Congenital Bleeding DisorderPhase 3guardian™1NCT00840086
Safety and Efficacy of Turoctocog Alfa in Haemophilia A Subjects
A Phase 3 study testing turoctocog alfa in people with Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 63 sites in 18 countries.
Trial summary
This trial is conducted in Asia, Europe, and North and South America. The trial consists of a main trial and a sub-trial. The main trial investigates safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in haemophilia A subjects, while the sub-trial investigates safety and efficacy of… Read more about this study
- Who is it for?
- Ages 12 to 65 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
- 150 participants
- Where
- 63 sitesin 18 countries
- Study dates
- Apr 2009to Sep 2011
- 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 to 65 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male subjects with the diagnosis of severe (FVIII less than or equal to 1%) haemophilia A from age 12 (except for Israel where the age limit will be 18 for the first 10 subjects recruited in the trial) to 56 years having a weight of 10 to 120 kg
- Documented history of at least 150 exposure days to any other FVIII products (prevention or treatment of bleeds)
- No history of FVIII inhibitors greater than or equal to 0.6 BU/mL. The inhibitor should be measured regularly for at least the last 8 years or since the first treatment of haemophilia A
- No detectable inhibitors to FVIII (greater than or equal to 0.6 BU/mL) (as assessed by a Central Laboratory at the time of screening)
Who cannot join
- Congenital or acquired coagulation disorders other than haemophilia A
- Creatinine levels 50% above normal level (as defined by central laboratory range)
- Known or suspected allergy to trial product (N8) or related products
Trial locations and contacts
63 sites in 18 countries.
63 sites in 18 countries
United States18 sites
Novo Nordisk Investigational Site
Atlanta, Georgia, 30322
Novo Nordisk Investigational Site
Augusta, Georgia, 30912
Novo Nordisk Investigational Site
Cincinnati, Ohio, 45229
Novo Nordisk Investigational Site
Dayton, Ohio, 45404
Novo Nordisk Investigational Site
Fort Worth, Texas, 76104
Novo Nordisk Investigational Site
Indianapolis, Indiana, 46260
Novo Nordisk Investigational Site
Iowa City, Iowa, 52242
Novo Nordisk Investigational Site
Little Rock, Arkansas, 72202
Novo Nordisk Investigational Site
Long Beach, California, 90806
Novo Nordisk Investigational Site
Memphis, Tennessee, 38163
Novo Nordisk Investigational Site
Nashville, Tennessee, 37232-9830
Novo Nordisk Investigational Site
Newark, New Jersey, 07102
Novo Nordisk Investigational Site
Omaha, Nebraska, 68198-2168
Novo Nordisk Investigational Site
Philadelphia, Pennsylvania, 19134-1095
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
Japan9 sites
Novo Nordisk Investigational Site
Hiroshima-shi, Hiroshima, 734 8551
Novo Nordisk Investigational Site
Itabashi-ku, Tokyo, 173 8606
Novo Nordisk Investigational Site
Kashihara-shi, Nara, 634 8522
Novo Nordisk Investigational Site
Maebashi-shi, Gunma, 371-8511
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
Novo Nordisk Investigational Site
Suginami-ku, Tokyo, 1670035
Serbia and Montenegro5 sites
Novo Nordisk Investigational Site
Belgrade, 11000
Novo Nordisk Investigational Site
Belgrade, 11070
Novo Nordisk Investigational Site
Nis, 18000
Novo Nordisk Investigational Site
Novi Sad, 21 000
Novo Nordisk Investigational Site
Novi Sad, 21000
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
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
Hungary2 sites
Novo Nordisk Investigational Site
Budapest, H-1134
Novo Nordisk Investigational Site
Debrecen, 4012
Italy2 sites
Novo Nordisk Investigational Site
Florence, 50134
Novo Nordisk Investigational Site
Milan, 20124
Spain2 sites
Novo Nordisk Investigational Site
Madrid, 28046
Novo Nordisk Investigational Site
Valencia, 46026
Brazil1 site
Novo Nordisk Investigational Site
Rio de Janeiro, Rio de Janeiro, 20211-030
About this study
Official title: A Multi-centre, Open-label, Non-controlled Trial on Safety and Efficacy of N8 in Prevention and Treatment of Bleeds in Previously Treated Subjects With Haemophilia A. Sub-trial: Safety and Efficacy of N8 in Prevention and Treatment of Bleeding During Surgical Procedures in Subjects With Haemophilia A
This trial is conducted in Asia, Europe, and North and South America. The trial consists of a main trial and a sub-trial. The main trial investigates safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in haemophilia A subjects, while the sub-trial investigates safety and efficacy of turoctocog alfa in prevention and treatment of bleeding episodes during surgical procedures.
- 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
Subjects will receive bleeding preventive treatment (home treatment with self-injection i.v.) with turoctocog alfa at a dose of 20-40 IU/kg body weight every second day or 20-50 IU/kg body weight three times per week at the investigator's discretion.
| Study group | Role | What they receive |
|---|---|---|
| rFVIII | Experimental | Not described Receives: Drug: turoctocog alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Incidence Rate of FVIII Inhibitors (Greater Than or Equal to 0.6 Bethesda Units (BU))
The incidence rate of FVIII inhibitors was calculated by including all patients with inhibitors in the nominator and including all patients with a minimum 50 exposure plus any patients with less than 50 exposures but with inhibitors in denominator.
Measured: The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject.
Secondary outcomes (1)
Frequency of Adverse Events (AEs)
Adverse event was defined as events occurring after administration of trial product. Severe AEs: considerable interference with subject's daily activities, unacceptable. Moderate AEs: Marked symptoms, moderate interference with the patient's daily activities. Mild AEs: No or transient symptoms, no interference with the patient's daily activities. Serious AEs: AE that at any dose results in any of the following: death, a life-threatening experience, in-subject hospitalization/prolongation of existing hospitalization, persistent/significant disability/incapacity/congenital anomaly/birth defect.
Measured: The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Feb 2009 (estimated)
- Study startApr 2009
- Primary completionSep 2011
- Study completionSep 2011
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
- Single group
- Masking
- None (open label)
- Enrolment
- 150 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
- NCT00840086
- Sponsor's protocol ID
- NN7008-3543
- Other IDs
- 2008-003960-20, 101151, 2008
- Acronym
- guardian™1
- Status last verified
- Feb 2017
- First submitted
- 6 Feb 2009
- Last updated
- 17 Mar 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Lentz SR, Misgav M, Ozelo M, Salek SZ, Veljkovic D, Recht M, Cerqueira M, Tiede A, Brand B, Mancuso ME, Seremetis S, Lindblom A, Martinowitz U. Results from a large multinational clinical trial (guardian1) using prophylactic treatment with turoctocog alfa in adolescent and adult patients with severe haemophilia A: safety and efficacy. Haemophilia. 2013 Sep;19(5):691-7. doi: 10.1111/hae.12159. Epub 2013 May 7.
PubMed 23647704Santagostino E, Lentz SR, Misgav M, Brand B, Chowdary P, Savic A, Kilinc Y, Amit Y, Amendola A, Solimeno LP, Saugstrup T, Matytsina I. Safety and efficacy of turoctocog alfa (NovoEight(R)) during surgery in patients with haemophilia A: results from the multinational guardian clinical trials. Haemophilia. 2015 Jan;21(1):34-40. doi: 10.1111/hae.12518. Epub 2014 Oct 2.
PubMed 25273984Ozelo M, Chowdary P, Regnault A, Busk AK. Impact of severe haemophilia A on patients' health status: results from the guardian() 1 clinical trial of turoctocog alfa (NovoEight((R)) ). Haemophilia. 2015 Jul;21(4):451-7. doi: 10.1111/hae.12617. Epub 2015 Feb 12.
PubMed 25677761Ozelo MC. Updates from guardian: a comprehensive registration programme. Eur J Haematol. 2015 Dec;95 Suppl 81:22-9. doi: 10.1111/ejh.12648.
PubMed 26679394
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-centre, Open-label, Non-controlled Trial on Safety and Efficacy of N8 in Prevention and Treatment of Bleeds in Previously Treated Subjects With Haemophilia A. Sub-trial: Safety and Efficacy of N8 in Prevention and Treatment of Bleeding During Surgical Procedures in Subjects With Haemophilia A
- ClinicalTrials.gov ID
- NCT00840086
- Acronym / sponsor ID
- guardian™1 / NN7008-3543
- Conditions
- Congenital Bleeding Disorder, Haemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 150 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- Apr 2009
- Primary completion
- Sep 2011 (actual)
- Study completion
- Sep 2011 (actual)
- First posted
- 10 Feb 2009 (estimated)
- Results
- Posted 4 Sep 2014
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, and North and South America. The trial consists of a main trial and a sub-trial. The main trial investigates safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in haemophilia A subjects, while the sub-trial investigates safety and efficacy of turoctocog alfa in prevention and treatment of bleeding episodes during surgical procedures.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| rFVIII | Experimental | Drug: turoctocog alfa |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Male subjects with the diagnosis of severe (FVIII less than or equal to 1%) haemophilia A from age 12 (except for Israel where the age limit will be 18 for the first 10 subjects recruited in the trial) to 56 years having a weight of 10 to 120 kg
- Documented history of at least 150 exposure days to any other FVIII products (prevention or treatment of bleeds)
- No history of FVIII inhibitors greater than or equal to 0.6 BU/mL. The inhibitor should be measured regularly for at least the last 8 years or since the first treatment of haemophilia A
- No detectable inhibitors to FVIII (greater than or equal to 0.6 BU/mL) (as assessed by a Central Laboratory at the time of screening)
Key exclusion criteria (3 listed)
- Congenital or acquired coagulation disorders other than haemophilia A
- Creatinine levels 50% above normal level (as defined by central laboratory range)
- Known or suspected allergy to trial product (N8) or related products
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 alfaSubjects will receive bleeding preventive treatment (home treatment with self-injection i.v.) with turoctocog alfa at a dose of 20-40 IU/kg body weight every second day or 20-50 IU/kg body weight three times per week at the investigator's discretion. | Drug | rFVIII |
Primary outcome
The Incidence Rate of FVIII Inhibitors (Greater Than or Equal to 0.6 Bethesda Units (BU))
Time frame: The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject.
The incidence rate of FVIII inhibitors was calculated by including all patients with inhibitors in the nominator and including all patients with a minimum 50 exposure plus any patients with less than 50 exposures but with inhibitors in denominator.
1 secondary outcome
Frequency of Adverse Events (AEs)
Time frame: The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Mar 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 23647704Lentz SR, Misgav M, Ozelo M, Salek SZ, Veljkovic D, Recht M, Cerqueira M, Tiede A, Brand B, Mancuso ME, Seremetis S, Lindblom A, Martinowitz U. Results from a large multinational clinical trial (guardian1) using prophylactic treatment with turoctocog alfa in adolescent and adult patients with severe haemophilia A: safety and efficacy. Haemophilia. 2013 Sep;19(5):691-7. doi: 10.1111/hae.12159. Epub 2013 May 7.
- Results publication: PubMed 25273984Santagostino E, Lentz SR, Misgav M, Brand B, Chowdary P, Savic A, Kilinc Y, Amit Y, Amendola A, Solimeno LP, Saugstrup T, Matytsina I. Safety and efficacy of turoctocog alfa (NovoEight(R)) during surgery in patients with haemophilia A: results from the multinational guardian clinical trials. Haemophilia. 2015 Jan;21(1):34-40. doi: 10.1111/hae.12518. Epub 2014 Oct 2.
- Results publication: PubMed 25677761Ozelo M, Chowdary P, Regnault A, Busk AK. Impact of severe haemophilia A on patients' health status: results from the guardian() 1 clinical trial of turoctocog alfa (NovoEight((R)) ). Haemophilia. 2015 Jul;21(4):451-7. doi: 10.1111/hae.12617. Epub 2015 Feb 12.
- Results publication: PubMed 26679394Ozelo MC. Updates from guardian: a comprehensive registration programme. Eur J Haematol. 2015 Dec;95 Suppl 81:22-9. doi: 10.1111/ejh.12648.
- 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
63 sites in 18 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 63 sites in 18 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 3 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 17 Mar 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 63 sites in United States, Japan, Serbia and Montenegro, Turkey (Türkiye), Germany and 13 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 4 Sep 2014). 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?
The Incidence Rate of FVIII Inhibitors (Greater Than or Equal to 0.6 Bethesda Units (BU)) (time frame: The adverse events were collected throughout the trial, corresponding to an average of 188 days per subject.). 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 NCT00840086, 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 17 Mar 2017.