Clinical Trial: Treatment of Congenital Factor VII Deficiency

Study Status: Completed
Recruit Status: Completed
Study Type: Observational

Official Title: Treatment of Congenital Factor VII Deficiency. A Prospective Observational Study

Brief 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.


Detailed Summary:
Sponsor: Novo Nordisk A/S

Current Primary Outcome:

  • 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&

    Original Primary Outcome: Same as current

    Current Secondary Outcome:

    • 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 ]


    Original Secondary Outcome: Same as current

    Information By: Novo Nordisk A/S

    Dates:
    Date Received: January 15, 2013
    Date Started: October 2005
    Date Completion:
    Last Updated: January 11, 2017
    Last Verified: January 2017