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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03528551




Registration number
NCT03528551
Ethics application status
Date submitted
18/04/2018
Date registered
18/05/2018

Titles & IDs
Public title
A Research Study Looking at How a Factor VIII Medicine Called Turoctocog Alfa Pegol (N8-GP) Works in People With Haemophilia A
Scientific title
Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Prophylaxis and Treatment of Bleeds in Previously N8-GP Treated Patients With Severe Haemophilia A
Secondary ID [1] 0 0
U1111-1202-2780
Secondary ID [2] 0 0
NN7088-4410
Universal Trial Number (UTN)
Trial acronym
pathfinder8
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Congenital Bleeding Disorder 0 0
Haemophilia A 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 - Turoctocog alfa pegol
Treatment: Drugs - Turoctocog alfa pegol
Treatment: Drugs - Turoctocog alfa pegol

Experimental: N8-GP, once weekly - All participants will receive turoctocog alfa pegol (N8-GP) once weekly.

Experimental: N8-GP, twice weekly - All participants will receive N8-GP twice weekly.

Experimental: N8-GP, three times weekly - All participants will receive N8-GP three times weekly.


Treatment: Drugs: Turoctocog alfa pegol
Turoctocog alfa pegol 75 IU/kg body weight will be administered once weekly as intravenous injections for a duration of 2 years.

Treatment: Drugs: Turoctocog alfa pegol
Turoctocog alfa pegol 60 IU/kg body weight (for patients younger than 12 years) and 50 IU/kg body weight (for patients, 12 years or older) will be administered twice weekly as intravenous injections for a duration of 2 years.

Treatment: Drugs: Turoctocog alfa pegol
Turoctocog alfa pegol 50 IU/kg body weight will be administered three times weekly as intravenous injections for a duration of 2 years.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Adverse Events Reported
Timepoint [1] 0 0
Week 0 to week 108
Secondary outcome [1] 0 0
Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) =0.6 Bethesda Units (BU)
Timepoint [1] 0 0
Week 0 to week 104
Secondary outcome [2] 0 0
Number of Bleeding Episodes on Prophylaxis
Timepoint [2] 0 0
Week 0 to week 104
Secondary outcome [3] 0 0
Number of Spontaneous Bleeding Episodes on Prophylaxis
Timepoint [3] 0 0
Week 0 to week 104
Secondary outcome [4] 0 0
Haemostatic Effect of N8-GP When Used for Treatment of Bleeding Episodes Assessed as: Excellent, Good, Moderate, or None
Timepoint [4] 0 0
Week 0 to week 104
Secondary outcome [5] 0 0
Mean Number of N8-GP Injections Required Per Bleeding Episode
Timepoint [5] 0 0
Week 0 to week 104
Secondary outcome [6] 0 0
Pre-dose FVIII Activity Levels on N8-GP Prophylaxis
Timepoint [6] 0 0
Week 0 to week 104
Secondary outcome [7] 0 0
Change in Joint Health Status From Start to End of Trial (Based on Haemophilia Joint Health Score)
Timepoint [7] 0 0
Week 0, Week 104
Secondary outcome [8] 0 0
Haemostatic Response During Major Surgical Interventions Assessed as: Excellent, Good, Moderate, or None
Timepoint [8] 0 0
Week 0 to week 104
Secondary outcome [9] 0 0
Consumption of N8-GP Per Bleed
Timepoint [9] 0 0
Week 0 to week 104
Secondary outcome [10] 0 0
Consumption of N8-GP During Prophylaxis Treatment
Timepoint [10] 0 0
Week 0 to week 104
Secondary outcome [11] 0 0
Change From Start Till End of Trial in Treatment Satisfaction (Based on Hemo-SAT Score)
Timepoint [11] 0 0
Week 0, Week 104

Eligibility
Key inclusion criteria
* Male patients of all ages with the diagnosis of severe congenital haemophilia A (coagulation Factor VIII [FVIII] activity less than 1%) based on medical records
* On-going participation in NN7088-3859 (pathfinder2), or NN7088-3885 (pathfinder5) at the time of transfer
Minimum age
No limit
Maximum age
No limit
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
* Known or suspected hypersensitivity to trial product including allergy to hamster protein or related products
* Any disorder, except for conditions associated with haemophilia, which in the investigator's opinion might jeopardise patient's safety or compliance with the protocol - Current participation in any clinical trial (except NN7088-3859 (pathfinder2) or NN7088-3885 (pathfinder5)) of an approved or non-approved investigational medicinal product

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
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
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
30/04/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
3/12/2020
Sample size
Target
Accrual to date
Final
160
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment hospital [1] 0 0
Novo Nordisk Investigational Site - Camperdown
Recruitment hospital [2] 0 0
Novo Nordisk Investigational Site - Parkville
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arizona
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Iowa
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
Michigan
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
New York
Country [9] 0 0
United States of America
State/province [9] 0 0
North Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Ohio
Country [11] 0 0
United States of America
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Oregon
Country [12] 0 0
United States of America
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Pennsylvania
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United States of America
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South Carolina
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United States of America
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Tennessee
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United States of America
State/province [15] 0 0
Texas
Country [16] 0 0
United States of America
State/province [16] 0 0
Virginia
Country [17] 0 0
Brazil
State/province [17] 0 0
Sao Paulo
Country [18] 0 0
Canada
State/province [18] 0 0
Ontario
Country [19] 0 0
Croatia
State/province [19] 0 0
Zagreb
Country [20] 0 0
Denmark
State/province [20] 0 0
Århus N
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France
State/province [21] 0 0
Bron Cedex
Country [22] 0 0
France
State/province [22] 0 0
Le Kremlin Bicetre
Country [23] 0 0
France
State/province [23] 0 0
Nantes Cedex 1
Country [24] 0 0
Germany
State/province [24] 0 0
Berlin
Country [25] 0 0
Germany
State/province [25] 0 0
Frankfurt/M.
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Germany
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Homburg
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Greece
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Athens
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Hungary
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Budapest
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Hungary
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Debrecen
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Israel
State/province [30] 0 0
Tel-Hashomer
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Italy
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Milano
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Italy
State/province [32] 0 0
Vicenza
Country [33] 0 0
Japan
State/province [33] 0 0
Kitakyusyu-shi, Fukuoka
Country [34] 0 0
Japan
State/province [34] 0 0
Nara
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Japan
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Tokyo
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Korea, Republic of
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Daejeon
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Lithuania
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Vilnius
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Malaysia
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Selangor Darul Ehsan
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Netherlands
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Groningen
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Netherlands
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Rotterdam
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Norway
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Oslo
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Portugal
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Porto
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Puerto Rico
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San Juan
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Spain
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Madrid
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Spain
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Málaga
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Switzerland
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Bellinzona
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Switzerland
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Lausanne
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Switzerland
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Zürich
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Taiwan
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Taipei
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Turkey
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Adana
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Turkey
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Antalya
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Turkey
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Bornova-IZMIR
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Turkey
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Izmit
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Turkey
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Samsun
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Ukraine
State/province [55] 0 0
Lviv
Country [56] 0 0
United Kingdom
State/province [56] 0 0
Basingstoke
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United Kingdom
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Cardiff
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United Kingdom
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London
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United Kingdom
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Oxford
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United Kingdom
State/province [60] 0 0
Sheffield

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clinical Reporting Anchor and Disclosure 2834
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
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
When will data be available (start and end dates)?
Available to whom?
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: http://novonordisk-trials.com


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03528551