Congenital Bleeding DisorderPhase 3pathfinder8NCT03528551
A Research Study Looking at How a Factor VIII Medicine Called Turoctocog Alfa Pegol (N8-GP) Works in People With Haemophilia A
A Phase 3 study testing Turoctocog alfa pegol in people with Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 67 sites in 26 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Congenital Bleeding Disorder, Haemophilia A
- What is being tested?
- Turoctocog alfa pegol
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 160 participants
- Where
- 67 sitesin 26 countries
- Study dates
- 30 Apr 2018to 3 Dec 2020
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
67 sites in 26 countries.
67 sites in 26 countries
United States18 sites
Novo Nordisk Investigational Site
Baltimore, Maryland, 21205
Novo Nordisk Investigational Site
Charleston, South Carolina, 29425
Novo Nordisk Investigational Site
Charlotte, North Carolina, 28204
Novo Nordisk Investigational Site
Cincinnati, Ohio, 45229
Novo Nordisk Investigational Site
Dayton, Ohio, 45404
Novo Nordisk Investigational Site
Detroit, Michigan, 48201
Novo Nordisk Investigational Site
East Lansing, Michigan, 48824
Novo Nordisk Investigational Site
Houston, Texas, 77030
Novo Nordisk Investigational Site
Iowa City, Iowa, 52242
Novo Nordisk Investigational Site
Long Beach, California, 90806
Novo Nordisk Investigational Site
Minneapolis, Minnesota, 55404
Novo Nordisk Investigational Site
Nashville, Tennessee, 37232-9830
Novo Nordisk Investigational Site
New Hyde Park, New York, 11040
Novo Nordisk Investigational Site
New Orleans, Louisiana, 70118-5720
Novo Nordisk Investigational Site
Norfolk, Virginia, 23507
Novo Nordisk Investigational Site
Philadelphia, Pennsylvania, 19104
Novo Nordisk Investigational Site
Phoenix, Arizona, 85016-7710
Novo Nordisk Investigational Site
Portland, Oregon, 97239
United Kingdom7 sites
Novo Nordisk Investigational Site
Basingstoke, RG24 9NA
Novo Nordisk Investigational Site
Cardiff, CF14 4XW
Novo Nordisk Investigational Site
London, NW3 2QG
Novo Nordisk Investigational Site
London, SE1 7EH
Novo Nordisk Investigational Site
Oxford, OX3 7LJ
Novo Nordisk Investigational Site
Oxford, OX3 9DU
Novo Nordisk Investigational Site
Sheffield, S10 2JF
Turkey (Türkiye)5 sites
Novo Nordisk Investigational Site
Adana, 01130
Novo Nordisk Investigational Site
Antalya, 01010
Novo Nordisk Investigational Site
Bornova-IZMIR, 35100
Novo Nordisk Investigational Site
İzmit, 41380
Novo Nordisk Investigational Site
Samsun, 55139
Switzerland4 sites
Novo Nordisk Investigational Site
Bellinzona, 6500
Novo Nordisk Investigational Site
Lausanne, 1011
Novo Nordisk Investigational Site
Zurich, 8032
Novo Nordisk Investigational Site
Zurich, 8091
France3 sites
Novo Nordisk Investigational Site
Bron, 69677
Novo Nordisk Investigational Site
Le Kremlin-Bicêtre, 94270
Novo Nordisk Investigational Site
Nantes, 44093
Germany3 sites
Novo Nordisk Investigational Site
Berlin, 10249
Novo Nordisk Investigational Site
Frankfurt/M., 60590
Novo Nordisk Investigational Site
Homburg, 66421
Japan3 sites
Novo Nordisk Investigational Site
Kitakyusyu-shi, Fukuoka, 807 8555
Novo Nordisk Investigational Site
Nara, 634-8522
Novo Nordisk Investigational Site
Tokyo, 167-0035
Australia2 sites
Novo Nordisk Investigational Site
Camperdown, New South Wales, 2050
Novo Nordisk Investigational Site
Parkville, Victoria, 3052
Hungary2 sites
Novo Nordisk Investigational Site
Budapest, H-1134
Novo Nordisk Investigational Site
Debrecen, 4012
Italy2 sites
Novo Nordisk Investigational Site
Milan, 20124
Novo Nordisk Investigational Site
Vicenza, 36100
Netherlands2 sites
Novo Nordisk Investigational Site
Groningen, 9713 GZ
Novo Nordisk Investigational Site
Rotterdam, 3015 CE
Spain2 sites
Novo Nordisk Investigational Site
Madrid, 28046
Novo Nordisk Investigational Site
Málaga, 29010
About this study
Official 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
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.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A
- Being tested
- Turoctocog alfa pegol
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Turoctocog alfa pegolDrug
Turoctocog alfa pegol 75 IU/kg body weight will be administered once weekly as intravenous injections for a duration of 2 years.
Turoctocog alfa pegolDrug
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.
Turoctocog alfa pegolDrug
Turoctocog alfa pegol 50 IU/kg body weight will be administered three times weekly as intravenous injections for a duration of 2 years.
| Study group | Role | What they receive |
|---|---|---|
| N8-GP, once weekly | Experimental | All participants will receive turoctocog alfa pegol (N8-GP) once weekly. Receives: Drug: Turoctocog alfa pegol |
| N8-GP, twice weekly | Experimental | All participants will receive N8-GP twice weekly. Receives: Drug: Turoctocog alfa pegol |
| N8-GP, three times weekly | Experimental | All participants will receive N8-GP three times weekly. Receives: Drug: Turoctocog alfa pegol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Adverse Events Reported
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).
Measured: Week 0 to week 108
Secondary outcomes (11)
Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) ≥0.6 Bethesda Units (BU)
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.
Measured: Week 0 to week 104
Number of Bleeding Episodes on Prophylaxis
Number of bleeding episodes per participant in the prophylaxis regimen was evaluated during 104 weeks.
Measured: Week 0 to week 104
Number of Spontaneous Bleeding Episodes on Prophylaxis
Spontaneous bleeding referred as bleeding episodes that occurred without apparent cause. The number of spontaneous bleeding episodes were evaluated during 104 weeks.
Measured: Week 0 to week 104
Haemostatic Effect of N8-GP When Used for Treatment of Bleeding Episodes Assessed as: Excellent, Good, Moderate, or None
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.
Measured: Week 0 to week 104
Mean Number of N8-GP Injections Required Per Bleeding Episode
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.
Measured: Week 0 to week 104
Pre-dose FVIII Activity Levels on N8-GP Prophylaxis
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.
Measured: Week 0 to week 104
Change in Joint Health Status From Start to End of Trial (Based on Haemophilia Joint Health Score)
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.
Measured: Week 0, Week 104
Haemostatic Response During Major Surgical Interventions Assessed as: Excellent, Good, Moderate, or None
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.
Measured: Week 0 to week 104
Consumption of N8-GP Per Bleed
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.
Measured: Week 0 to week 104
Consumption of N8-GP During Prophylaxis Treatment
The average dose of N8-GP consumed for prevention of bleed was assessed. This endpoint was evaluated from week 0 to week 104.
Measured: Week 0 to week 104
Change From Start Till End of Trial in Treatment Satisfaction (Based on Hemo-SAT Score)
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.
Measured: Week 0, Week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2018
- Study start30 Apr 2018
- Primary completion3 Dec 2020
- Study completion3 Dec 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Congenital Bleeding DisorderStudies of the same condition, open and under way.
- More trials run by Novo Nordisk A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 160 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Reporting Anchor and Disclosure 2834, Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03528551
- Sponsor's protocol ID
- NN7088-4410
- Other IDs
- U1111-1202-2780, 2017-003788-36, JapicCTI-183952, 2017
- Acronym
- pathfinder8
- Status last verified
- Dec 2022
- First submitted
- 18 Apr 2018
- Last updated
- 22 Dec 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
Study at a glance
From the registry record, last updated by the sponsor on 22 Dec 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT03528551
- Acronym / sponsor ID
- pathfinder8 / NN7088-4410
- Conditions
- Congenital Bleeding Disorder, Haemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 160 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 30 Apr 2018 (actual)
- Primary completion
- 3 Dec 2020 (actual)
- Study completion
- 3 Dec 2020 (actual)
- First posted
- 18 May 2018 (actual)
- Results
- Posted 26 Nov 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| N8-GP, once weekly | Experimental | Drug: Turoctocog alfa pegolAll participants will receive turoctocog alfa pegol (N8-GP) once weekly. |
| N8-GP, twice weekly | Experimental | Drug: Turoctocog alfa pegolAll participants will receive N8-GP twice weekly. |
| N8-GP, three times weekly | Experimental | Drug: Turoctocog alfa pegolAll participants will receive N8-GP three times weekly. |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Turoctocog alfa pegolTuroctocog alfa pegol 75 IU/kg body weight will be administered once weekly as intravenous injections for a duration of 2 years. | Drug | N8-GP, once weekly |
| Turoctocog alfa pegolTuroctocog 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. | Drug | N8-GP, twice weekly |
| Turoctocog alfa pegolTuroctocog alfa pegol 50 IU/kg body weight will be administered three times weekly as intravenous injections for a duration of 2 years. | Drug | N8-GP, three times weekly |
Primary outcome
Number of Adverse Events Reported
Time frame: Week 0 to week 108
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).
11 secondary outcomes
Number of Participants With Inhibitory Antibodies Against Coagulation Factor VIII (FVIII) ≥0.6 Bethesda Units (BU)
Time frame: Week 0 to week 104
Number of Bleeding Episodes on Prophylaxis
Time frame: Week 0 to week 104
Number of Spontaneous Bleeding Episodes on Prophylaxis
Time frame: Week 0 to week 104
Haemostatic Effect of N8-GP When Used for Treatment of Bleeding Episodes Assessed as: Excellent, Good, Moderate, or None
Time frame: Week 0 to week 104
Mean Number of N8-GP Injections Required Per Bleeding Episode
Time frame: Week 0 to week 104
Pre-dose FVIII Activity Levels on N8-GP Prophylaxis
Time frame: Week 0 to week 104
Change in Joint Health Status From Start to End of Trial (Based on Haemophilia Joint Health Score)
Time frame: Week 0, Week 104
Haemostatic Response During Major Surgical Interventions Assessed as: Excellent, Good, Moderate, or None
Time frame: Week 0 to week 104
Consumption of N8-GP Per Bleed
Time frame: Week 0 to week 104
Consumption of N8-GP During Prophylaxis Treatment
Time frame: Week 0 to week 104
Change From Start Till End of Trial in Treatment Satisfaction (Based on Hemo-SAT Score)
Time frame: Week 0, Week 104
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Dec 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: Yes. According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Reporting Anchor and Disclosure 2834 study director
Novo Nordisk A/S
67 sites in 26 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Turoctocog alfa pegol in people with Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 67 sites in 26 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Dec 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 67 sites in United States, United Kingdom, Turkey (Türkiye), Switzerland, France and 21 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 26 Nov 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Adverse Events Reported (time frame: Week 0 to week 108). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Dec 2022.