Congenital Bleeding DisorderPhase 3guardian™ 3NCT01138501
Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A
A Phase 3 study testing turoctocog alfa in people with Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 38 sites in 13 countries.
Trial summary
This trial is conducted in Asia, Europe, and North and South America. The aim of this clinical trial is to investigate the safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in male previously treated paediatric subjects with haemophilia A. Read more about this study
- Who is it for?
- Ages Up to 11 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
- 65 participants
- Where
- 38 sitesin 13 countries
- Study dates
- Jun 2010to Nov 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 Up to 11 years
- Men only
- No healthy volunteers
- Child
Who may be able to join
- Male patients with severe (baseline FVIII less than or equal to 1%) haemophilia A
- Age below 12 years and weight at least 11 kg
Who cannot join
- Surgery planned to occur during trial participation (exceptions are port placement, dental extractions, and minor, uncomplicated emergent procedures)
- Congenital or acquired coagulation disorders other than haemophilia A
- Any history of FVIII inhibitors (greater than or equal to 0.6 BU/mL)
Trial locations and contacts
38 sites in 13 countries.
38 sites in 13 countries
United States18 sites
Novo Nordisk Investigational Site
Augusta, Georgia, 30912
Novo Nordisk Investigational Site
Boston, Massachusetts, 02115
Novo Nordisk Investigational Site
Brooklyn, New York, 11201-5425
Novo Nordisk Investigational Site
Charleston, South Carolina, 29425
Novo Nordisk Investigational Site
Dayton, Ohio, 45404
Novo Nordisk Investigational Site
Detroit, Michigan, 48201
Novo Nordisk Investigational Site
East Lansing, Michigan, 48823
Novo Nordisk Investigational Site
Fort Worth, Texas, 76104
Novo Nordisk Investigational Site
Houston, Texas, 77030
Novo Nordisk Investigational Site
Iowa City, Iowa, 52242
Novo Nordisk Investigational Site
Kansas City, Missouri, 64108-4619
Novo Nordisk Investigational Site
Omaha, Nebraska, 68198-2168
Novo Nordisk Investigational Site
Orange, California, 92868
Novo Nordisk Investigational Site
Phoenix, Arizona, 85016-7710
Novo Nordisk Investigational Site
Portland, Oregon, 97239-3098
Novo Nordisk Investigational Site
Tampa, Florida, 33607
Novo Nordisk Investigational Site
Torrance, California, 90502-2004
Novo Nordisk Investigational Site
Valhalla, New York, 10595
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, Rio de Janeiro, 20211-030
Novo Nordisk Investigational Site
São Paulo, São Paulo, 05403-000
Turkey (Türkiye)4 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
Poland2 sites
Novo Nordisk Investigational Site
Warsaw, 00-576
Novo Nordisk Investigational Site
Wroclaw, 50-345
Russia2 sites
Novo Nordisk Investigational Site
Moscow, 105077
Novo Nordisk Investigational Site
Saint Petersburg, 191119
About this study
Official title: A Multi-Centre, Open-Label, Non-Controlled Trial on Safety and Efficacy of N8 in Previously Treated Paediatric Patients With Haemophilia A
This trial is conducted in Asia, Europe, and North and South America. The aim of this clinical trial is to investigate the safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in male previously treated paediatric subjects with haemophilia A.
- 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 be treated 3 times per week or every second day with intravenous injections of turoctocog alfa. The dose range for preventive treatment is 25-60 IU/kg body weight depending on treatment regimen.
turoctocog alfaDrug
Subjects will undergo half-life evaluation of their current factor VIII product and pharmacokinetic session with turoctocog alfa before entering preventive treatment (subjects with available evaluation of terminal half-life within the last year are to be excluded from receiving factor VIII). Intravenous injections. The dose range for preventive treatment is 25-60 IU/kg body weight depending on treatment regimen.
| 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 138 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 AE: AE that at any dose results in any of the following: death, a life-threatening experience, in-subject hospitalization/prolongation of existing hospitalisation, persistent/significant disability/incapacity/congenital anomaly/birth defect.
Measured: The adverse events were collected throughout the trial, corresponding to an average of 138 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 posted7 Jun 2010 (estimated)
- Study startJun 2010
- Primary completionNov 2011
- Study completionNov 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 (3)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
- 65 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
- NCT01138501
- Sponsor's protocol ID
- NN7008-3545
- Other IDs
- U1111-1113-7182, 2009-016383-36, 2009
- Acronym
- guardian™ 3
- Status last verified
- Feb 2017
- First submitted
- 28 May 2010
- Last updated
- 17 Mar 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Kulkarni R, Karim FA, Glamocanin S, Janic D, Vdovin V, Ozelo M, Rageliene L, Carboni E, Laguna P, Dobaczewski G, Seremetis S, Lindblom A, Santagostino E. Results from a large multinational clinical trial (guardian3) using prophylactic treatment with turoctocog alfa in paediatric patients with severe haemophilia A: safety, efficacy and pharmacokinetics. Haemophilia. 2013 Sep;19(5):698-705. doi: 10.1111/hae.12165. Epub 2013 May 8.
PubMed 23651313Santagostino 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 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 Previously Treated Paediatric Patients With Haemophilia A
- ClinicalTrials.gov ID
- NCT01138501
- Acronym / sponsor ID
- guardian™ 3 / NN7008-3545
- Conditions
- Congenital Bleeding Disorder, Haemophilia A
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 65 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- Jun 2010
- Primary completion
- Nov 2011 (actual)
- Study completion
- Nov 2011 (actual)
- First posted
- 7 Jun 2010 (estimated)
- Results
- Posted 11 Jun 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 aim of this clinical trial is to investigate the safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in male previously treated paediatric subjects with haemophilia A.
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
- Up to 11 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (2 listed)
- Male patients with severe (baseline FVIII less than or equal to 1%) haemophilia A
- Age below 12 years and weight at least 11 kg
Key exclusion criteria (3 listed)
- Surgery planned to occur during trial participation (exceptions are port placement, dental extractions, and minor, uncomplicated emergent procedures)
- Congenital or acquired coagulation disorders other than haemophilia A
- Any history of FVIII inhibitors (greater than or equal to 0.6 BU/mL)
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 be treated 3 times per week or every second day with intravenous injections of turoctocog alfa. The dose range for preventive treatment is 25-60 IU/kg body weight depending on treatment regimen. | Drug | rFVIII |
| turoctocog alfaSubjects will undergo half-life evaluation of their current factor VIII product and pharmacokinetic session with turoctocog alfa before entering preventive treatment (subjects with available evaluation of terminal half-life within the last year are to be excluded from receiving factor VIII). Intravenous injections. The dose range for preventive treatment is 25-60 IU/kg body weight depending on treatment regimen. | 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 138 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 138 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 23651313Kulkarni R, Karim FA, Glamocanin S, Janic D, Vdovin V, Ozelo M, Rageliene L, Carboni E, Laguna P, Dobaczewski G, Seremetis S, Lindblom A, Santagostino E. Results from a large multinational clinical trial (guardian3) using prophylactic treatment with turoctocog alfa in paediatric patients with severe haemophilia A: safety, efficacy and pharmacokinetics. Haemophilia. 2013 Sep;19(5):698-705. doi: 10.1111/hae.12165. Epub 2013 May 8.
- 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 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.
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
38 sites in 13 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 38 sites in 13 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 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 38 sites in United States, Brazil, Turkey (Türkiye), Poland, Russia and 8 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 11 Jun 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 138 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 NCT01138501, which also lists 3 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.