Treatment of Inherited Factor VII Deficiency (STER)

Tracking Information
First Submitted Date	December 17, 2010
First Posted Date	January 4, 2011
Last Update Posted Date	November 27, 2012
Study Start Date	January 2007
Primary Completion Date	Not Provided
Current Primary Outcome Measures; (submitted: January 3, 2011)	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.
Original Primary Outcome Measures	Same as current
Change History	Complete list of historical versions of study NCT01269138 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures; (submitted: January 3, 2011)	Evaluation of Inhibitor Development [ Time Frame: Monthly ]; To evaluate the presence (in already treated patients) and/or the appearance of inhibiting antibodies to FVII.
Original Secondary Outcome Measures	Same as current
Current Other Outcome Measures	Not Provided
Original Other Outcome Measures	Not Provided
Descriptive Information
Brief Title	Treatment of Inherited Factor VII Deficiency
Official Title	Treatment of Congenital Factor VII Deficiency. A Prospective Observational Study
Brief 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. 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 Type	Observational
Study Design	Observational Model: Cohort; Time Perspective: Prospective
Target Follow-Up Duration	Not Provided
Biospecimen	Retention: Samples Without DNA; Description: 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
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
Condition	Factor VII Deficiency
Intervention	Drug: plasma derived Factor VII Treatment of bleeding episodes,treatment during surgery and prophylaxis Other Names: Facteur VII, LFB (plasma-derived); Factor VII, PFL (plasma-derived) Factor VII, Baxter Immuno (plasma-derived); Drug: recombinant FVIIa Treatment of bleeding episodes,treatment during surgery and prophylaxis Other Name: Novoseven; Drug: Fresh Frozen Plasma Treatment of bleeding episodes,treatment during surgery and prophylaxis Other Name: Blood Transfusion Centre Fresh Frozen Plasma; Drug: Activated Prothrombin Complex Concentrates Treatment of bleeding episodes,treatment during surgery and prophylaxis; Drug: Virus Inactivated plasma Treatment of bleeding episodes,treatment during surgery and prophylaxis Other Name: Octaplas
Study Groups/Cohorts	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.; Interventions: Drug: plasma derived Factor VII; Drug: recombinant FVIIa; Drug: Fresh Frozen Plasma; Drug: Activated Prothrombin Complex Concentrates; Drug: Virus Inactivated plasma
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.; 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 Information
Recruitment Status	Completed
Actual Enrollment; (submitted: November 26, 2012)	223
Original Estimated Enrollment; (submitted: January 3, 2011)	250
Actual Study Completion Date	November 2012
Primary Completion Date	Not Provided
Eligibility Criteria	Inclusion Criteria: 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. Exclusion Criteria:
Sex/Gender	Sexes Eligible for Study: All
Ages	up to 90 Years (Child, Adult, Senior)
Accepts Healthy Volunteers	No
Contacts	Contact information is only displayed when the study is recruiting subjects
Listed Location Countries	Italy
Removed Location Countries
Administrative Information
NCT Number	NCT01269138
Other Study ID Numbers	STERProtocol7.0
Has Data Monitoring Committee	Yes
U.S. FDA-regulated Product	Not Provided
IPD Sharing Statement	Not Provided
Responsible Party	Guglielmo Mariani, University of L'Aquila
Study Sponsor	University of L'Aquila
Collaborators	TRIB s.r.l.
Investigators	Study Director: guglielmo mariani, md University of L'Aquila
PRS Account	University of L'Aquila
Verification Date	November 2012