Treatment of Inherited Factor VII Deficiency (STER)

This study has been completed.
Sponsor:
Collaborator:
TRIB s.r.l.
Information provided by (Responsible Party):
Guglielmo Mariani, University of L'Aquila
ClinicalTrials.gov Identifier:
NCT01269138
First received: December 17, 2010
Last updated: November 26, 2012
Last verified: November 2012
  Purpose

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.


Condition Intervention
Factor VII Deficiency
Drug: plasma derived Factor VII
Drug: recombinant FVIIa
Drug: Fresh Frozen Plasma
Drug: Activated Prothrombin Complex Concentrates
Drug: Virus Inactivated plasma

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Treatment of Congenital Factor VII Deficiency. A Prospective Observational Study

Resource links provided by NLM:


Further study details as provided by University of L'Aquila:

Primary Outcome Measures:
  • 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.



Secondary Outcome Measures:
  • 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.


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


Enrollment: 223
Study Start Date: January 2007
Study Completion Date: November 2012
Groups/Cohorts Assigned Interventions
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.
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

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
  Eligibility

Ages Eligible for Study:   up to 90 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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

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:

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01269138

Locations
Italy
San Salvatore Hospital
L'Aquila, AQ, Italy, 67100
Sponsors and Collaborators
University of L'Aquila
TRIB s.r.l.
Investigators
Study Director: guglielmo mariani, md University of L'Aquila
  More Information

No publications provided by University of L'Aquila

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Guglielmo Mariani, Professor of Hematology, University of L'Aquila
ClinicalTrials.gov Identifier: NCT01269138     History of Changes
Other Study ID Numbers: STERProtocol7.0
Study First Received: December 17, 2010
Last Updated: November 26, 2012
Health Authority: Italy: The Italian Medicines Agency
United States: Food and Drug Administration

Keywords provided by University of L'Aquila:
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

Additional relevant MeSH terms:
Factor VII Deficiency
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Thrombin
Hemostatics
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 20, 2014