Congenital Bleeding DisorderPhase 3pathfinder™3NCT01489111
Evaluating the Haemostatic Effect of NNC 0129-0000-1003 During Surgical Procedures in Subjects 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 70 sites in 21 countries.
Trial summary
This trial is conducted globally. The aim of this trial is to evaluate the haemostatic effect of NNC 0129-0000-1003 during surgical procedures in subjects with haemophilia A. Read more about this study
- Who is it for?
- Ages 12 years and older, 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
- 36 participants
- Where
- 70 sitesin 21 countries
- Study dates
- 3 Aug 2012to 10 Dec 2018
- 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 12 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Informed consent obtained before any trial-related activities. (Trial-related activities are any procedure that would not have been performed during normal management of the subject.)
- Ongoing participation in the pathfinder™2 (NN7088-3859) or the pathfinderTM 4 (NN7088-3861) trial and having received greater than or equal to 5 doses of N8-GP
- Undergoing major surgery requiring daily monitoring of FVIII:C (FVIII activity) and wound status for at least 3 days
- The patient and/or Legally Acceptable Representative (LAR) is capable of assessing a bleeding episode, keeping an eDiary, capable of home treatment of bleeding episodes and otherwise capable of following the trial procedures
Who cannot join
- Known or suspected hypersensitivity to trial product including allergy to hamster protein or related products
- Previous withdrawal from the pathfinder™2 (NN7088-3859) or the pathfinderTM 4 (NN7088-3861) trial after administration of trial product, except interruption due to inclusion in this pathfinderTM 3 trial (NN7088-3860)
- The receipt of any investigational medicinal product (except N8-GP) within 30 days prior to enrolment into the trial. (For Brazil, only: Participation in a previous clinical trial within one year prior to screening for this trial (Visit 1), unless there is a direct benefit to the research subject, at the Investigator's discretion)
- FVIII inhibitors at least 0.6 BU (Bethesda Units)/mL at screening
- Previous arterial thrombotic events (e.g. myocardial infarction and intracranial thrombosis) or previous deep venous thrombosis or pulmonary embolism (as defined by available medical records)
- Immune modulating or chemotherapeutic medication
- Any disease (liver, kidney, inflammatory and mental disorders included) or condition which, according to the Investigator's judgement, could imply a potential hazard to the patient, interfere with trial participation or trial outcome
- Unwillingness, language or other barriers precluding adequate understanding and/or cooperation
Trial locations and contacts
70 sites in 21 countries.
70 sites in 21 countries
United States19 sites
Novo Nordisk Investigational Site
Baltimore, Maryland, 21287
Novo Nordisk Investigational Site
Boise, Idaho, 83712
Novo Nordisk Investigational Site
Charleston, South Carolina, 29425
Novo Nordisk Investigational Site
Cincinnati, Ohio, 452289
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
Nashville, Tennessee, 37232-9830
Novo Nordisk Investigational Site
New Orleans, Louisiana, 70118-5720
Novo Nordisk Investigational Site
Newark, New Jersey, 07102
Novo Nordisk Investigational Site
Norfolk, Virginia, 23507
Novo Nordisk Investigational Site
Orlando, Florida, 32827
Novo Nordisk Investigational Site
Philadelphia, Pennsylvania, 19134
Novo Nordisk Investigational Site
Phoenix, Arizona, 85016-7710
Novo Nordisk Investigational Site
Portland, Oregon, 97239
Novo Nordisk Investigational Site
Sacramento, California, 95817
Novo Nordisk Investigational Site
Spokane, Washington, 99204
Novo Nordisk Investigational Site
Tampa, Florida, 33607
Novo Nordisk Investigational Site
Torrance, California, 90502-2004
Germany6 sites
Novo Nordisk Investigational Site
Berlin, 10249
Novo Nordisk Investigational Site
Bonn, 53127
Novo Nordisk Investigational Site
Frankfurt/M., 60590
Novo Nordisk Investigational Site
Hanover, 30625
Novo Nordisk Investigational Site
Homburg, 66421
Novo Nordisk Investigational Site
München, 80336
United Kingdom6 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
Sheffield, S11 8RN
France4 sites
Novo Nordisk Investigational Site
Bron, 69677
Novo Nordisk Investigational Site
Le Kremlin-Bicêtre, 94270
Novo Nordisk Investigational Site
Nantes, 44093
Novo Nordisk Investigational Site
Paris, 75015
Japan4 sites
Novo Nordisk Investigational Site
Kashihara-shi, Nara, 634 8522
Novo Nordisk Investigational Site
Shinjuku-ku, Tokyo, 160 0023
Novo Nordisk Investigational Site
Suginami-ku, Tokyo, 167 0035
Novo Nordisk Investigational Site
Tokyo, 108-8639
Australia3 sites
Novo Nordisk Investigational Site
Camperdown, New South Wales, 2050
Novo Nordisk Investigational Site
Parkville, Victoria, 3052
Novo Nordisk Investigational Site
South Brisbane, Queensland, 4101
Italy3 sites
Novo Nordisk Investigational Site
Milan, 20124
Novo Nordisk Investigational Site
Udine, 33100
Novo Nordisk Investigational Site
Vicenza, 36100
Switzerland3 sites
Novo Nordisk Investigational Site
Geneva, 1211
Novo Nordisk Investigational Site
Lausanne, 1011
Novo Nordisk Investigational Site
Zurich, 8091
Turkey (Türkiye)3 sites
Novo Nordisk Investigational Site
Adana, 01130
Novo Nordisk Investigational Site
Bornova-IZMIR, 35100
Novo Nordisk Investigational Site
Samsun, 55319
Croatia2 sites
Novo Nordisk Investigational Site
Split, 21 000
Novo Nordisk Investigational Site
Zagreb, 10 000
Denmark2 sites
Novo Nordisk Investigational Site
Århus N, 8200
Novo Nordisk Investigational Site
København Ø, 2100
Hungary2 sites
Novo Nordisk Investigational Site
Budapest, H-1134
Novo Nordisk Investigational Site
Debrecen, 4012
Malaysia2 sites
Novo Nordisk Investigational Site
Kuala Lumpur, 50400
Novo Nordisk Investigational Site
Selangor Darul Ehsan, 68000
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
Taiwan2 sites
Novo Nordisk Investigational Site
Changhua, 500
Novo Nordisk Investigational Site
Taipei, 100
About this study
Official title: Efficacy and Safety of NNC 0129-0000-1003 During Surgical Procedures in Patients With Haemophilia A
This trial is conducted globally. The aim of this trial is to evaluate the haemostatic effect of NNC 0129-0000-1003 during surgical procedures in subjects with haemophilia A.
- 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
Also called NNC 0129-0000-1003
Bleeding preventive treatment administered i.v. before, during and after surgery. Individually adjusted doses.
| Study group | Role | What they receive |
|---|---|---|
| Surgery | Experimental | Not described Receives: Drug: turoctocog alfa pegol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Haemostatic Effect During Surgery Evaluated by the Four-point Scale, Assessed by the Investigator/Surgeon at the Day of Surgery - Four-point Response Scale: Excellent, Good, Moderate or None
Haemostatic effect during surgery was evaluated on a four-point response scale as 'none', 'moderate', 'good' and 'excellent'. This was assessed after completion of surgery (defined as "last stitch").
Excellent: Better than expected/predicted in this type of procedure. Good: As expected in this type of procedure. Moderate: Less than optimal for the type of procedure but haemostatic response maintained without change of treatment regimen.
None: Bleeding due to inadequate therapeutic response with adequate dosing, change of regimen required.
Measured: Assessed by the Investigator/surgeon at the day of surgery
Secondary outcomes (11)
Average Consumption of N8-GP During Surgery
Average consumption of N8-GP, during surgery is presented. The time during surgery is defined from 'knife to skin' until 'last stitch'.
Measured: During surgery, defined as the time from "knife to skin" until "last stitch"
Haemostatic Effect of N8-GP During the Post-operative Period Days 1-6
Haemostatic effect during post-operative period days 1-6 as evaluated on a four-point response scale as 'none', 'moderate', 'good' and 'excellent'.
Excellent: Better than expected/predicted in this type of procedure. Good: As expected in this type of procedure. Moderate: Less than optimal for the type of procedure but haemostatic response maintained without change of treatment regimen.
None: Bleeding due to inadequate therapeutic response with adequate dosing, change of regimen required.
Measured: During the post-operative period, days 1-6
Haemostatic Effect of N8-GP During the Post-operative Period Days 7-14
Haemostatic effect during post-operative period days 1-6 and days 7-14 was evaluated on a four-point response scale as 'none', 'moderate', 'good' and 'excellent'.
Excellent: Better than expected/predicted in this type of procedure. Good: As expected in this type of procedure. Moderate: Less than optimal for the type of procedure but haemostatic response maintained without change of treatment regimen.
None: Bleeding due to inadequate therapeutic response with adequate dosing, change of regimen required.
Measured: During the post-operative period, days 7-14
Average Consumption of N8-GP During the Post-operative Period Days 1-6
Average consumption of N8-GP during post operative period days 1-6 is presented. Analysis population: Full analysis set included all subjects exposed to the trial drug (N8-GP) and completed surgery.
Measured: During the post-operative period, days 1-6
Incidence Rate of Inhibitors Against Factor VIII (FVIII) (≥0.6 BU/mL)
Incidence rate of inhibitors is the number of newly developed inhibitors per surgery. Development of FVIII inhibitors was measured by a validated Nijmegen modified Bethesda assay. A positive inhibitor test was defined as ≥0.6 bethesda unit. Number of participants with inhibitors at the end of trial is presented.
Measured: during the trial (2-5 weeks)
Estimated Blood Loss During Surgery
The mean estimated blood loss following surgery is presented. Estimated blood loss (mL) was evaluated post surgery.
Measured: During surgery
Number of Transfusions During the Post-operative Period Days 1-6
Number of blood product transfusions (transfusion of red blood cells) during the post-surgery period, Days 1-6 is presented.
Measured: Post-operative period, days 1-6
Length of Stay in the Hospital
Mean number of days stayed at the hospital during the trial.
Measured: During the trial (2-5 weeks)
Number of Days in Intensive Care
Mean number of days in the intensive care due to surgery during the trial is presented.
Measured: During the trial (2-5 weeks)
Adverse Events Reported During the Trial Period
Number of adverse event during the trial is presented. This includes events from the first trial related activity after the patient has signed the informed consent and until the end of trial (earliest at day 14).
Measured: During the trial (2-5 weeks)
Serious Adverse Events Reported During the Trial Period
Number of serious adverse event during the trial is presented. This includes events from the first trial related activity after the patient has signed the informed consent and until the end of trial (earliest at day 14).
Measured: During the trial (2-5 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Dec 2011 (estimated)
- Study start3 Aug 2012
- Primary completion10 Dec 2018
- Study completion10 Dec 2018
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.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Global Clinical Registry (GCR, 1452), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01489111
- Sponsor's protocol ID
- NN7088-3860
- Other IDs
- U1111-1119-7326, 2011-001144-30, 132215
- Acronym
- pathfinder™3
- Status last verified
- Jul 2020
- First submitted
- 23 Nov 2011
- Last updated
- 10 Aug 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Tosetto A, Neff A, Lentz SR, Santagostino E, Nemes L, Sathar J, Meijer K, Chowdary P, Shen C, Landorph A, Hampton K. Turoctocog alfa pegol provides effective management for major and minor surgical procedures in patients across all age groups with severe haemophilia A: Full data set from the pathfinder 3 and 5 phase III trials. Haemophilia. 2020 May;26(3):450-458. doi: 10.1111/hae.13980. Epub 2020 Apr 15.
PubMed 32293786
Study at a glance
From the registry record, last updated by the sponsor on 10 Aug 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of NNC 0129-0000-1003 During Surgical Procedures in Patients With Haemophilia A
- ClinicalTrials.gov ID
- NCT01489111
- Acronym / sponsor ID
- pathfinder™3 / NN7088-3860
- Conditions
- Congenital Bleeding Disorder, Haemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 3 Aug 2012 (actual)
- Primary completion
- 10 Dec 2018 (actual)
- Study completion
- 10 Dec 2018 (actual)
- First posted
- 9 Dec 2011 (estimated)
- Results
- Posted 21 Jan 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This trial is conducted globally. The aim of this trial is to evaluate the haemostatic effect of NNC 0129-0000-1003 during surgical procedures in subjects with haemophilia A.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Surgery | Experimental | Drug: turoctocog alfa pegol |
Who may qualify
- Ages
- 12 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Informed consent obtained before any trial-related activities. (Trial-related activities are any procedure that would not have been performed during normal management of the subject.)
- Ongoing participation in the pathfinder™2 (NN7088-3859) or the pathfinderTM 4 (NN7088-3861) trial and having received greater than or equal to 5 doses of N8-GP
- Undergoing major surgery requiring daily monitoring of FVIII:C (FVIII activity) and wound status for at least 3 days
- The patient and/or Legally Acceptable Representative (LAR) is capable of assessing a bleeding episode, keeping an eDiary, capable of home treatment of bleeding episodes and otherwise capable of following the trial procedures
Key exclusion criteria (8 listed)
- Known or suspected hypersensitivity to trial product including allergy to hamster protein or related products
- Previous withdrawal from the pathfinder™2 (NN7088-3859) or the pathfinderTM 4 (NN7088-3861) trial after administration of trial product, except interruption due to inclusion in this pathfinderTM 3 trial (NN7088-3860)
- The receipt of any investigational medicinal product (except N8-GP) within 30 days prior to enrolment into the trial. (For Brazil, only: Participation in a previous clinical trial within one year prior to screening for this trial (Visit 1), unless there is a direct benefit to the research subject, at the Investigator's discretion)
- FVIII inhibitors at least 0.6 BU (Bethesda Units)/mL at screening
- Previous arterial thrombotic events (e.g. myocardial infarction and intracranial thrombosis) or previous deep venous thrombosis or pulmonary embolism (as defined by available medical records)
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 pegolAlso: NNC 0129-0000-1003Bleeding preventive treatment administered i.v. before, during and after surgery. Individually adjusted doses. | Drug | Surgery |
Primary outcome
Haemostatic Effect During Surgery Evaluated by the Four-point Scale, Assessed by the Investigator/Surgeon at the Day of Surgery - Four-point Response Scale: Excellent, Good, Moderate or None
Time frame: Assessed by the Investigator/surgeon at the day of surgery
Haemostatic effect during surgery was evaluated on a four-point response scale as 'none', 'moderate', 'good' and 'excellent'. This was assessed after completion of surgery (defined as "last stitch"). Excellent: Better than expected/predicted in this type of procedure. Good: As expected in this type of procedure. Moderate: Less than optimal for the type of procedure but haemostatic response maintained without change of treatment regimen. None: Bleeding due to inadequate therapeutic response with adequate dosing, change of regimen required.
11 secondary outcomes
Average Consumption of N8-GP During Surgery
Time frame: During surgery, defined as the time from "knife to skin" until "last stitch"
Haemostatic Effect of N8-GP During the Post-operative Period Days 1-6
Time frame: During the post-operative period, days 1-6
Haemostatic Effect of N8-GP During the Post-operative Period Days 7-14
Time frame: During the post-operative period, days 7-14
Average Consumption of N8-GP During the Post-operative Period Days 1-6
Time frame: During the post-operative period, days 1-6
Incidence Rate of Inhibitors Against Factor VIII (FVIII) (≥0.6 BU/mL)
Time frame: during the trial (2-5 weeks)
Estimated Blood Loss During Surgery
Time frame: During surgery
Number of Transfusions During the Post-operative Period Days 1-6
Time frame: Post-operative period, days 1-6
Length of Stay in the Hospital
Time frame: During the trial (2-5 weeks)
Number of Days in Intensive Care
Time frame: During the trial (2-5 weeks)
Adverse Events Reported During the Trial Period
Time frame: During the trial (2-5 weeks)
Serious Adverse Events Reported During the Trial Period
Time frame: During the trial (2-5 weeks)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Aug 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 32293786Tosetto A, Neff A, Lentz SR, Santagostino E, Nemes L, Sathar J, Meijer K, Chowdary P, Shen C, Landorph A, Hampton K. Turoctocog alfa pegol provides effective management for major and minor surgical procedures in patients across all age groups with severe haemophilia A: Full data set from the pathfinder 3 and 5 phase III trials. Haemophilia. 2020 May;26(3):450-458. doi: 10.1111/hae.13980. Epub 2020 Apr 15.
- Clinical Trials at Novo NordiskLink provided by the sponsor.
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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.
Global Clinical Registry (GCR, 1452) study director
Novo Nordisk A/S
70 sites in 21 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 70 sites in 21 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 4 inclusion and 8 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 10 Aug 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 70 sites in United States, Germany, United Kingdom, France, Japan and 16 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 21 Jan 2020). 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?
Haemostatic Effect During Surgery Evaluated by the Four-point Scale, Assessed by the Investigator/Surgeon at the Day of Surgery - Four-point Response Scale: Excellent, Good, Moderate or None (time frame: Assessed by the Investigator/surgeon at the day of surgery). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01489111, which also lists 1 linked publication.
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 10 Aug 2020.