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Treatment of Congenital Factor VII Deficiency (F7CONDEF)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S
ClinicalTrials.gov Identifier:
NCT01779921
First received: January 15, 2013
Last updated: January 11, 2017
Last verified: January 2017
  Purpose

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.


Condition Intervention
Congenital Bleeding Disorder
Congenital FVII Deficiency
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
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 Novo Nordisk A/S:

Primary Outcome Measures:
  • Treatment of bleeding episodes at clinic/hospital: Treatment efficacy evaluation for each treatment modality: excellent, effective, partly effective, ineffective, or not evaluable [ Time Frame: 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 [ Time Frame: Evaluated after 30 days ]
  • Treatment of bleeding episodes at clinic/hospital: Time to achieve arrest of bleeding [ Time Frame: Time to achieve arrest of bleeding ]
  • Treatment of bleeding episodes at clinic/hospital: Number of re-bleeding episodes [ Time Frame: 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 [ Time Frame: Evaluated at 6 hours ]
  • Treatment of bleeding episodes at home: Time to achieve arrest of bleeding [ Time Frame: 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 [ Time Frame: After surgery ]
  • Treatment efficacy (of first and/or second treatment modality) evaluated after surgery: good, partially effective, not evaluable, or ineffective [ Time Frame: Evaluated after 30 days ]
  • Estimated blood loss volume [ Time Frame: During surgery/delivery ]
  • Number of red blood cell units administered [ Time Frame: During surgery ]
  • Number of days spent in hospital [ Time Frame: Until last data collection (20 Jan 2012) ]
  • Number of re-bleeding episodes (associated with the surgery) [ Time Frame: Within 5 days after surgery ]
  • Prophylactic treatment efficacy evaluation: excellent, excellent, partially effective, or effective [ Time Frame: 30 days after first prophylaxis dose ]

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

Biospecimen Retention:   Samples Without DNA
Plasma for assaying of inhibiting antibodies to FVII. All samples, which are analysed at the local hospital laboratory, are to be stored and destroyed according to local rules. Inhibitor samples analysed at the central laboratory will be stored until data validation has taken place at the end of the study after which the samples will be destroyed.

Enrollment: 163
Study Start Date: October 2005
Study Completion Date: January 2012
Primary Completion Date: January 2012 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
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.

  Eligibility

Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.
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
  • 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
  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: NCT01779921

Locations
United States, New Jersey
Novo Nordisk Investigational Site
Princeton, New Jersey, United States, 08540
France
Novo Nordisk Investigational Site
Paris La défense cedex, France, 92932
Germany
Novo Nordisk Investigational Site
Mainz, Germany, 55127
Greece
Novo Nordisk Investigational Site
Vouliagment, Greece, 16671
Hong Kong
Novo Nordisk Investigational Site
Kowloon, Hong Kong
India
Novo Nordisk Investigational Site
Bangalore, India, 560001
Iran, Islamic Republic of
Novo Nordisk Investigational Site
Teheran, Iran, Islamic Republic of
Israel
Novo Nordisk Investigational Site
Kfar Saba, Israel, 44425
Italy
Novo Nordisk Investigational Site
Rome, Italy, 00144
Pakistan
Novo Nordisk Investigational Site
Karachi, Pakistan
Serbia
Novo Nordisk Investigational Site
Belgrade, Serbia, 11070
Slovakia
Novo Nordisk Investigational Site
Bratislava, Slovakia, 811 05
Spain
Novo Nordisk Investigational Site
Madrid, Spain, 28033
Thailand
Novo Nordisk Investigational Site
Bangkok, Thailand, 10500
Turkey
Novo Nordisk Investigational Site
Istanbul, Turkey, 34335
Venezuela
Novo Nordisk Investigational Site
Caracas, Venezuela
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
Study Director: Global Clinical Registry (GCR, 1452) Novo Nordisk A/S
  More Information

Additional Information:
Publications:
Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT01779921     History of Changes
Other Study ID Numbers: F7HAEM-3578
Study First Received: January 15, 2013
Last Updated: January 11, 2017

Additional relevant MeSH terms:
Blood Coagulation Disorders
Hemostatic Disorders
Factor VII Deficiency
Hematologic Diseases
Vascular Diseases
Cardiovascular Diseases
Hemorrhagic Disorders
Blood Coagulation Disorders, Inherited
Coagulation Protein Disorders
Genetic Diseases, Inborn
Thrombin
Hemostatics
Coagulants

ClinicalTrials.gov processed this record on March 23, 2017