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Trial details imported from ClinicalTrials.gov

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




Registration number
NCT03528551
Ethics application status
Date submitted
18/04/2018
Date registered
15/05/2018
Date last updated
29/01/2019

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
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system

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 - Count of adverse events
Timepoint [1] 0 0
Weeks 0-104
Secondary outcome [1] 0 0
Incidence of FVIII inhibitors =0.6 BU - Count of presence of inhibitors
Timepoint [1] 0 0
Weeks 0-104
Secondary outcome [2] 0 0
Number of bleeding episodes on prophylaxis - Count of episodes
Timepoint [2] 0 0
Weeks 0-104
Secondary outcome [3] 0 0
Number of spontaneous bleeding episodes on prophylaxis - Count of episodes
Timepoint [3] 0 0
Weeks 0-104
Secondary outcome [4] 0 0
Haemostatic effect of N8-GP when used for treatment of bleeding episodes assessed as: Excellent, Good, Moderate, or None - Assessed as: Excellent, good, moderate, or none
Timepoint [4] 0 0
Weeks 0-104
Secondary outcome [5] 0 0
Number of N8-GP injections required per bleeding episode - Number of injections
Timepoint [5] 0 0
Weeks 0-104
Secondary outcome [6] 0 0
Pre-dose FVIII activity levels on N8-GP prophylaxis (IU/dL) - Calculated based on plasma FVIII activity measured in blood
Timepoint [6] 0 0
Weeks 0-104
Secondary outcome [7] 0 0
Change in joint health status from start to end of trial (based on Haemophilia Joint Health Score) - 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 - Assessed as: Excellent, good, moderate, or none
Timepoint [8] 0 0
Weeks 0-104
Secondary outcome [9] 0 0
Consumption of N8-GP per bleed - Number of infusions and IU/kg per bleed
Timepoint [9] 0 0
Weeks 0-104
Secondary outcome [10] 0 0
Consumption of N8-GP during prophylaxis treatment - Number of infusions and IU/kg per month and per year
Timepoint [10] 0 0
Weeks 0-104
Secondary outcome [11] 0 0
Change from start till end of trial in treatment satisfaction (based on Hemo-SAT score) - Based on Haemophilia Satisfaction Survey (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
Gender
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
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)
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
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United States of America
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California
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Iowa
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United States of America
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Louisiana
Country [5] 0 0
United States of America
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Maryland
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United States of America
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Michigan
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Minnesota
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United States of America
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New York
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North Carolina
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Ohio
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Oregon
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Pennsylvania
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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
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France
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Nantes Cedex 1
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Germany
State/province [23] 0 0
Berlin
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Germany
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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
Country [29] 0 0
Israel
State/province [29] 0 0
Tel-Hashomer
Country [30] 0 0
Italy
State/province [30] 0 0
Milano
Country [31] 0 0
Italy
State/province [31] 0 0
Vicenza
Country [32] 0 0
Japan
State/province [32] 0 0
Kashihara-shi, Nara
Country [33] 0 0
Japan
State/province [33] 0 0
Kitakyusyu, Fukuoka
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Japan
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Suginami-ku, 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
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Lviv
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United Kingdom
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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 [59] 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
This study will look at how a known study medicine N8-GP works in previously N8-GP treated
people with haemophilia A. The aim is to look at how N8-GP works during regular use.
Participants will get N8-GP. N8-GP has been tested in more than 200 people with haemophilia A
for several years. Participants will get an injection of N8-GP into a blood vessel, one, two
or three times weekly. Participants will get more doses if they bleed or if they will need a
surgery. The study will last for about 2 years. Participants will have at least 9 visits with
the study doctor. If participants agree to be in this study, they will get their first
injection (in this study) at the first visit. Participants will also get an injection at
visit 3, 5 and 7. Participants will be trained to give all other injections themselves.
Participants must not use any clotting factors other than N8-GP or any anticoagulants (blood
thinners) during the study.
Trial website
https://clinicaltrials.gov/show/NCT03528551
Trial related presentations / publications
Public notes

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

No data has been provided for results reporting
Summary results
Not applicable