Trial Review

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Trial details imported from ClinicalTrials.gov

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

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results not provided in https://clinicaltrials.gov/study/NCT02137850