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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/NCT03528551
Registration number
NCT03528551
Ethics application status
Date submitted
18/04/2018
Date registered
18/05/2018
Date last updated
22/12/2022
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
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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
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Secondary ID [1]
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U1111-1202-2780
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Secondary ID [2]
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NN7088-4410
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Universal Trial Number (UTN)
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Trial acronym
pathfinder8
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Congenital Bleeding Disorder
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Haemophilia A
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Condition category
Condition code
Cardiovascular
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Diseases of the vasculature and circulation including the lymphatic system
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Blood
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Clotting disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Intervention/exposure
Study type
Interventional
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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.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of Adverse Events Reported
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Assessment method [1]
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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).
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Timepoint [1]
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Week 0 to week 108
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Secondary outcome [1]
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Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) =0.6 Bethesda Units (BU)
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Assessment method [1]
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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.
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Timepoint [1]
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Week 0 to week 104
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Secondary outcome [2]
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Number of Bleeding Episodes on Prophylaxis
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Assessment method [2]
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Number of bleeding episodes per participant in the prophylaxis regimen was evaluated during 104 weeks.
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Timepoint [2]
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Week 0 to week 104
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Secondary outcome [3]
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Number of Spontaneous Bleeding Episodes on Prophylaxis
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Assessment method [3]
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Spontaneous bleeding referred as bleeding episodes that occurred without apparent cause. The number of spontaneous bleeding episodes were evaluated during 104 weeks.
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Timepoint [3]
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Week 0 to week 104
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Secondary outcome [4]
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Haemostatic Effect of N8-GP When Used for Treatment of Bleeding Episodes Assessed as: Excellent, Good, Moderate, or None
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Assessment method [4]
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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.
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Timepoint [4]
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Week 0 to week 104
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Secondary outcome [5]
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Mean Number of N8-GP Injections Required Per Bleeding Episode
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Assessment method [5]
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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.
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Timepoint [5]
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Week 0 to week 104
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Secondary outcome [6]
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Pre-dose FVIII Activity Levels on N8-GP Prophylaxis
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Assessment method [6]
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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.
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Timepoint [6]
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Week 0 to week 104
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Secondary outcome [7]
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Change in Joint Health Status From Start to End of Trial (Based on Haemophilia Joint Health Score)
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Assessment method [7]
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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.
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Timepoint [7]
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Week 0, Week 104
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Secondary outcome [8]
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Haemostatic Response During Major Surgical Interventions Assessed as: Excellent, Good, Moderate, or None
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Assessment method [8]
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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.
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Timepoint [8]
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Week 0 to week 104
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Secondary outcome [9]
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Consumption of N8-GP Per Bleed
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Assessment method [9]
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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.
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Timepoint [9]
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Week 0 to week 104
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Secondary outcome [10]
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Consumption of N8-GP During Prophylaxis Treatment
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Assessment method [10]
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The average dose of N8-GP consumed for prevention of bleed was assessed. This endpoint was evaluated from week 0 to week 104.
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Timepoint [10]
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Week 0 to week 104
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Secondary outcome [11]
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Change From Start Till End of Trial in Treatment Satisfaction (Based on Hemo-SAT Score)
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Assessment method [11]
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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.
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Timepoint [11]
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Week 0, Week 104
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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
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Minimum age
No limit
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Maximum age
No limit
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Sex
Males
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Can healthy volunteers participate?
No
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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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
30/04/2018
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
3/12/2020
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Sample size
Target
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Accrual to date
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Final
160
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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Novo Nordisk Investigational Site - Camperdown
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Recruitment hospital [2]
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Novo Nordisk Investigational Site - Parkville
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Recruitment postcode(s) [1]
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2050 - Camperdown
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Recruitment postcode(s) [2]
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3052 - Parkville
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Recruitment outside Australia
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United States of America
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Arizona
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California
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Iowa
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Louisiana
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Kitakyusyu-shi, Fukuoka
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Nara
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Lausanne
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Lviv
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Oxford
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Sheffield
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Novo Nordisk A/S
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Address
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Ethics approval
Ethics application status
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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.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT03528551
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Clinical Reporting Anchor and Disclosure 2834
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Address
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Novo Nordisk A/S
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT03528551
Download to PDF