Treatment of Congenital Factor VII Deficiency

Congenital Bleeding Disorder Clinical Trial

— F7CONDEF  

Official title:

Treatment of Congenital Factor VII Deficiency. A Prospective Observational Study

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01779921
• Other study ID #: F7HAEM-3578
• Status: Completed
• Phase: N/A
• First received: January 15, 2013
• Last updated: January 11, 2017
• Start date: October 2005
• Est. completion date: January 2012

Study information

• Verified date: January 2017
• Source: Novo Nordisk A/S
• Contact: n/a
• Is FDA regulated: No
• Health authority: France: Ministry of HealthGermany: Paul-Ehrlich-InstitutGreece: National Organization of MedicinesHong Kong: Department of HealthIndia: Drugs Controller General of IndiaIran: Ministry of HealthIsrael: Israeli Health Ministry Pharmaceutical AdministrationItaly: The Italian Medicines AgencyPakistan: Ministry of HealthSerbia: Agency for Drugs and Medicinal DevicesSlovakia: State Institute for Drug ControlSpain: Spanish Agency of MedicinesThailand: Food and Drug AdministrationTurkey: Ministry of HealthUnited States: Food and Drug AdministrationVenezuela: Ministry of Health and Social Development
• Study type: Observational

Clinical Trial Summary

This study is conducted globally. The aim of this study is to describe the treatment modalities and outcomes of bleeding episodes, surgery and prophylaxis in patients with factor VII (FVII) deficiency in addition to evaluate the presence (in already treated patients) and/or the appearance of inhibiting antibodies to FVII and/or therapy-related thrombosis.

Due to a Novo Nordisk commitment to the Committee for Medicinal Products for Human Use (CHMP), Novo Nordisk receives data on treatment with activated recombinant human FVII (rFVIIa, NovoSeven®) in patients with FVII deficiency from the Seven Treatment Evaluation Registry (STER, NCT01269138). These patients can also have been treated with other haemostatics for systemic administration.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 163
• Est. completion date: January 2012
• Est. primary completion date: January 2012
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

• 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

• Patients with FVII deficiency without any immediate need for treatment will be entered as stand by registered patients with capture of baseline- and demographic data only. Admission data is entered once an event occurs

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Blood Coagulation Disorders
• Congenital Bleeding Disorder
• Congenital FVII Deficiency
• Factor VII Deficiency
• Hemostatic Disorders

Intervention

Drug:
• activated recombinant human factor VII
Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.
• Fresh frozen plasma (Source unspecified)
Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.
• Plasma-derived FVII (LFB)
Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.
• Prothrombin Complex conc. (PCC)
Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.
• Plasma-derived FVII conc. (pdFVII Baxter)
Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.
• Plasma-derived FVII conc. (pdFVII PFL)
Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.

Locations

Country	Name	City	State
France	Novo Nordisk Investigational Site	Paris La défense cedex
Germany	Novo Nordisk Investigational Site	Mainz
Greece	Novo Nordisk Investigational Site	Vouliagment
Hong Kong	Novo Nordisk Investigational Site	Kowloon
India	Novo Nordisk Investigational Site	Bangalore
Iran, Islamic Republic of	Novo Nordisk Investigational Site	Teheran
Israel	Novo Nordisk Investigational Site	Kfar Saba
Italy	Novo Nordisk Investigational Site	Rome
Pakistan	Novo Nordisk Investigational Site	Karachi
Serbia	Novo Nordisk Investigational Site	Belgrade
Slovakia	Novo Nordisk Investigational Site	Bratislava
Spain	Novo Nordisk Investigational Site	Madrid
Thailand	Novo Nordisk Investigational Site	Bangkok
Turkey	Novo Nordisk Investigational Site	Istanbul
United States	Novo Nordisk Investigational Site	Princeton	New Jersey
Venezuela	Novo Nordisk Investigational Site	Caracas

Sponsors (1)

Lead Sponsor	Collaborator
Novo Nordisk A/S

Countries where clinical trial is conducted

United States,  Venezuela,  France,  Germany,  Greece,  Hong Kong,  India,  Iran, Islamic Republic of,  Israel,  Italy,  Pakistan,  Serbia,  Slovakia,  Spain,  Thailand,  Turkey, 

References & Publications (2)

Mariani G, Dolce A, Batorova A, Auerswald G, Schved JF, Siragusa S, Napolitano M, Knudsen JB, Ingerslev J; STER and the International Factor VII Deficiency Study Groups.. Recombinant, activated factor VII for surgery in factor VII deficiency: a prospectiv — View Citation

Mariani G, Dolce A, Napolitano M, Ingerslev J, Giansily-Blaizot M, Di Minno MD, Auerswald G, De Saez AR, Tagliaferri A, Batorova A; STER (Seven Treatment Evaluation Registry).. Invasive procedures and minor surgery in factor VII deficiency. Haemophilia. 2 — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Treatment of bleeding episodes at clinic/hospital: Treatment efficacy evaluation for each treatment modality: excellent, effective, partly effective, ineffective, or not evaluable		Evaluated at 6 hours	No
Primary	Treatment of bleeding episodes at clinic/hospital: Treatment efficacy evaluation for each treatment modality: excellent, effective, partly effective, ineffective, or not evaluable		Evaluated after 30 days	No
Primary	Treatment of bleeding episodes at clinic/hospital: Time to achieve arrest of bleeding		Time to achieve arrest of bleeding	No
Primary	Treatment of bleeding episodes at clinic/hospital: Number of re-bleeding episodes		Within 5 days after first product administration	No
Primary	Treatment of bleeding episodes at home: Treatment efficacy evaluation for each treatment modality: excellent, effective, partly effective, ineffective, or not evaluable		Evaluated at 6 hours	No
Primary	Treatment of bleeding episodes at home: Time to achieve arrest of bleeding		Time to achieve arrest of bleeding	No
Primary	Treatment efficacy (of first and/or second treatment modality) evaluated after surgery: good, partially effective, not evaluable, or ineffective		After surgery	No
Primary	Treatment efficacy (of first and/or second treatment modality) evaluated after surgery: good, partially effective, not evaluable, or ineffective		Evaluated after 30 days	No
Primary	Estimated blood loss volume		During surgery/delivery	No
Primary	Number of red blood cell units administered		During surgery	No
Primary	Number of days spent in hospital		Until last data collection (20 Jan 2012)	No
Primary	Number of re-bleeding episodes (associated with the surgery)		Within 5 days after surgery	No
Primary	Prophylactic treatment efficacy evaluation: excellent, excellent, partially effective, or effective		30 days after first prophylaxis dose	No
Secondary	Number of bleeding episodes during prophylaxis per year		Up to one year	No
Secondary	Number of intensive care unit (ICU) and/or the number of ward days		After first haemostatic product administration until day 30	No
Secondary	Mortality		Within a 30-day (follow-up) period	Yes
Secondary	Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen)		Prior to dosing	No
Secondary	Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen)		After 15 minutes	No
Secondary	Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen)		After 30 days	No
Secondary	Presence of and/or de novo appearance of inhibiting antibodies to FVII		Prior to dosing	Yes
Secondary	Presence of and/or de novo appearance of inhibiting antibodies to FVII		After 30 days	Yes
Secondary	Number of Adverse Events		Until Day 5	No
Secondary	Number of Serious Adverse Events		Until Day 30	No

External Links

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