Treatment of Congenital Factor VII Deficiency

Study Overview
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Brief Summary
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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.

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

Conditions
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Congenital Bleeding Disorder, Congenital FVII Deficiency

Intervention / Treatment
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* 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)


Participation Criteria
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Eligibility Criteria
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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
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| FVII<br> | Drug: activated recombinant human factor VII<br>* Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.<br>Drug: Fresh frozen plasma (Source unspecified)<br>* Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.<br>Drug: Plasma-derived FVII (LFB)<br>* Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.<br>Drug: Prothrombin Complex conc. (PCC)<br>* Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.<br>Drug: Plasma-derived FVII conc. (pdFVII Baxter)<br>* Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.<br>Drug: Plasma-derived FVII conc. (pdFVII PFL)<br>* Treatment of bleeding episodes and treatment during surgery and prophylaxis as per discretion of the investigator.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 
| 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 |  | Prior to dosing | 
| Presence of and/or de novo appearance of inhibiting antibodies to FVII |  | After 30 days | 
| Number of Adverse Events |  | Until Day 5 | 
| Number of Serious Adverse Events |  | Until Day 30 |