Congenital Bleeding DisorderPhase 3NCT00984126
Safety and Efficacy of Turoctocog Alfa (N8) in Prevention and On-demand Treatment of Bleeding Episodes in Subjects With Haemophilia A: An Extension to Trials NN7008-3543, NN7008-3545, NN7008-3600, NN7008-3893 and NN7008-4015
A Phase 3 study testing turoctocog alfa in people with Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 57 sites in 20 countries.
Trial summary
This trial is conducted in Asia, Europe, Japan, Oceania, North America and South America. The aim of the trial is to investigate the safety and efficacy of turoctocog alfa (N8) in Haemophilia A patients. Read more about this study
- Who is it for?
- Ages 6 months to 70 years, men only
- Conditions the study is for
- Congenital Bleeding Disorder, Haemophilia A
- What is being tested?
- turoctocog alfa
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 214 participants
- Where
- 57 sitesin 20 countries
- Study dates
- 26 Oct 2009to 29 Jun 2016
- 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 6 months to 70 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Informed Consent obtained before any trial-related activities
- Completion of trial NN7008-3543 or paediatric trial NN7008-3545 or Japanese trial NN7008-3600 or pharmacokinetic trial NN7008-3893 or NN7008-4015
Who cannot join
- Previous participation in the current trial (defined as withdrawal) or withdrawn subjects from NN7008-3522, NN7008-3543, NN7008-3545, NN7008-3600, NN7008-3893 or NN7008-4015 after administration of trial product
Trial locations and contacts
57 sites in 20 countries.
57 sites in 20 countries
United States13 sites
Novo Nordisk Investigational Site
Atlanta, Georgia, 30322
Novo Nordisk Investigational Site
Boston, Massachusetts, 02115
Novo Nordisk Investigational Site
Cincinnati, Ohio, 45229
Novo Nordisk Investigational Site
Dayton, Ohio, 45404
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
Phoenix, Arizona, 85016-7710
Novo Nordisk Investigational Site
Portland, Oregon, 97239
Novo Nordisk Investigational Site
Providence, Rhode Island, 02903
Novo Nordisk Investigational Site
Spokane, Washington, 99204
Novo Nordisk Investigational Site
Tampa, Florida, 33607
Japan5 sites
Novo Nordisk Investigational Site
Kashihara-shi, Nara, 634 8522
Novo Nordisk Investigational Site
Nagoya-shi, Aichi, 466 8560
Novo Nordisk Investigational Site
Shimotsuke-shi, Tochigi, 329 0498
Novo Nordisk Investigational Site
Shinjuku-ku, Tokyo, 160 0023
Novo Nordisk Investigational Site
Shizuoka-shi, Shizuoka, 4208660
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, 55319
Brazil4 sites
Novo Nordisk Investigational Site
Campinas, São Paulo, 13081970
Novo Nordisk Investigational Site
Curitiba, Paraná, 80250-060
Novo Nordisk Investigational Site
Rio de Janeiro, 20211-030
Novo Nordisk Investigational Site
São Paulo, São Paulo, 05403-000
Germany4 sites
Novo Nordisk Investigational Site
Berlin, 10249
Novo Nordisk Investigational Site
Bonn, 53127
Novo Nordisk Investigational Site
Giessen, 35392
Novo Nordisk Investigational Site
Hanover, 30625
Serbia4 sites
Novo Nordisk Investigational Site
Belgrade, 11000
Novo Nordisk Investigational Site
Belgrade, 11070
Novo Nordisk Investigational Site
Niš, 18000
Novo Nordisk Investigational Site
Novi Sad, 21000
United Kingdom3 sites
Novo Nordisk Investigational Site
London, NW3 2QG
Novo Nordisk Investigational Site
London, SE1 7EH
Novo Nordisk Investigational Site
Manchester, M13 9WL
Croatia2 sites
Novo Nordisk Investigational Site
Split, 21 000
Novo Nordisk Investigational Site
Zagreb, 10 000
Israel2 sites
Novo Nordisk Investigational Site
Tel Aviv
Novo Nordisk Investigational Site
Tel HaShomer, 52621
Italy2 sites
Novo Nordisk Investigational Site
Florence, 50134
Novo Nordisk Investigational Site
Milan, 20124
Poland2 sites
Novo Nordisk Investigational Site
Warsaw, 00-576
Novo Nordisk Investigational Site
Wroclaw, 50-556
Russia2 sites
Novo Nordisk Investigational Site
Moscow, 119049
Novo Nordisk Investigational Site
Saint Petersburg, 191119
Spain2 sites
Novo Nordisk Investigational Site
Madrid, 28046
Novo Nordisk Investigational Site
Valencia, 46026
About this study
Official title: Safety and Efficacy of N8 in Prevention and On-demand Treatment of Bleeding Episodes in Subjects With Haemophilia A
This trial is conducted in Asia, Europe, Japan, Oceania, North America and South America.
The aim of the trial is to investigate the safety and efficacy of turoctocog alfa (N8) in Haemophilia A patients.
The trial is an extension to trials NN7008-3543 (start: March 2009, stop: September 2011) and NN7008-3545 (start: May 2010, stop: November 2011) and the pharmacokinetic trials NN7008-3600 (start: November 2010, stop: October 2011), NN7008-3893 (start: June 2011, stop: September 2011) and NN7008-4015 (start: August 2012, stop: March 2013).
- Conditions
- Congenital Bleeding Disorder, Haemophilia A
- Being tested
- turoctocog alfa
- 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 alfaDrug
The preventative treatment is administered intravenously (i.v.) at specific intervals either every second day or three times a week. Bleeding treatment will be administered if a bleed should occur.
turoctocog alfaDrug
Treatment is administered intravenously (i.v.) during bleeds and occasionally as a preventative treatment (e.g. before physical activity)
| Study group | Role | What they receive |
|---|---|---|
| Turoctocog alfa | Experimental | Not described Receives: Drug: turoctocog alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of Development of FVIII Inhibitors (Greater Than or Equal to 0.6 Bethesda Units (BU)/mL)
The frequency of inhibitors was calculated as number of patients with inhibitors during the trial divided by number of patients in the trial. This endpoint was measured during the trial.
Measured: After 90 months
Secondary outcomes (3)
Frequency of Adverse Events and Serious Adverse Events
The number of adverse events and serious adverse events reported during the main trial and the on-demand sub-trial (during 90 months).
Measured: After 90 months
Annualised Bleeding Rate Reported During the Prevention Period (Only Applicable for Subjects in the Preventive Regimen)
The number of bleeding episodes per year reported during the prevention period (during 90 months).
Measured: After 90 months
Haemostatic Response to Turoctocog Alfa (None, Moderate, Good or Excellent) in Treatment of Bleeds.
Haemostatic response to turoctocog alfa (none, moderate, good or excellent) in treatment of bleeds using a four-point response scale: none, moderate, good or excellent. The evaluation was done by patient, caregiver and/or investigator based on experience as follows: 1. Excellent: Abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single infusion 2. Good: Definite pain relief and/or improvement in signs of bleeding within approximately 8 hours after an infusion, but possibly requiring more than 1 infusion for complete resolution. 3. Moderate: Probable or slight beneficial effect within approximately 8 hours after the first infusion; usually requiring more than 1 infusion. 4. None: No improvement, or worsening of symptoms. This endpoint is measured during the preventive and on-demand sub-trial (during 90 months).
Measured: After 90 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Sep 2009 (estimated)
- Study start26 Oct 2009
- Primary completion28 Jun 2016
- Study completion29 Jun 2016
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 (4)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
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 214 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
- NCT00984126
- Sponsor's protocol ID
- NN7008-3568
- Other IDs
- 2008-005945-46, U1111-1111-9377, JapicCTI-101357, 2008
- Status last verified
- Jun 2017
- First submitted
- 21 Sep 2009
- Last updated
- 27 Jul 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Ozelo M, Misgav M, Abdul-Karim F, Lentz S, Martin-Salces M, Matytsina I, Saugstrup T, Santagostino E. Stabilization of turoctocog alfa dose administered in a preventive regimen: 3-year interim results of the guardianTM-2 extension trial. Haemophilia - Special Issue: Abstracts of the WFH 2014 World Congress, May 11-15, Melbourne, Australia; 20 (3): 1-200
Recht M, Lentz S, Zupancic-Salek S, Matytsina I, Landorph A, Saugstrup T. Factor VIII Dosing and Preventive Efficacy in Obese Patients with Hemophilia (BMI =30 kg/m2) - a Post-Hoc Sub-Analysis of the guardian™ Trials. American Society of Hematology - 56th Annual Meeting (ASH); Country: US City: San Francisco, CA
Ozelo M, Misgav M, Abdul Karim F, Lentz SR, Martin-Salces M, Matytsina I, Saugstrup T, Santagostino E. Long-term patterns of safety and efficacy of bleeding prophylaxis with turoctocog alfa (NovoEight((R)) ) in previously treated patients with severe haemophilia A: interim results of the guardian() 2 extension trial. Haemophilia. 2015 Sep;21(5):e436-9. doi: 10.1111/hae.12737. Epub 2015 Jun 8. No abstract available.
PubMed 26058730Lentz SR, Cerqueira M, Janic D, Kempton C, Matytsina I, Misgav M, Oldenburg J, Ozelo M, Recht M, Rosholm A, Savic A, Suzuki T, Tiede A, Santagostino E. Interim results from a large multinational extension trial (guardian() 2) using turoctocog alfa for prophylaxis and treatment of bleeding in patients with severe haemophilia A. Haemophilia. 2016 Sep;22(5):e445-9. doi: 10.1111/hae.12990. Epub 2016 Jun 13. No abstract available.
PubMed 27291066
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of N8 in Prevention and On-demand Treatment of Bleeding Episodes in Subjects With Haemophilia A
- ClinicalTrials.gov ID
- NCT00984126
- Acronym / sponsor ID
- NN7008-3568
- Conditions
- Congenital Bleeding Disorder, Haemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 214 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 26 Oct 2009 (actual)
- Primary completion
- 28 Jun 2016 (actual)
- Study completion
- 29 Jun 2016 (actual)
- First posted
- 25 Sep 2009 (estimated)
- Results
- Posted 27 Jul 2017
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 in Asia, Europe, Japan, Oceania, North America and South America.
The aim of the trial is to investigate the safety and efficacy of turoctocog alfa (N8) in Haemophilia A patients.
The trial is an extension to trials NN7008-3543 (start: March 2009, stop: September 2011) and NN7008-3545 (start: May 2010, stop: November 2011) and the pharmacokinetic trials NN7008-3600 (start: November 2010, stop: October 2011), NN7008-3893 (start: June 2011, stop: September 2011) and NN7008-4015 (start: August 2012, stop: March 2013).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Turoctocog alfa | Experimental | Drug: turoctocog alfa |
Who may qualify
- Ages
- 6 months to 70 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Informed Consent obtained before any trial-related activities
- Completion of trial NN7008-3543 or paediatric trial NN7008-3545 or Japanese trial NN7008-3600 or pharmacokinetic trial NN7008-3893 or NN7008-4015
Key exclusion criteria (1 listed)
- Previous participation in the current trial (defined as withdrawal) or withdrawn subjects from NN7008-3522, NN7008-3543, NN7008-3545, NN7008-3600, NN7008-3893 or NN7008-4015 after administration of trial 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 alfaThe preventative treatment is administered intravenously (i.v.) at specific intervals either every second day or three times a week. Bleeding treatment will be administered if a bleed should occur. | Drug | Turoctocog alfa |
| turoctocog alfaTreatment is administered intravenously (i.v.) during bleeds and occasionally as a preventative treatment (e.g. before physical activity) | Drug | Turoctocog alfa |
Primary outcome
Frequency of Development of FVIII Inhibitors (Greater Than or Equal to 0.6 Bethesda Units (BU)/mL)
Time frame: After 90 months
The frequency of inhibitors was calculated as number of patients with inhibitors during the trial divided by number of patients in the trial. This endpoint was measured during the trial.
3 secondary outcomes
Frequency of Adverse Events and Serious Adverse Events
Time frame: After 90 months
Annualised Bleeding Rate Reported During the Prevention Period (Only Applicable for Subjects in the Preventive Regimen)
Time frame: After 90 months
Haemostatic Response to Turoctocog Alfa (None, Moderate, Good or Excellent) in Treatment of Bleeds.
Time frame: After 90 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publicationOzelo M, Misgav M, Abdul-Karim F, Lentz S, Martin-Salces M, Matytsina I, Saugstrup T, Santagostino E. Stabilization of turoctocog alfa dose administered in a preventive regimen: 3-year interim results of the guardianTM-2 extension trial. Haemophilia - Special Issue: Abstracts of the WFH 2014 World Congress, May 11-15, Melbourne, Australia; 20 (3): 1-200
- Results publicationRecht M, Lentz S, Zupancic-Salek S, Matytsina I, Landorph A, Saugstrup T. Factor VIII Dosing and Preventive Efficacy in Obese Patients with Hemophilia (BMI =30 kg/m2) - a Post-Hoc Sub-Analysis of the guardian™ Trials. American Society of Hematology - 56th Annual Meeting (ASH); Country: US City: San Francisco, CA
- Results publication: PubMed 26058730Ozelo M, Misgav M, Abdul Karim F, Lentz SR, Martin-Salces M, Matytsina I, Saugstrup T, Santagostino E. Long-term patterns of safety and efficacy of bleeding prophylaxis with turoctocog alfa (NovoEight((R)) ) in previously treated patients with severe haemophilia A: interim results of the guardian() 2 extension trial. Haemophilia. 2015 Sep;21(5):e436-9. doi: 10.1111/hae.12737. Epub 2015 Jun 8. No abstract available.
- Results publication: PubMed 27291066Lentz SR, Cerqueira M, Janic D, Kempton C, Matytsina I, Misgav M, Oldenburg J, Ozelo M, Recht M, Rosholm A, Savic A, Suzuki T, Tiede A, Santagostino E. Interim results from a large multinational extension trial (guardian() 2) using turoctocog alfa for prophylaxis and treatment of bleeding in patients with severe haemophilia A. Haemophilia. 2016 Sep;22(5):e445-9. doi: 10.1111/hae.12990. Epub 2016 Jun 13. No abstract available.
- Clinical Trials at Novo NordiskLink provided by the sponsor.
Related trials for Congenital Bleeding Disorder
RecruitingObservational study
A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma
Thrombotic Thrombocytopenic Purpura (TTP)
Not yet recruitingPhase 1
Topical TOR-582 Treatment of Epistaxis in HHT
Hereditary Hemorrhagic Telangiectasia (HHT), Epistaxis
RecruitingPhase 1/2
Harmony-HHT: ATV-1601 in Participants With Hereditary Hemorrhagic Telangiectasia (HHT)
Hereditary Hemorrhagic Telangiectasia (HHT), Osler Weber Rendu Disease
RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Recruiting
Optimization of Saturation Targets And Resuscitation Trial (OptiSTART)
Premature Infants, Bronchopulmonary Dysplasia
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
57 sites in 20 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing turoctocog alfa in people with Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 57 sites in 20 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 1 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 27 Jul 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 57 sites in United States, Japan, Turkey (Türkiye), Brazil, Germany and 15 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 27 Jul 2017). 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?
Frequency of Development of FVIII Inhibitors (Greater Than or Equal to 0.6 Bethesda Units (BU)/mL) (time frame: After 90 months). 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 NCT00984126, which also lists 4 linked publications.
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 27 Jul 2017.