
Country
Japan
Facility Name
Novo Nordisk Investigational Site
City
Georgetown, Penang
ZIP/Postal Code
10450
Country
Malaysia
Facility Name
Novo Nordisk Investigational Site
City
Kuala Lumpur
ZIP/Postal Code
50300
Country
Malaysia
Facility Name
Novo Nordisk Investigational Site
City
Kuala Lumpur
ZIP/Postal Code
50400
Country
Malaysia
Facility Name
Novo Nordisk Investigational Site
City
Timisoara
State/Province
Timis
ZIP/Postal Code
300011
Country
Romania
Facility Name
Novo Nordisk Investigational Site
City
Bucharest
ZIP/Postal Code
022328
Country
Romania
Facility Name
Novo Nordisk Investigational Site
City
Constanta
ZIP/Postal Code
900591
Country
Romania
Facility Name
Novo Nordisk Investigational Site
City
A Coruña
ZIP/Postal Code
15006
Country
Spain
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
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
Ubon Ratchathani
ZIP/Postal Code
34000
Country
Thailand

12. IPD Sharing Statement

Links:
URL
http://novonordisk-trials.com
Description
Clinical Trials at Novo Nordisk

Learn more about this trial

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

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