Treatment of Inherited Factor VII Deficiency — STER  

Factor VII Deficiency Clinical Trial

Official title: Treatment of Congenital Factor VII Deficiency. A Prospective Observational Study

Status Completed

Clinical 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. 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.

Clinical Trial 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

Related Conditions & MeSH terms

• Factor VII Deficiency

• NCT number: NCT01269138
• Study type: Observational
• Source: University of L'Aquila
• Status: Completed
• Phase: N/A
• Start date: January 2007
• Completion date: November 2012