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.

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

163

participants targeted

Target at P50-P75 for all trials

Timeline

Completed

Started Oct 2005

Longer than P75 for all trials

Geographic Reach

16 countries

16 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

6.3 years study duration

Study Start

First participant enrolled

October 1, 2005

Completed

6.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 1, 2012

Completed

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2012

Completed

1 year until next milestone

First Submitted

Initial submission to the registry

January 15, 2013

Completed

15 days until next milestone

First Posted

Study publicly available on registry

January 30, 2013

Completed

Last Updated

January 12, 2017

Status Verified

January 1, 2017

Enrollment Period

6.3 years

First QC Date

January 15, 2013

Last Update Submit

January 11, 2017

Conditions

Congenital Bleeding Disorder Congenital FVII Deficiency

Outcome Measures

Primary Outcomes (13)

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 Outcomes (10)

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
+5 more secondary outcomes

Study Arms (1)

FVII

Drug: activated recombinant human factor VIIDrug: 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)

Interventions

activated recombinant human factor VIIDRUG

Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.

FVII

Fresh frozen plasma (Source unspecified)DRUG

Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.

FVII

Plasma-derived FVII (LFB)DRUG

Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.

FVII

Prothrombin Complex conc. (PCC)DRUG

Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.

FVII

Plasma-derived FVII conc. (pdFVII Baxter)DRUG

Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.

FVII

Plasma-derived FVII conc. (pdFVII PFL)DRUG

Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.

FVII

Eligibility Criteria

Sexall

Healthy VolunteersNo

Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

Sampling MethodNon-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.

You may qualify if:

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

Contact the study team to confirm eligibility.