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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
Assessment method [1] 0 0
An adverse event (AE) was defined as any unfavourable and unintended sign (including an abnormal laboratory finding), symptom or disease temporally associated with the use of a product, whether or not considered related to the product. All AEs mentioned here are treatment emergent adverse events (TEAEs). The TEAE is defined as an event reported from date of first trial product administration until end of the treatment visit (week 104) or follow-up visit if relevant (1 month after end of the treatment).
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)
Assessment method [1] 0 0
The Incidence of inhibitors against coagulation factor eight (FVIII) is defined as titre greater than or equal to (=) 0.6 Bethesda unit. The inhibitor antibodies were measured using a heat modified Nijmegen FVIII Bethesda assay. The number of participants who developed inhibitors against FVIII are reported.
Timepoint [1] 0 0
Week 0 to week 104
Secondary outcome [2] 0 0
Number of Bleeding Episodes on Prophylaxis
Assessment method [2] 0 0
Number of bleeding episodes per participant in the prophylaxis regimen was evaluated during 104 weeks.
Timepoint [2] 0 0
Week 0 to week 104
Secondary outcome [3] 0 0
Number of Spontaneous Bleeding Episodes on Prophylaxis
Assessment method [3] 0 0
Spontaneous bleeding referred as bleeding episodes that occurred without apparent cause. The number of spontaneous bleeding episodes were evaluated during 104 weeks.
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
Assessment method [4] 0 0
The haemostatic effect after treatment of a bleed with N8-GP was assessed using a 4-point scale: 'excellent', 'good', 'moderate' or 'none'. The evaluation was done as follows: 1. Excellent: Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single injection. 2. Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hours after an injection, but possibly requiring more than one injection for complete resolution. 3. Moderate: Probable or slight beneficial effect within approximately 8 hours after the first injection; usually requiring more than one injection 4. None: No improvement or worsening of symptoms.
Timepoint [4] 0 0
Week 0 to week 104
Secondary outcome [5] 0 0
Mean Number of N8-GP Injections Required Per Bleeding Episode
Assessment method [5] 0 0
The mean number of N8-GP injections required per bleeding episode from start to stop of a bleed for participants was presented from week 0 to week 104.
Timepoint [5] 0 0
Week 0 to week 104
Secondary outcome [6] 0 0
Pre-dose FVIII Activity Levels on N8-GP Prophylaxis
Assessment method [6] 0 0
The pre-dose FVIII activity levels were assessed in International units per millilitre (IU/mL) units from week 0 to week 104 to get an estimate of the pre-dose level for N8-GP at steady-state using mixed model.
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)
Assessment method [7] 0 0
Haemophilia Joint Health Score is a validated outcome tool developed for the assessment of joint health in patients with hemophilia. It comprises an evaluation of the elbow, knee and ankle joints with regards to swelling, muscular atrophy, crepitation and range of motion, joint pain, strength, motion and axial alignment. The score range is from 0 to 24 points (a score of 0 indicates no joint damage. Higher the score higher the joint damage). Change from week 0 to end of trial (week 104) in the domain scores was presented.
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
Assessment method [8] 0 0
The Haemostatic response to N8-GP during major surgical interventions was assessed using a 4-point scale: 'excellent', 'good', 'moderate' or 'none'. The evaluation was done as follows: 1. Excellent: Better than expected/predicted in this type of procedure. 2. Good: As expected in this type of procedure 3. Moderate: Less than optimal for the type of procedure but haemostatic response maintained without change of treatment regimen 4. None: Bleeding due to inadequate therapeutic response with adequate dosing, change of regimen required. This endpoint was measured from week 0 to week 104.
Timepoint [8] 0 0
Week 0 to week 104
Secondary outcome [9] 0 0
Consumption of N8-GP Per Bleed
Assessment method [9] 0 0
The average dose of N8-GP consumed for treatment of bleed was assessed in International units per kilogram per bleed(IU/kg/bleed). This endpoint was evaluated from week 0 to week 104.
Timepoint [9] 0 0
Week 0 to week 104
Secondary outcome [10] 0 0
Consumption of N8-GP During Prophylaxis Treatment
Assessment method [10] 0 0
The average dose of N8-GP consumed for prevention of bleed was assessed. This endpoint was evaluated from week 0 to week 104.
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)
Assessment method [11] 0 0
The treatment satisfaction of a bleed with N8-GP was assessed using HEMO-SAT assessment tool which contains a questionnaire with 6 domains (Ease and convenience, efficacy, burden, specialist/nurses, centre/hospital, general satisfaction), with a scale of 0-100. The lower scores reflecting greater treatment satisfaction. In other words, decrease in the score would mean improvement. The summary of change presented was based on individual changes since week 0. Data is presented for total 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
State/province [11] 0 0
Oregon
Country [12] 0 0
United States of America
State/province [12] 0 0
Pennsylvania
Country [13] 0 0
United States of America
State/province [13] 0 0
South Carolina
Country [14] 0 0
United States of America
State/province [14] 0 0
Tennessee
Country [15] 0 0
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
Country [21] 0 0
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.
Country [26] 0 0
Germany
State/province [26] 0 0
Homburg
Country [27] 0 0
Greece
State/province [27] 0 0
Athens
Country [28] 0 0
Hungary
State/province [28] 0 0
Budapest
Country [29] 0 0
Hungary
State/province [29] 0 0
Debrecen
Country [30] 0 0
Israel
State/province [30] 0 0
Tel-Hashomer
Country [31] 0 0
Italy
State/province [31] 0 0
Milano
Country [32] 0 0
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
Country [35] 0 0
Japan
State/province [35] 0 0
Tokyo
Country [36] 0 0
Korea, Republic of
State/province [36] 0 0
Daejeon
Country [37] 0 0
Lithuania
State/province [37] 0 0
Vilnius
Country [38] 0 0
Malaysia
State/province [38] 0 0
Selangor Darul Ehsan
Country [39] 0 0
Netherlands
State/province [39] 0 0
Groningen
Country [40] 0 0
Netherlands
State/province [40] 0 0
Rotterdam
Country [41] 0 0
Norway
State/province [41] 0 0
Oslo
Country [42] 0 0
Portugal
State/province [42] 0 0
Porto
Country [43] 0 0
Puerto Rico
State/province [43] 0 0
San Juan
Country [44] 0 0
Spain
State/province [44] 0 0
Madrid
Country [45] 0 0
Spain
State/province [45] 0 0
Málaga
Country [46] 0 0
Switzerland
State/province [46] 0 0
Bellinzona
Country [47] 0 0
Switzerland
State/province [47] 0 0
Lausanne
Country [48] 0 0
Switzerland
State/province [48] 0 0
Zürich
Country [49] 0 0
Taiwan
State/province [49] 0 0
Taipei
Country [50] 0 0
Turkey
State/province [50] 0 0
Adana
Country [51] 0 0
Turkey
State/province [51] 0 0
Antalya
Country [52] 0 0
Turkey
State/province [52] 0 0
Bornova-IZMIR
Country [53] 0 0
Turkey
State/province [53] 0 0
Izmit
Country [54] 0 0
Turkey
State/province [54] 0 0
Samsun
Country [55] 0 0
Ukraine
State/province [55] 0 0
Lviv
Country [56] 0 0
United Kingdom
State/province [56] 0 0
Basingstoke
Country [57] 0 0
United Kingdom
State/province [57] 0 0
Cardiff
Country [58] 0 0
United Kingdom
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London
Country [59] 0 0
United Kingdom
State/province [59] 0 0
Oxford
Country [60] 0 0
United Kingdom
State/province [60] 0 0
Sheffield

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

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
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
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

TypeOther DetailsAttachment
Study protocol
Statistical analysis plan



Results publications and other study-related documents

No documents have been uploaded by study researchers.


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