Congenital Bleeding Disorderguardian™ 5NCT02035384
Safety and Efficacy of Turoctocog Alfa During Long-Term Treatment of Severe and Moderately Severe Haemophilia A
An observational study of Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 41 sites in 15 countries.
Trial summary
This study is conducted in Europe, and North and South America. The aim of this study is to provide additional documentation of the immunogenicity, and obtain additional clinical data, of turoctocog alfa in the setting of normal clinical practise in patients previously treated with a factor VIII agent (FVIII). 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
- How many people
- 69 participants
- Where
- 41 sitesin 15 countries
- Study dates
- 5 Jun 2014to 15 Jan 2020
- Phase
- Observational
- Are there results?
- None posted in the registry
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
Study population: Previously FVIII treated (more than 150 exposure days) patients with severe and moderately severe haemophilia A with FVIII below or equal to 2%.
Who may be able to join
- Informed consent obtained before any study-related activities. Study-related activities are any procedure related to recording of data according to the protocol
- Previously FVIII treated (150 exposure days at the time of first dosing with turoctocog alfa) male patients with the diagnosis of severe and moderately severe haemophilia A (FVIII below or equal to 2%)
- The decision to initiate treatment with commercially available turoctocog alfa has been made by the patient/parent and the patient's treating physician before and independently from the decision to include the patient in this study
- A negative FVIII inhibitor test obtained not more than four weeks prior to first dosing with turoctocog alfa
Who cannot join
- Contraindications for use according to the approved product information text (US Package insert (PI), European Summary of Product Characteristics (SmPC), or corresponding local prescribing information)
- Treatment with any investigational drug within 30 days prior to enrolment into the study
- Previous participation in any clinical trial with turoctocog alfa
- Treatment with other FVIII products after initiation of treatment with turoctocog alfa
Trial locations and contacts
41 sites in 15 countries.
41 sites in 15 countries
Germany8 sites
Novo Nordisk Investigational Site
Braunschweig, 38118
Novo Nordisk Investigational Site
Duisburg, 47051
Novo Nordisk Investigational Site
Frankfurt am Main, 60596
Novo Nordisk Investigational Site
Giessen, 35392
Novo Nordisk Investigational Site
Leipzig, 04289
Novo Nordisk Investigational Site
Mörfelden-Walldorf, 64546
Novo Nordisk Investigational Site
München, 80331
Novo Nordisk Investigational Site
Münster, 48143
United States6 sites
Novo Nordisk Investigational Site
Albuquerque, New Mexico, 87106
Novo Nordisk Investigational Site
Charlotte, North Carolina, 28203
Novo Nordisk Investigational Site
Mobile, Alabama, 36604
Novo Nordisk Investigational Site
Peoria, Illinois, 61615
Novo Nordisk Investigational Site
Salt Lake City, Utah, 84113
Novo Nordisk Investigational Site
Torrance, California, 90502-2004
France5 sites
Novo Nordisk Investigational Site
Bordeaux, 33076
Novo Nordisk Investigational Site
Montmorency, 95160
Novo Nordisk Investigational Site
Rennes, 35033
Novo Nordisk Investigational Site
Saint-Etienne, 42055
Novo Nordisk Investigational Site
Strasbourg, 67098
Italy5 sites
Novo Nordisk Investigational Site
Castelfranco Veneto, 31033
Novo Nordisk Investigational Site
Catania, 95123
Novo Nordisk Investigational Site
Florence, 50134
Novo Nordisk Investigational Site
Padova, 35128
Novo Nordisk Investigational Site
Palermo, 90127
Hungary3 sites
Novo Nordisk Investigational Site
Budapest, H-1134
Novo Nordisk Investigational Site
Debrecen, 4032
Novo Nordisk Investigational Site
Mohács, 7700
Austria2 sites
Novo Nordisk Investigational Site
Graz, 8036
Novo Nordisk Investigational Site
Linz, 4020
Netherlands2 sites
Novo Nordisk Investigational Site
The Hague, 2545AA
Novo Nordisk Investigational Site
Utrecht, 3584 CX
Slovakia2 sites
Novo Nordisk Investigational Site
Banská Bystrica, 975 17
Novo Nordisk Investigational Site
Košice, 04001
Sweden2 sites
Novo Nordisk Investigational Site
Malmö, 205 02
Novo Nordisk Investigational Site
Stockholm, 171 76
About this study
Official title: A Multi-centre Non-interventional Study of Safety and Efficacy of Turoctocog Alfa (rFVIII) During Long-Term Treatment of Severe and Moderately Severe Haemophilia A (FVIII =<2%)
This study is conducted in Europe, and North and South America. The aim of this study is to provide additional documentation of the immunogenicity, and obtain additional clinical data, of turoctocog alfa in the setting of normal clinical practise in patients previously treated with a factor VIII agent (FVIII).
- 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
Patients will be treated with commercially available turoctocog alfa as prescribed by the treating physician in clinical daily practice and preferably according to the label for turoctocog alfa in the respective countries.
| Study group | Role | What they receive |
|---|---|---|
| All patients | Not described Receives: Drug: turoctocog alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence rate of FVIII inhibitors (at least 0.6 Bethesda Units (BU) for central laboratory analyses, or above the specific local laboratory reference range) represented as the percentage of patients developing inhibitors
Measured: Within approximately 7 years
Secondary outcomes (6)
Number of adverse reactions reported
Measured: During approximately 7 years
Number of serious adverse reactions reported
Measured: During approximately 7 years
Haemostatic effect of turoctocog alfa in the treatment of bleeds as assessed by the patient or the physician according to a predefined four point scale: Excellent, Good, Moderate, or None
Measured: Within approximately 7 years
Haemostatic effect of turoctocog alfa during surgical procedures as assessed by an evaluation according to a predefined four point scale: Excellent, Good, Moderate, or None
Measured: Within approximately 7 years
Annualised bleeding rate for patients using turoctocog alfa for preventive treatment
Measured: Within approximately 7 years
Annualised bleeding rate for patients using turoctocog alfa for on-demand treatment
Measured: Within approximately 7 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jan 2014 (estimated)
- Study start5 Jun 2014
- Primary completion15 Jan 2020
- Study completion15 Jan 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 69 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
- NCT02035384
- Sponsor's protocol ID
- NN7008-3553
- Other IDs
- U1111-1126-0353, ENCEPP/SDPP/5501
- Acronym
- guardian™ 5
- Status last verified
- Apr 2020
- First submitted
- 10 Jan 2014
- Last updated
- 27 Apr 2020
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-centre Non-interventional Study of Safety and Efficacy of Turoctocog Alfa (rFVIII) During Long-Term Treatment of Severe and Moderately Severe Haemophilia A (FVIII =<2%)
- ClinicalTrials.gov ID
- NCT02035384
- Acronym / sponsor ID
- guardian™ 5 / NN7008-3553
- Conditions
- Congenital Bleeding Disorder, Haemophilia A
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 69 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 5 Jun 2014 (actual)
- Primary completion
- 15 Jan 2020 (actual)
- Study completion
- 15 Jan 2020 (actual)
- First posted
- 14 Jan 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is conducted in Europe, and North and South America. The aim of this study is to provide additional documentation of the immunogenicity, and obtain additional clinical data, of turoctocog alfa in the setting of normal clinical practise in patients previously treated with a factor VIII agent (FVIII).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All patients | – | Drug: turoctocog alfa |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Previously FVIII treated (more than 150 exposure days) patients with severe and moderately severe haemophilia A with FVIII below or equal to 2%.
Key inclusion criteria (4 listed)
- Informed consent obtained before any study-related activities. Study-related activities are any procedure related to recording of data according to the protocol
- Previously FVIII treated (150 exposure days at the time of first dosing with turoctocog alfa) male patients with the diagnosis of severe and moderately severe haemophilia A (FVIII below or equal to 2%)
- The decision to initiate treatment with commercially available turoctocog alfa has been made by the patient/parent and the patient's treating physician before and independently from the decision to include the patient in this study
- A negative FVIII inhibitor test obtained not more than four weeks prior to first dosing with turoctocog alfa
Key exclusion criteria (4 listed)
- Contraindications for use according to the approved product information text (US Package insert (PI), European Summary of Product Characteristics (SmPC), or corresponding local prescribing information)
- Treatment with any investigational drug within 30 days prior to enrolment into the study
- Previous participation in any clinical trial with turoctocog alfa
- Treatment with other FVIII products after initiation of treatment with turoctocog alfa
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 alfaPatients will be treated with commercially available turoctocog alfa as prescribed by the treating physician in clinical daily practice and preferably according to the label for turoctocog alfa in the respective countries. | Drug | All patients |
Primary outcome
Incidence rate of FVIII inhibitors (at least 0.6 Bethesda Units (BU) for central laboratory analyses, or above the specific local laboratory reference range) represented as the percentage of patients developing inhibitors
Time frame: Within approximately 7 years
6 secondary outcomes
Number of adverse reactions reported
Time frame: During approximately 7 years
Number of serious adverse reactions reported
Time frame: During approximately 7 years
Haemostatic effect of turoctocog alfa in the treatment of bleeds as assessed by the patient or the physician according to a predefined four point scale: Excellent, Good, Moderate, or None
Time frame: Within approximately 7 years
Haemostatic effect of turoctocog alfa during surgical procedures as assessed by an evaluation according to a predefined four point scale: Excellent, Good, Moderate, or None
Time frame: Within approximately 7 years
Annualised bleeding rate for patients using turoctocog alfa for preventive treatment
Time frame: Within approximately 7 years
Annualised bleeding rate for patients using turoctocog alfa for on-demand treatment
Time frame: Within approximately 7 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Apr 2020.
- 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
41 sites in 15 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Congenital Bleeding Disorder and Haemophilia A, run by Novo Nordisk A/S at 41 sites in 15 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 4 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 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 41 sites in Germany, United States, France, Italy, Hungary and 10 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Incidence rate of FVIII inhibitors (at least 0.6 Bethesda Units (BU) for central laboratory analyses, or above the specific local laboratory reference range) represented as the percentage of patients developing inhibitors (time frame: Within approximately 7 years). 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 27 Apr 2020.