Treatment of Inherited Factor VII Deficiency (STER)
|First Received Date ICMJE||December 17, 2010|
|Last Updated Date||November 26, 2012|
|Start Date ICMJE||January 2007|
|Primary Completion Date||Not Provided|
|Current Primary Outcome Measures ICMJE
||Changes in Factor VII levels [ Time Frame: Time 0 (before treatment), 15 minutes and 1 month after treatment ] [ Designated as safety issue: Yes ]
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.
|Original Primary Outcome Measures ICMJE||Same as current|
|Change History||Complete list of historical versions of study NCT01269138 on ClinicalTrials.gov Archive Site|
|Current Secondary Outcome Measures ICMJE
||Evaluation of Inhibitor Development [ Time Frame: Monthly ] [ Designated as safety issue: Yes ]
To evaluate the presence (in already treated patients) and/or the appearance of inhibiting antibodies to FVII.
|Original Secondary Outcome Measures ICMJE||Same as current|
|Current Other Outcome Measures ICMJE||Not Provided|
|Original Other Outcome Measures ICMJE||Not Provided|
|Brief Title ICMJE||Treatment of Inherited Factor VII Deficiency|
|Official Title ICMJE||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.
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.
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.
The database is set up to capture the following assessments, if available:
• 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
• 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
• 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
|Study Type ICMJE||Observational|
|Study Design ICMJE||Observational Model: Cohort
Time Perspective: Prospective
|Target Follow-Up Duration||Not Provided|
|Biospecimen||Retention: Samples Without DNA
Plasma for Factor VII inhibitor assay is stored and sent to a Central Laboratory for the inhibitor determination. Samples are discharged after the inhibitor determination
|Sampling Method||Non-Probability Sample|
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
|Condition ICMJE||Factor VII Deficiency|
|Study Group/Cohort (s)||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.
|Publications *||Napolitano 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.|
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
|Recruitment Status ICMJE||Completed|
|Completion Date||November 2012|
|Primary Completion Date||Not Provided|
|Eligibility Criteria ICMJE||
|Ages||up to 90 Years|
|Accepts Healthy Volunteers||No|
|Contacts ICMJE||Contact information is only displayed when the study is recruiting subjects|
|Location Countries ICMJE||Italy|
|NCT Number ICMJE||NCT01269138|
|Other Study ID Numbers ICMJE||STERProtocol7.0|
|Has Data Monitoring Committee||Yes|
|Responsible Party||Guglielmo Mariani, University of L'Aquila|
|Study Sponsor ICMJE||University of L'Aquila|
|Collaborators ICMJE||TRIB s.r.l.|
|Information Provided By||University of L'Aquila|
|Verification Date||November 2012|
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP