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

December 17, 2010
November 26, 2012
January 2007
Not Provided
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.

Same as current
Complete list of historical versions of study NCT01269138 on ClinicalTrials.gov Archive Site
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.
Same as current
Not Provided
Not Provided
 
Treatment of Inherited Factor VII Deficiency
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.

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
Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
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

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

Factor VII Deficiency
  • 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
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
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.
 
Completed
223
November 2012
Not Provided

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:

Both
up to 90 Years
No
Contact information is only displayed when the study is recruiting subjects
Italy
 
NCT01269138
STERProtocol7.0
Yes
Guglielmo Mariani, University of L'Aquila
University of L'Aquila
TRIB s.r.l.
Study Director: guglielmo mariani, md University of L'Aquila
University of L'Aquila
November 2012

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP