Clinical Trial: Treatment of Inherited Factor VII Deficiency

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

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 Summary:

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:

• Recor
Sponsor: University of L'Aquila

Current Primary Outcome: Changes in Factor VII levels [ Time Frame: Time 0 (before treatment), 15 minutes and 1 month after treatment ]

Original Primary Outcome: Same as current

Current Secondary Outcome: Evaluation of Inhibitor Development [ Time Frame: Monthly ]

Original Secondary Outcome: Same as current

Information By: University of L'Aquila

Dates:
Date Received: December 17, 2010
Date Started: January 2007
Date Completion:
Last Updated: November 26, 2012
Last Verified: November 2012