Treatment of Inherited Factor VII Deficiency

NCT01269138 | Completed DMC

• 1 other identifier: STERProtocol7.0
• Study Type: observational
• Target: 223
• Locations: 1 country
• Sites: 1

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.

Conditions

Factor VII Deficiency

Keywords

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

Outcome Measures

Primary Outcomes (1)

• Changes in Factor VII levels

  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.

  Time 0 (before treatment), 15 minutes and 1 month after treatment

Secondary Outcomes (1)

• Evaluation of Inhibitor Development

  Monthly

Study Arms (1)

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; Drug: recombinant FVIIa; Drug: Fresh Frozen Plasma; Drug: Activated Prothrombin Complex Concentrates; Drug: Virus Inactivated plasma

Interventions

plasma derived Factor VII DRUG

Treatment of bleeding episodes,treatment during surgery and prophylaxis

Also known as: Facteur VII, LFB (plasma-derived);, Factor VII, PFL (plasma-derived), Factor VII, Baxter Immuno (plasma-derived)

Factor VII Deficient Patients

recombinant FVIIa DRUG

Treatment of bleeding episodes,treatment during surgery and prophylaxis

Also known as: Novoseven

Factor VII Deficient Patients

Fresh Frozen Plasma DRUG

Treatment of bleeding episodes,treatment during surgery and prophylaxis

Also known as: Blood Transfusion Centre Fresh Frozen Plasma

Factor VII Deficient Patients

Activated Prothrombin Complex Concentrates DRUG

Treatment of bleeding episodes,treatment during surgery and prophylaxis

Factor VII Deficient Patients

Virus Inactivated plasma DRUG

Treatment of bleeding episodes,treatment during surgery and prophylaxis

Also known as: Octaplas

Factor VII Deficient Patients

Eligibility Criteria

• Age: 1 Day - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• 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.

Sponsors & Collaborators

• University of L'Aquila: lead
• TRIB s.r.l.: collaborator

Study Sites (1)

San Salvatore Hospital

L’Aquila, AQ, 67100, Italy

Location

Related Publications (2)

• 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. No abstract available.

  PMID: 24763923 DERIVED

• 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.

  PMID: 23403322 DERIVED

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

MeSH Terms

Conditions

Factor VII Deficiency

Interventions

Factor VII; recombinant FVIIa

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Enzyme Precursors; Enzymes and Coenzymes; Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Study Officials

• guglielmo mariani, md

  University of L'Aquila

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Hematology

Study Record Dates

• First Submitted: December 17, 2010
• First Posted: January 4, 2011
• Study Start: January 1, 2007
• Study Completion: November 1, 2012
• Last Updated: November 27, 2012

Record last verified: 2012-11

Locations

IT (1)