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: August 6, 2014
Last verified: August 2014
  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 ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • Treatment of bleeding episodes at clinic/hospital: Time to achieve arrest of bleeding [ Time Frame: Time to achieve arrest of bleeding ] [ Designated as safety issue: No ]
  • Treatment of bleeding episodes at clinic/hospital: Number of re-bleeding episodes [ Time Frame: Within 5 days after first product administration ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • Treatment of bleeding episodes at home: Time to achieve arrest of bleeding [ Time Frame: Time to achieve arrest of bleeding ] [ Designated as safety issue: No ]
  • Treatment efficacy (of first and/or second treatment modality) evaluated after surgery: good, partially effective, not evaluable, or ineffective [ Time Frame: After surgery ] [ Designated as safety issue: No ]
  • 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 ] [ Designated as safety issue: No ]
  • Estimated blood loss volume [ Time Frame: During surgery/delivery ] [ Designated as safety issue: No ]
  • Number of red blood cell units administered [ Time Frame: During surgery ] [ Designated as safety issue: No ]
  • Number of days spent in hospital [ Time Frame: Until last data collection (20 Jan 2012) ] [ Designated as safety issue: No ]
  • Number of re-bleeding episodes (associated with the surgery) [ Time Frame: Within 5 days after surgery ] [ Designated as safety issue: No ]
  • Prophylactic treatment efficacy evaluation: excellent, excellent, partially effective, or effective [ Time Frame: 30 days after first prophylaxis dose ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Number of bleeding episodes during prophylaxis per year [ Time Frame: Up to one year ] [ Designated as safety issue: No ]
  • Number of intensive care unit (ICU) and/or the number of ward days [ Time Frame: After first haemostatic product administration until day 30 ] [ Designated as safety issue: No ]
  • Mortality [ Time Frame: Within a 30-day (follow-up) period ] [ Designated as safety issue: Yes ]
  • Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen) [ Time Frame: Prior to dosing ] [ Designated as safety issue: No ]
  • Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen) [ Time Frame: After 15 minutes ] [ Designated as safety issue: No ]
  • Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen) [ Time Frame: After 30 days ] [ Designated as safety issue: No ]
  • Presence of and/or de novo appearance of inhibiting antibodies to FVII [ Time Frame: Prior to dosing ] [ Designated as safety issue: Yes ]
  • Presence of and/or de novo appearance of inhibiting antibodies to FVII [ Time Frame: After 30 days ] [ Designated as safety issue: Yes ]
  • Number of Adverse Events [ Time Frame: Until Day 5 ] [ Designated as safety issue: No ]
  • Number of Serious Adverse Events [ Time Frame: Until Day 30 ] [ Designated as safety issue: No ]

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:   up to 90 Years
Genders Eligible for Study:   Both
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 Clinical Trial Call Center
Princeton, New Jersey, United States, 08540
France
Paris La défense cedex, France, 92932
Germany
Mainz, Germany, 55127
Greece
Vouliagment, Greece, 16671
Hong Kong
Kowloon, Hong Kong
India
Bangalore, India, 560001
Iran, Islamic Republic of
Teheran, Iran, Islamic Republic of
Israel
Kfar Saba, Israel, 44425
Italy
Rome, Italy, 00144
Pakistan
Karachi, Pakistan
Serbia
Belgrade, Serbia, 11070
Slovakia
Bratislava, Slovakia, 811 05
Spain
Madrid, Spain, 28033
Thailand
Bangkok, Thailand, 10500
Turkey
Istanbul, Turkey, 34335
Venezuela
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:
No publications provided

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: August 6, 2014
Health Authority: France: Ministry of Health
Germany: Paul-Ehrlich-Institut
Greece: National Organization of Medicines
Hong Kong: Department of Health
India: Drugs Controller General of India
Iran: Ministry of Health
Israel: Israeli Health Ministry Pharmaceutical Administration
Italy: The Italian Medicines Agency
Pakistan: Ministry of Health
Serbia: Agency for Drugs and Medicinal Devices
Slovakia: State Institute for Drug Control
Spain: Spanish Agency of Medicines
Thailand: Food and Drug Administration
Turkey: Ministry of Health
United States: Food and Drug Administration
Venezuela: Ministry of Health and Social Development

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

ClinicalTrials.gov processed this record on October 21, 2014