Treatment of Congenital Factor VII Deficiency

Completed

• Conditions: Congenital FVII Deficiency; Congenital Bleeding Disorder
• Interventions: 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)

• Registration Number: NCT01779921
• Lead Sponsor: Novo Nordisk A/S

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 Description

Not available

Study Timeline

• Study Start: 2005-10
• Primary Completion: 2012-01
• Study Completion: 2012-01
• Study First Submitted: 2013-01-15
• Study First Submitted QC: 2013-01-29
• Study First Posted: 2013-01-30
• Status Verified: 2017-01
• Last Update Submitted: 2017-01-11
• Last Update Posted: 2017-01-12

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 163

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

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Exclusion Criteria

Not provided

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
FVII	Plasma-derived FVII conc. (pdFVII Baxter)	-
FVII	activated recombinant human factor VII	-
FVII	Plasma-derived FVII (LFB)	-
FVII	Plasma-derived FVII conc. (pdFVII PFL)	-
FVII	Fresh frozen plasma (Source unspecified)	-
FVII	Prothrombin Complex conc. (PCC)	-

Primary Outcome Measures

Name	Time	Method
Number of re-bleeding episodes (associated with the surgery)	Within 5 days after surgery
Treatment of bleeding episodes at clinic/hospital: Time to achieve arrest of bleeding	Time to achieve arrest of bleeding
Treatment of bleeding episodes at home: Time to achieve arrest of bleeding	Time to achieve arrest of bleeding
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 efficacy (of first and/or second treatment modality) evaluated after surgery: good, partially effective, not evaluable, or ineffective	Evaluated after 30 days
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
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)
Prophylactic treatment efficacy evaluation: excellent, excellent, partially effective, or effective	30 days after first prophylaxis dose

Secondary Outcome Measures

Name	Time	Method
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)	After 30 days
Presence of and/or de novo appearance of inhibiting antibodies to FVII	After 30 days
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
Number of Adverse Events	Until Day 5
Number of Serious Adverse Events	Until Day 30

Trial Locations

Locations (1)

Novo Nordisk Investigational Site; 🇻🇪 Caracas, Venezuela