Factor VII DeficiencySTERNCT01269138
Treatment of Inherited Factor VII Deficiency
An observational study of Factor VII Deficiency, run by University of L'Aquila at 1 site in Italy.
Trial summary
FVII deficiency is a rare coagulation disorder. A limited number of patients are found in most treatment centres and countries. Treatment demands vary considerably amongst FVII deficient patients. Therefore, regular clinical studies will meet with recruitment problems in this particular patient population. Read more about this study
- Who is it for?
- Ages 1 day to 90 years
- Conditions the study is for
- Factor VII Deficiency
- What is being tested?
- plasma derived Factor VII, recombinant FVIIa and Fresh Frozen Plasma
- How many people
- 223 participants
- Where
- 1 siteItaly
- Study dates
- Jan 2007to Nov 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 1 day to 90 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Number of patients affected by inherited Factor VII deficiency to be studied Planned number of patients to be recruited: >200 Planned number of evaluable bleeding episodes >100 Planned number of surgical procedures >50 It is planned to include patients to the registry from all over the world
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. 2.Males and females 0 to 90 years of age. 3. 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.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Treatment of Congenital Factor VII Deficiency. A Prospective Observational Study
FVII deficiency is a rare coagulation disorder. A limited number of patients are found in most treatment centres and countries. Treatment demands vary considerably amongst FVII deficient patients. Therefore, regular clinical studies will meet with recruitment problems in this particular patient population. The present study intends to elucidate the bleeding patterns in a well-defined collective of FVII deficiency patients who are carefully characterised, to document the actual use of different treatment modalities in different subgroups and to evaluate the efficacy and safety of current available treatment modalities in bleedings, surgery and prophylaxis. The purpose is to gain some evidence based knowledge of treatment of patients with FVII deficiency - an area where treatment decisions are made more on personal clinical experience than on consolidated clinical evidence.
This study intends to register treatment practices as they are actually performed - in a structured and documented way.
Read the detailed description
To describe the treatment modalities and outcomes of: - bleeding episodes - surgery - prophylaxis in a well defined, international cohort of FVII deficient patients characterised following the methodology used by the International Factor VII deficiency Study Group (IF7SG).
To evaluate the presence (in already treated patients) and/or the appearance of inhibiting antibodies to FVII and/or therapy-related thrombosis.
Study Design:
Prospective observational study on treatment of FVII deficiency patients. This is an outcome study conducted through the procedures set up by the IF7SG, in patients already enrolled or newly enrolled in the database.
Study population and products:
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. All pharmaceutical products considered useful for treatment of FVII deficiency by the centres can be included in the study.
Key assessments:
The database is set up to capture the following assessments, if available:
Bleeding episodes:
- Recording of bleeding location, symptom onset and treatment onset \& location • Recording of substitution therapy, concomitant medications and concomitant illness • Recording of 6 hour treatment evaluation for bleeding episodes • Recording of re-bleeding episodes
Surgery/delivery:
- Recording of surgery description , date of surgery and indication • Recording of substitution therapy, concomitant medications and concomitant illness during surgery • Recording of antifibrinolytic drugs used prior, during and/or after surgery •Recording of • Recording of overall treatment evaluation
Prophylaxis:
- Recording of prophylaxis type (primary/secondary), indication for prophylaxis and start/stop date •Recording of prophylaxis therapy, concomitant medications and concomitant illness • Recording of bleeding episodes during prophylaxis •Recording of clinical prophylaxis evaluation
For all:
- Laboratory values (PT/INR, APTT, FVII:C, platelet count, fibrinogen, inhibitor samples) at time points specified in section 7.3.
- Adverse events (including thrombotic events, anaphylactic reactions, DIC reactions, Re- bleedings and mortality)
- Recording of 30 day treatment evaluation (excellent, effective, partly effective, ineffective or not evaluable)
- Duration of hospital stay (ward, ICU) within 30 days after first product treatment • Mortality within 30days after first product administration
- Conditions
- Factor VII Deficiency
- Being tested
- plasma derived Factor VII, recombinant FVIIa, Fresh Frozen Plasma, Activated Prothrombin Complex Concentrates, Virus Inactivated plasma
- Keywords
- Inherited Factor VII deficiency, Inhibitor development in Factor VII deficiency, Bleeding in Factor VII Deficiency, Surgery in Factor VII deficiency, Prophylaxis in Factor VII Deficiency
- Medical subject headings
- Factor VII Deficiency
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
plasma derived Factor VIIDrug
Also called Facteur VII, LFB (plasma-derived);, Factor VII, PFL (plasma-derived), Factor VII, Baxter Immuno (plasma-derived)
Treatment of bleeding episodes,treatment during surgery and prophylaxis
recombinant FVIIaDrug
Also called Novoseven
Treatment of bleeding episodes,treatment during surgery and prophylaxis
Fresh Frozen PlasmaDrug
Also called Blood Transfusion Centre Fresh Frozen Plasma
Treatment of bleeding episodes,treatment during surgery and prophylaxis
Activated Prothrombin Complex ConcentratesDrug
Treatment of bleeding episodes,treatment during surgery and prophylaxis
Virus Inactivated plasmaDrug
Also called Octaplas
Treatment of bleeding episodes,treatment during surgery and prophylaxis
| Study group | Role | What they receive |
|---|---|---|
| Factor VII Deficient Patients | Patients affected by Inherited Factor VII deficiency undergoing treatment for bleeding episodes, surgery , prophylaxis.Any patient with levels of FVII less than 50% of normal or a mutation known to be associated to a FVII deficiency. Any patient with a FVII deficiency for whom treatment of bleeding episodes, prevention related to surgery and primary/secondary prophylaxis is considered necessary by his/her treating physician can be enrolled. Receives: Drug: plasma derived Factor VII; Drug: recombinant FVIIa; Drug: Fresh Frozen Plasma; Drug: Activated Prothrombin Complex Concentrates; Drug: Virus Inactivated plasma |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in Factor VII levels
To describe the treatment modalities and clinical and laboratory outcomes in:
-bleeding episodes - surgery - prophylaxis in a well defined, international cohort of FVII deficient patients characterised following the methodology used by the IF7SG.
Measured: Time 0 (before treatment), 15 minutes and 1 month after treatment
Secondary outcomes (1)
Evaluation of Inhibitor Development
To evaluate the presence (in already treated patients) and/or the appearance of inhibiting antibodies to FVII.
Measured: Monthly
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jan 2011 (estimated)
- Study startJan 2007
- Study completionNov 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 Factor VII DeficiencyStudies of the same condition, open and under way.
- 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
- 223 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of L'Aquila
- Sponsor type
- University, hospital or other
- Collaborators
- TRIB s.r.l.
- Responsible party
- Principal investigator, Guglielmo Mariani, University of L'Aquila
- Investigators
- guglielmo mariani, md, Study director, University of L'Aquila
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01269138
- Sponsor's protocol ID
- STERProtocol7.0
- Acronym
- STER
- Status last verified
- Nov 2012
- First submitted
- 17 Dec 2010
- Last updated
- 27 Nov 2012
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Morfini M, Batorova A, Mariani G, Auerswald G, Bernardi F, Di Minno G, Dolce A, Fede C, Giansily-Blaizot M, Ingerslev J, Martinowitz U, Napolitano M, Pinotti M, Schved JF; International FVII [IF7] and Seven Treatment Evaluation Registry [STER] Study Groups. Pharmacokinetic properties of recombinant FVIIa in inherited FVII deficiency account for a large volume of distribution at steady state and a prolonged pharmacodynamic effect. Thromb Haemost. 2014 Aug;112(2):424-5. doi: 10.1160/TH13-12-1045. Epub 2014 Apr 24. No abstract available.
PubMed 24763923Napolitano M, Giansily-Blaizot M, Dolce A, Schved JF, Auerswald G, Ingerslev J, Bjerre J, Altisent C, Charoenkwan P, Michaels L, Chuansumrit A, Di Minno G, Caliskan U, Mariani G. Prophylaxis in congenital factor VII deficiency: indications, efficacy and safety. Results from the Seven Treatment Evaluation Registry (STER). Haematologica. 2013 Apr;98(4):538-44. doi: 10.3324/haematol.2012.074039. Epub 2013 Feb 12.
PubMed 23403322
Study at a glance
From the registry record, last updated by the sponsor on 27 Nov 2012. 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
- NCT01269138
- Acronym / sponsor ID
- STER / STERProtocol7.0
- Conditions
- Factor VII Deficiency
- Keywords
- Inherited Factor VII deficiency, Inhibitor development in Factor VII deficiency, Bleeding in Factor VII Deficiency, Surgery in Factor VII deficiency, Prophylaxis in Factor VII Deficiency
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 223 participants (actual)
- Lead sponsor
- University of L'AquilaUniversity, hospital or other
- Collaborators
- TRIB s.r.l.
- Study start
- Jan 2007
- Study completion
- Nov 2012 (actual)
- First posted
- 4 Jan 2011 (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.
FVII deficiency is a rare coagulation disorder. A limited number of patients are found in most treatment centres and countries. Treatment demands vary considerably amongst FVII deficient patients. Therefore, regular clinical studies will meet with recruitment problems in this particular patient population. The present study intends to elucidate the bleeding patterns in a well-defined collective of FVII deficiency patients who are carefully characterised, to document the actual use of different treatment modalities in different subgroups and to evaluate the efficacy and safety of current available treatment modalities in bleedings, surgery and prophylaxis. The purpose is to gain some evidence based knowledge of treatment of patients with FVII deficiency - an area where treatment decisions are made more on personal clinical experience than on consolidated clinical evidence.
This study intends to register treatment practices as they are actually performed - in a structured and documented way.
Detailed description
To describe the treatment modalities and outcomes of: - bleeding episodes - surgery - prophylaxis in a well defined, international cohort of FVII deficient patients characterised following the methodology used by the International Factor VII deficiency Study Group (IF7SG).
To evaluate the presence (in already treated patients) and/or the appearance of inhibiting antibodies to FVII and/or therapy-related thrombosis.
Study Design:
Prospective observational study on treatment of FVII deficiency patients. This is an outcome study conducted through the procedures set up by the IF7SG, in patients already enrolled or newly enrolled in the database.
Study population and products:
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. All pharmaceutical products considered useful for treatment of FVII deficiency by the centres can be included in the study.
Key assessments:
The database is set up to capture the following assessments, if available:
Bleeding episodes:
- Recording of bleeding location, symptom onset and treatment onset \& location • Recording of substitution therapy, concomitant medications and concomitant illness • Recording of 6 hour treatment evaluation for bleeding episodes • Recording of re-bleeding episodes
Surgery/delivery:
- Recording of surgery description , date of surgery and indication • Recording of substitution therapy, concomitant medications and concomitant illness during surgery • Recording of antifibrinolytic drugs used prior, during and/or after surgery •Recording of • Recording of overall treatment evaluation
Prophylaxis:
- Recording of prophylaxis type (primary/secondary), indication for prophylaxis and start/stop date •Recording of prophylaxis therapy, concomitant medications and concomitant illness • Recording of bleeding episodes during prophylaxis •Recording of clinical prophylaxis evaluation
For all:
- Laboratory values (PT/INR, APTT, FVII:C, platelet count, fibrinogen, inhibitor samples) at time points specified in section 7.3.
- Adverse events (including thrombotic events, anaphylactic reactions, DIC reactions, Re- bleedings and mortality)
- Recording of 30 day treatment evaluation (excellent, effective, partly effective, ineffective or not evaluable)
- Duration of hospital stay (ward, ICU) within 30 days after first product treatment • Mortality within 30days after first product administration
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Factor VII Deficient Patients | – | Drug: plasma derived Factor VII, Drug: recombinant FVIIa, Drug: Fresh Frozen Plasma, Drug: Activated Prothrombin Complex Concentrates, Drug: Virus Inactivated plasmaPatients affected by Inherited Factor VII deficiency undergoing treatment for bleeding episodes, surgery , prophylaxis.Any patient with levels of FVII less than 50% of normal or a mutation known to be associated to a FVII deficiency. Any patient with a FVII deficiency for whom treatment of bleeding episodes, prevention related to surgery and primary/secondary prophylaxis is considered necessary by his/her treating physician can be enrolled. |
Who may qualify
- Ages
- 1 day to 90 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Number of patients affected by inherited Factor VII deficiency to be studied Planned number of patients to be recruited: >200 Planned number of evaluable bleeding episodes >100 Planned number of surgical procedures >50 It is planned to include patients to the registry from all over the world
Key inclusion criteria (2 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. 2.Males and females 0 to 90 years of age. 3. 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.
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 |
|---|---|---|
| plasma derived Factor VIIAlso: Facteur VII, LFB (plasma-derived);, Factor VII, PFL (plasma-derived), Factor VII, Baxter Immuno (plasma-derived)Treatment of bleeding episodes,treatment during surgery and prophylaxis | Drug | Factor VII Deficient Patients |
| recombinant FVIIaAlso: NovosevenTreatment of bleeding episodes,treatment during surgery and prophylaxis | Drug | Factor VII Deficient Patients |
| Fresh Frozen PlasmaAlso: Blood Transfusion Centre Fresh Frozen PlasmaTreatment of bleeding episodes,treatment during surgery and prophylaxis | Drug | Factor VII Deficient Patients |
| Activated Prothrombin Complex ConcentratesTreatment of bleeding episodes,treatment during surgery and prophylaxis | Drug | Factor VII Deficient Patients |
| Virus Inactivated plasmaAlso: OctaplasTreatment of bleeding episodes,treatment during surgery and prophylaxis | Drug | Factor VII Deficient Patients |
Primary outcome
Changes in Factor VII levels
Time frame: Time 0 (before treatment), 15 minutes and 1 month after treatment
To describe the treatment modalities and clinical and laboratory outcomes in: -bleeding episodes - surgery - prophylaxis in a well defined, international cohort of FVII deficient patients characterised following the methodology used by the IF7SG.
1 secondary outcome
Evaluation of Inhibitor Development
Time frame: Monthly
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 Nov 2012.
- Derived from this study: PubMed 24763923Morfini M, Batorova A, Mariani G, Auerswald G, Bernardi F, Di Minno G, Dolce A, Fede C, Giansily-Blaizot M, Ingerslev J, Martinowitz U, Napolitano M, Pinotti M, Schved JF; International FVII [IF7] and Seven Treatment Evaluation Registry [STER] Study Groups. Pharmacokinetic properties of recombinant FVIIa in inherited FVII deficiency account for a large volume of distribution at steady state and a prolonged pharmacodynamic effect. Thromb Haemost. 2014 Aug;112(2):424-5. doi: 10.1160/TH13-12-1045. Epub 2014 Apr 24. No abstract available.
- Derived from this study: PubMed 23403322Napolitano M, Giansily-Blaizot M, Dolce A, Schved JF, Auerswald G, Ingerslev J, Bjerre J, Altisent C, Charoenkwan P, Michaels L, Chuansumrit A, Di Minno G, Caliskan U, Mariani G. Prophylaxis in congenital factor VII deficiency: indications, efficacy and safety. Results from the Seven Treatment Evaluation Registry (STER). Haematologica. 2013 Apr;98(4):538-44. doi: 10.3324/haematol.2012.074039. Epub 2013 Feb 12.
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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.
guglielmo mariani, md study director
University of L'Aquila
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Factor VII Deficiency, run by University of L'Aquila at 1 site in Italy.
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 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 27 Nov 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. 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?
Changes in Factor VII levels (time frame: Time 0 (before treatment), 15 minutes and 1 month after treatment). 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 NCT01269138, 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 27 Nov 2012.