Congenital Bleeding DisorderF7CONDEFNCT01779921
Treatment of Congenital Factor VII Deficiency
An observational study of Congenital Bleeding Disorder and Congenital FVII Deficiency, run by Novo Nordisk A/S at 16 sites in 16 countries.
Trial summary
This study is conducted globally. The aim of this study is to describe the treatment modalities and outcomes of bleeding episodes, surgery and prophylaxis in patients with factor VII (FVII) deficiency in addition to evaluate the presence (in already treated patients) and/or the appearance of inhibiting antibodies to… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Congenital Bleeding Disorder, Congenital FVII Deficiency
- What is being tested?
- activated recombinant human factor VII, Fresh frozen plasma (Source unspecified) and Plasma-derived FVII (LFB)
- How many people
- 163 participants
- Where
- 16 sitesin 16 countries
- Study dates
- Oct 2005to Jan 2012
- 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
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with FVII deficiency (levels of FVII less than 50% of normal or a mutation known to be associated to a FVII deficiency) can be enrolled.
Who may be able to join
- Signed informed consent by the patient or next of kin or legally acceptable representative to collect data on treatment of a given bleeding episode, surgical event or prophylactic regimen as specified in the protocol. If informed consent is provided by the next of kin or legally acceptable representative, consent must also be obtained from the patient as soon as he/she is able to do so. Informed consent should preferentially be obtained before initiation of treatment or as a minimum before entry of data into the database
- Any patient with a FVII deficiency for whom treatment of bleeding episodes, prevention related to surgery and primary/secondary prophylaxis is considered necessary by the treating physician can be enrolled
- Patients with FVII deficiency without any immediate need for treatment will be entered as stand by registered patients with capture of baseline- and demographic data only. Admission data is entered once an event occurs
Who cannot join
None listed.
Trial locations and contacts
16 sites in 16 countries.
16 sites in 16 countries
United States1 site
Novo Nordisk Investigational Site
Princeton, New Jersey, 08540
Turkey (Türkiye)1 site
Novo Nordisk Investigational Site
Istanbul, 34335
About this study
Official title: Treatment of Congenital Factor VII Deficiency. A Prospective Observational Study
This study is conducted globally. The aim of this study is to describe the treatment modalities and outcomes of bleeding episodes, surgery and prophylaxis in patients with factor VII (FVII) deficiency in addition to evaluate the presence (in already treated patients) and/or the appearance of inhibiting antibodies to FVII and/or therapy-related thrombosis.
Due to a Novo Nordisk commitment to the Committee for Medicinal Products for Human Use (CHMP), Novo Nordisk receives data on treatment with activated recombinant human FVII (rFVIIa, NovoSeven®) in patients with FVII deficiency from the Seven Treatment Evaluation Registry (STER, NCT01269138). These patients can also have been treated with other haemostatics for systemic administration.
- Being tested
- activated recombinant human factor VII, Fresh frozen plasma (Source unspecified), Plasma-derived FVII (LFB), Prothrombin Complex conc. (PCC), Plasma-derived FVII conc. (pdFVII Baxter)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
activated recombinant human factor VIIDrug
Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.
Fresh frozen plasma (Source unspecified)Drug
Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.
Plasma-derived FVII (LFB)Drug
Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.
Prothrombin Complex conc. (PCC)Drug
Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.
Plasma-derived FVII conc. (pdFVII Baxter)Drug
Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.
Plasma-derived FVII conc. (pdFVII PFL)Drug
Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.
| Study group | Role | What they receive |
|---|---|---|
| FVII | Not described Receives: Drug: activated recombinant human factor VII; Drug: Fresh frozen plasma (Source unspecified); Drug: Plasma-derived FVII (LFB); Drug: Prothrombin Complex conc. (PCC); Drug: Plasma-derived FVII conc. (pdFVII Baxter); Drug: Plasma-derived FVII conc. (pdFVII PFL) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment of bleeding episodes at clinic/hospital: Treatment efficacy evaluation for each treatment modality: excellent, effective, partly effective, ineffective, or not evaluable
Measured: Evaluated at 6 hours
Treatment of bleeding episodes at clinic/hospital: Treatment efficacy evaluation for each treatment modality: excellent, effective, partly effective, ineffective, or not evaluable
Measured: Evaluated after 30 days
Treatment of bleeding episodes at clinic/hospital: Time to achieve arrest of bleeding
Measured: Time to achieve arrest of bleeding
Treatment of bleeding episodes at clinic/hospital: Number of re-bleeding episodes
Measured: Within 5 days after first product administration
Treatment of bleeding episodes at home: Treatment efficacy evaluation for each treatment modality: excellent, effective, partly effective, ineffective, or not evaluable
Measured: Evaluated at 6 hours
Treatment of bleeding episodes at home: Time to achieve arrest of bleeding
Measured: Time to achieve arrest of bleeding
Treatment efficacy (of first and/or second treatment modality) evaluated after surgery: good, partially effective, not evaluable, or ineffective
Measured: After surgery
Treatment efficacy (of first and/or second treatment modality) evaluated after surgery: good, partially effective, not evaluable, or ineffective
Measured: Evaluated after 30 days
Estimated blood loss volume
Measured: During surgery/delivery
Number of red blood cell units administered
Measured: During surgery
Number of days spent in hospital
Measured: Until last data collection (20 Jan 2012)
Number of re-bleeding episodes (associated with the surgery)
Measured: Within 5 days after surgery
Prophylactic treatment efficacy evaluation: excellent, excellent, partially effective, or effective
Measured: 30 days after first prophylaxis dose
Secondary outcomes (10)
Number of bleeding episodes during prophylaxis per year
Measured: Up to one year
Number of intensive care unit (ICU) and/or the number of ward days
Measured: After first haemostatic product administration until day 30
Mortality
Measured: Within a 30-day (follow-up) period
Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen)
Measured: Prior to dosing
Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen)
Measured: After 15 minutes
Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen)
Measured: After 30 days
Presence of and/or de novo appearance of inhibiting antibodies to FVII
Measured: Prior to dosing
Presence of and/or de novo appearance of inhibiting antibodies to FVII
Measured: After 30 days
Number of Adverse Events
Measured: Until Day 5
Number of Serious Adverse Events
Measured: Until Day 30
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jan 2013 (estimated)
- Study startOct 2005
- Primary completionJan 2012
- Study completionJan 2012
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 163 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
- NCT01779921
- Sponsor's protocol ID
- F7HAEM-3578
- Acronym
- F7CONDEF
- Status last verified
- Jan 2017
- First submitted
- 15 Jan 2013
- Last updated
- 12 Jan 2017
- Results posted
- No
- Data monitoring committee
- No
Publications
Mariani G, Dolce A, Batorova A, Auerswald G, Schved JF, Siragusa S, Napolitano M, Knudsen JB, Ingerslev J; STER and the International Factor VII Deficiency Study Groups. Recombinant, activated factor VII for surgery in factor VII deficiency: a prospective evaluation - the surgical STER. Br J Haematol. 2011 Feb;152(3):340-6. doi: 10.1111/j.1365-2141.2010.08287.x. Epub 2010 Dec 16.
PubMed 21158750Mariani G, Dolce A, Napolitano M, Ingerslev J, Giansily-Blaizot M, Di Minno MD, Auerswald G, De Saez AR, Tagliaferri A, Batorova A; STER (Seven Treatment Evaluation Registry). Invasive procedures and minor surgery in factor VII deficiency. Haemophilia. 2012 May;18(3):e63-5. doi: 10.1111/j.1365-2516.2012.02751.x. Epub 2012 Feb 22. No abstract available.
PubMed 22356641
Study at a glance
From the registry record, last updated by the sponsor on 12 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Treatment of Congenital Factor VII Deficiency. A Prospective Observational Study
- ClinicalTrials.gov ID
- NCT01779921
- Acronym / sponsor ID
- F7CONDEF / F7HAEM-3578
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 163 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- Oct 2005
- Primary completion
- Jan 2012 (actual)
- Study completion
- Jan 2012 (actual)
- First posted
- 30 Jan 2013 (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 globally. The aim of this study is to describe the treatment modalities and outcomes of bleeding episodes, surgery and prophylaxis in patients with factor VII (FVII) deficiency in addition to evaluate the presence (in already treated patients) and/or the appearance of inhibiting antibodies to FVII and/or therapy-related thrombosis.
Due to a Novo Nordisk commitment to the Committee for Medicinal Products for Human Use (CHMP), Novo Nordisk receives data on treatment with activated recombinant human FVII (rFVIIa, NovoSeven®) in patients with FVII deficiency from the Seven Treatment Evaluation Registry (STER, NCT01269138). These patients can also have been treated with other haemostatics for systemic administration.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| FVII | – | Drug: activated recombinant human factor VII, Drug: Fresh frozen plasma (Source unspecified), Drug: Plasma-derived FVII (LFB), Drug: Prothrombin Complex conc. (PCC), Drug: Plasma-derived FVII conc. (pdFVII Baxter), Drug: Plasma-derived FVII conc. (pdFVII PFL) |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with FVII deficiency (levels of FVII less than 50% of normal or a mutation known to be associated to a FVII deficiency) can be enrolled.
Key inclusion criteria (3 listed)
- Signed informed consent by the patient or next of kin or legally acceptable representative to collect data on treatment of a given bleeding episode, surgical event or prophylactic regimen as specified in the protocol. If informed consent is provided by the next of kin or legally acceptable representative, consent must also be obtained from the patient as soon as he/she is able to do so. Informed consent should preferentially be obtained before initiation of treatment or as a minimum before entry of data into the database
- Any patient with a FVII deficiency for whom treatment of bleeding episodes, prevention related to surgery and primary/secondary prophylaxis is considered necessary by the treating physician can be enrolled
- Patients with FVII deficiency without any immediate need for treatment will be entered as stand by registered patients with capture of baseline- and demographic data only. Admission data is entered once an event occurs
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| activated recombinant human factor VIITreatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator. | Drug | FVII |
| Fresh frozen plasma (Source unspecified)Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator. | Drug | FVII |
| Plasma-derived FVII (LFB)Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator. | Drug | FVII |
| Prothrombin Complex conc. (PCC)Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator. | Drug | FVII |
| Plasma-derived FVII conc. (pdFVII Baxter)Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator. | Drug | FVII |
| Plasma-derived FVII conc. (pdFVII PFL)Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator. | Drug | FVII |
Primary outcome
Treatment of bleeding episodes at clinic/hospital: Treatment efficacy evaluation for each treatment modality: excellent, effective, partly effective, ineffective, or not evaluable
Time frame: Evaluated at 6 hours
Treatment of bleeding episodes at clinic/hospital: Treatment efficacy evaluation for each treatment modality: excellent, effective, partly effective, ineffective, or not evaluable
Time frame: Evaluated after 30 days
Treatment of bleeding episodes at clinic/hospital: Time to achieve arrest of bleeding
Time frame: Time to achieve arrest of bleeding
Treatment of bleeding episodes at clinic/hospital: Number of re-bleeding episodes
Time frame: Within 5 days after first product administration
Treatment of bleeding episodes at home: Treatment efficacy evaluation for each treatment modality: excellent, effective, partly effective, ineffective, or not evaluable
Time frame: Evaluated at 6 hours
Treatment of bleeding episodes at home: Time to achieve arrest of bleeding
Time frame: Time to achieve arrest of bleeding
Treatment efficacy (of first and/or second treatment modality) evaluated after surgery: good, partially effective, not evaluable, or ineffective
Time frame: After surgery
Treatment efficacy (of first and/or second treatment modality) evaluated after surgery: good, partially effective, not evaluable, or ineffective
Time frame: Evaluated after 30 days
Estimated blood loss volume
Time frame: During surgery/delivery
Number of red blood cell units administered
Time frame: During surgery
Number of days spent in hospital
Time frame: Until last data collection (20 Jan 2012)
Number of re-bleeding episodes (associated with the surgery)
Time frame: Within 5 days after surgery
Prophylactic treatment efficacy evaluation: excellent, excellent, partially effective, or effective
Time frame: 30 days after first prophylaxis dose
10 secondary outcomes
Number of bleeding episodes during prophylaxis per year
Time frame: Up to one year
Number of intensive care unit (ICU) and/or the number of ward days
Time frame: After first haemostatic product administration until day 30
Mortality
Time frame: Within a 30-day (follow-up) period
Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen)
Time frame: Prior to dosing
Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen)
Time frame: After 15 minutes
Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen)
Time frame: After 30 days
Presence of and/or de novo appearance of inhibiting antibodies to FVII
Time frame: Prior to dosing
Presence of and/or de novo appearance of inhibiting antibodies to FVII
Time frame: After 30 days
Number of Adverse Events
Time frame: Until Day 5
Number of Serious Adverse Events
Time frame: Until Day 30
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 12 Jan 2017.
- Results publication: PubMed 21158750Mariani G, Dolce A, Batorova A, Auerswald G, Schved JF, Siragusa S, Napolitano M, Knudsen JB, Ingerslev J; STER and the International Factor VII Deficiency Study Groups. Recombinant, activated factor VII for surgery in factor VII deficiency: a prospective evaluation - the surgical STER. Br J Haematol. 2011 Feb;152(3):340-6. doi: 10.1111/j.1365-2141.2010.08287.x. Epub 2010 Dec 16.
- Results publication: PubMed 22356641Mariani G, Dolce A, Napolitano M, Ingerslev J, Giansily-Blaizot M, Di Minno MD, Auerswald G, De Saez AR, Tagliaferri A, Batorova A; STER (Seven Treatment Evaluation Registry). Invasive procedures and minor surgery in factor VII deficiency. Haemophilia. 2012 May;18(3):e63-5. doi: 10.1111/j.1365-2516.2012.02751.x. Epub 2012 Feb 22. No abstract available.
- 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
16 sites in 16 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 Congenital FVII Deficiency, run by Novo Nordisk A/S at 16 sites in 16 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 3 inclusion and 0 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 12 Jan 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States, France, Germany, Greece, Hong Kong and 11 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?
Treatment of bleeding episodes at clinic/hospital: Treatment efficacy evaluation for each treatment modality: excellent, effective, partly effective, ineffective, or not evaluable (time frame: Evaluated at 6 hours). The study lists 13 primary outcomes in all. 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 NCT01779921, which also lists 2 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 12 Jan 2017.