Treatment of Congenital Factor VII Deficiency (F7CONDEF)

Treatment of Congenital Factor VII Deficiency. A Prospective Observational Study

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.

Study Overview

• Status: Completed
• Conditions: Congenital Bleeding Disorder; Congenital FVII Deficiency
• Intervention / Treatment: Drug: activated recombinant human factor VII; Drug: Fresh frozen plasma (Source unspecified); Drug: Plasma-derived FVII (LFB); Drug: Prothrombin Complex conc. (PCC); Drug: Plasma-derived FVII conc. (pdFVII Baxter); Drug: Plasma-derived FVII conc. (pdFVII PFL)

• Study Type: Observational
• Enrollment (Actual): 163

Contacts and Locations

Study Locations

• France
  • Paris La défense cedex, France, 92932
    • Novo Nordisk Investigational Site
• Germany
  • Mainz, Germany, 55127
    • Novo Nordisk Investigational Site
• Greece
  • Vouliagment, Greece, 16671
    • Novo Nordisk Investigational Site
• Hong Kong
  • Kowloon, Hong Kong
    • Novo Nordisk Investigational Site
• India
  • Bangalore, India, 560001
    • Novo Nordisk Investigational Site
• Iran, Islamic Republic of
  • Teheran, Iran, Islamic Republic of
    • Novo Nordisk Investigational Site
• Israel
  • Kfar Saba, Israel, 44425
    • Novo Nordisk Investigational Site
• Italy
  • Rome, Italy, 00144
    • Novo Nordisk Investigational Site
• Pakistan
  • Karachi, Pakistan
    • Novo Nordisk Investigational Site
• Serbia
  • Belgrade, Serbia, 11070
    • Novo Nordisk Investigational Site
• Slovakia
  • Bratislava, Slovakia, 811 05
    • Novo Nordisk Investigational Site
• Spain
  • Madrid, Spain, 28033
    • Novo Nordisk Investigational Site
• Thailand
  • Bangkok, Thailand, 10500
    • Novo Nordisk Investigational Site
• Turkey
  • Istanbul, Turkey, 34335
    • Novo Nordisk Investigational Site
• United States
  • New Jersey
    • Princeton, New Jersey, United States, 08540
      • Novo Nordisk Investigational Site
• Venezuela
  • Caracas, Venezuela
    • Novo Nordisk Investigational Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: ADULT; OLDER_ADULT; CHILD
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• Sampling Method: Non-Probability Sample

Study Population

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.

Description

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 Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

FVII

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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
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
Treatment of bleeding episodes at clinic/hospital: Time to achieve arrest of bleeding	Time to achieve arrest of bleeding
Treatment of bleeding episodes at clinic/hospital: Number of re-bleeding episodes	Within 5 days after first product administration
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
Treatment of bleeding episodes at home: Time to achieve arrest of bleeding	Time to achieve arrest of bleeding
Treatment efficacy (of first and/or second treatment modality) evaluated after surgery: good, partially effective, not evaluable, or ineffective	After surgery
Treatment efficacy (of first and/or second treatment modality) evaluated after surgery: good, partially effective, not evaluable, or ineffective	Evaluated after 30 days
Estimated blood loss volume	During surgery/delivery
Number of red blood cell units administered	During surgery
Number of days spent in hospital	Until last data collection (20 Jan 2012)
Number of re-bleeding episodes (associated with the surgery)	Within 5 days after surgery
Prophylactic treatment efficacy evaluation: excellent, excellent, partially effective, or effective	30 days after first prophylaxis dose

Secondary Outcome Measures

Outcome Measure	Time Frame
Number of bleeding episodes during prophylaxis per year	Up to one year
Number of intensive care unit (ICU) and/or the number of ward days	After first haemostatic product administration until day 30
Mortality	Within a 30-day (follow-up) period
Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen)	Prior to dosing
Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen)	After 15 minutes
Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen)	After 30 days
Presence of and/or de novo appearance of inhibiting antibodies to FVII	Prior to dosing
Presence of and/or de novo appearance of inhibiting antibodies to FVII	After 30 days
Number of Adverse Events	Until Day 5
Number of Serious Adverse Events	Until Day 30

Collaborators and Investigators

• Sponsor: Novo Nordisk A/S

Publications and helpful links

General Publications

• 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 prospective evaluation - the surgical STER. Br J Haematol. 2011 Feb;152(3):340-6. doi: 10.1111/j.1365-2141.2010.08287.x. Epub 2010 Dec 16.
• 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. 2012 May;18(3):e63-5. doi: 10.1111/j.1365-2516.2012.02751.x. Epub 2012 Feb 22. No abstract available.

Helpful Links

• Clinical Trials at Novo Nordisk

Study record dates

Study Major Dates

• Study Start: October 1, 2005
• Primary Completion (Actual): January 1, 2012
• Study Completion (Actual): January 1, 2012

Study Registration Dates

• First Submitted: January 15, 2013
• First Submitted That Met QC Criteria: January 29, 2013
• First Posted (Estimate): January 30, 2013

Study Record Updates

• Last Update Posted (Estimate): January 12, 2017
• Last Update Submitted That Met QC Criteria: January 11, 2017
• Last Verified: January 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Hemostatic Disorders; Blood Coagulation Disorders; Factor VII Deficiency; Hemostatics; Coagulants; Thrombin
• Other Study ID Numbers: F7HAEM-3578