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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT02137850




Registration number
NCT02137850
Ethics application status
Date submitted
28/03/2014
Date registered
14/05/2014
Date last updated
26/04/2024

Titles & IDs
Public title
Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A
Scientific 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
Secondary ID [1] 0 0
2013-004025-88
Secondary ID [2] 0 0
NN7088-3908
Universal Trial Number (UTN)
Trial acronym
pathfinder™6
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

Experimental: 50 EDs (exposure days) -


Treatment: Drugs: 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
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of inhibitory antibodies against coagulation factor VIII (FVIII)
Timepoint [1] 0 0
When the first 50 PUP have reached at least 50 exposure dates. (Expected to reach between 6 - 18 months)
Primary outcome [2] 0 0
Incidence of inhibitory antibodies against coagulation factor VIII (FVIII)
Timepoint [2] 0 0
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 [1] 0 0
Frequency of adverse events including serious adverse events and medical events of special interest
Timepoint [1] 0 0
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
Secondary outcome [2] 0 0
Incidence of confirmed high titre inhibitors (defined as inhibitor titre above 5 Bethesda Units (BU)
Timepoint [2] 0 0
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
Secondary outcome [3] 0 0
Number of breakthrough bleeding episodes during prophylaxis with turoctocog alfa pegol (N8-GP) (annualised bleeding rate)
Timepoint [3] 0 0
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
Secondary outcome [4] 0 0
Haemostatic effect of N8-GP in treatment of bleeding episodes, assessed by a predefined 4-point haemostatic response scale ("excellent", "good", "moderate" and "none")
Timepoint [4] 0 0
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
Secondary outcome [5] 0 0
Consumption of N8-GP for prophylaxis (number of injections and U/Kg per month and per year)
Timepoint [5] 0 0
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
Secondary outcome [6] 0 0
Consumption of N8-GP for treatment of bleeding episodes (number of injections and U/Kg required per bleeding episode)
Timepoint [6] 0 0
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
Secondary outcome [7] 0 0
Total consumption of N8-GP per patient (prevention and treatment of bleeding episodes) per month and annualised value
Timepoint [7] 0 0
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
Secondary outcome [8] 0 0
Outcome of immune tolerance induction (ITI), assessed by a predefined 4-point ITI outcome scale ("success", "partial success", "failure", "other")
Timepoint [8] 0 0
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
Key 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)
Minimum age
0 Years
Maximum age
6 Years
Sex
Males
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
N/A
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
Single group
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
26/06/2014
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
7/06/2023
Sample size
Target
125
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
Lady Cilento Children's Hospital - South Brisbane
Recruitment hospital [2] 0 0
Royal Children's Hospital - Parkville
Recruitment postcode(s) [1] 0 0
4101 - South Brisbane
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
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Iowa
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
North Carolina
Country [7] 0 0
United States of America
State/province [7] 0 0
Ohio
Country [8] 0 0
United States of America
State/province [8] 0 0
Oklahoma
Country [9] 0 0
United States of America
State/province [9] 0 0
Utah
Country [10] 0 0
Algeria
State/province [10] 0 0
Algiers
Country [11] 0 0
Algeria
State/province [11] 0 0
Setif
Country [12] 0 0
Argentina
State/province [12] 0 0
Cordoba
Country [13] 0 0
Austria
State/province [13] 0 0
Graz
Country [14] 0 0
Austria
State/province [14] 0 0
Innsbruck
Country [15] 0 0
Austria
State/province [15] 0 0
Klagenfurt
Country [16] 0 0
Austria
State/province [16] 0 0
Linz
Country [17] 0 0
Austria
State/province [17] 0 0
Salzburg
Country [18] 0 0
Austria
State/province [18] 0 0
St. Poelten
Country [19] 0 0
Austria
State/province [19] 0 0
Wien
Country [20] 0 0
Bulgaria
State/province [20] 0 0
Plovdiv
Country [21] 0 0
Canada
State/province [21] 0 0
Ontario
Country [22] 0 0
France
State/province [22] 0 0
Bron Cedex
Country [23] 0 0
France
State/province [23] 0 0
Nantes Cedex 1
Country [24] 0 0
France
State/province [24] 0 0
Paris
Country [25] 0 0
Germany
State/province [25] 0 0
Hannover
Country [26] 0 0
Germany
State/province [26] 0 0
Homburg/Saar
Country [27] 0 0
Greece
State/province [27] 0 0
Athens
Country [28] 0 0
Greece
State/province [28] 0 0
Thessaloniki
Country [29] 0 0
Israel
State/province [29] 0 0
Tel-Hashomer
Country [30] 0 0
Italy
State/province [30] 0 0
Firenze
Country [31] 0 0
Italy
State/province [31] 0 0
Torino
Country [32] 0 0
Japan
State/province [32] 0 0
Aichi
Country [33] 0 0
Japan
State/province [33] 0 0
Hyogo
Country [34] 0 0
Japan
State/province [34] 0 0
Kyoto
Country [35] 0 0
Japan
State/province [35] 0 0
Saitama
Country [36] 0 0
Japan
State/province [36] 0 0
Shizuoka
Country [37] 0 0
Japan
State/province [37] 0 0
Tokyo
Country [38] 0 0
Malaysia
State/province [38] 0 0
Georgetown, Penang
Country [39] 0 0
Malaysia
State/province [39] 0 0
Kuala Lumpur
Country [40] 0 0
Romania
State/province [40] 0 0
Timis
Country [41] 0 0
Romania
State/province [41] 0 0
Bucharest
Country [42] 0 0
Romania
State/province [42] 0 0
Constanta
Country [43] 0 0
Spain
State/province [43] 0 0
A Coruña
Country [44] 0 0
Spain
State/province [44] 0 0
Esplugues Llobregat
Country [45] 0 0
Spain
State/province [45] 0 0
Madrid
Country [46] 0 0
Taiwan
State/province [46] 0 0
Taipei
Country [47] 0 0
Thailand
State/province [47] 0 0
Bangkok
Country [48] 0 0
Thailand
State/province [48] 0 0
Chiang Mai
Country [49] 0 0
Thailand
State/province [49] 0 0
Ubon Ratchathani

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

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT02137850
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Global Clinical Registry (GCR, 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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT02137850