Clinical trial NCT01779921: Treatment of Congenital Factor VII Deficiency
Official title: Treatment of Congenital Factor VII Deficiency. A Prospective Observational Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Congenital Bleeding Disorder
Congenital FVII Deficiency
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2013-01-30
Enrollment: 163
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.


Eligibility 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

Primary outcomes:
measure: Treatment of bleeding episodes at clinic/hospital: Treatment efficacy evaluation for each treatment modality: excellent, effective, partly effective, ineffective, or not evaluable
timeFrame: Evaluated at 6 hours
measure: Treatment of bleeding episodes at clinic/hospital: Treatment efficacy evaluation for each treatment modality: excellent, effective, partly effective, ineffective, or not evaluable
timeFrame: Evaluated after 30 days
measure: Treatment of bleeding episodes at clinic/hospital: Time to achieve arrest of bleeding
timeFrame: Time to achieve arrest of bleeding
measure: Treatment of bleeding episodes at clinic/hospital: Number of re-bleeding episodes
timeFrame: Within 5 days after first product administration
measure: Treatment of bleeding episodes at home: Treatment efficacy evaluation for each treatment modality: excellent, effective, partly effective, ineffective, or not evaluable
timeFrame: Evaluated at 6 hours
measure: Treatment of bleeding episodes at home: Time to achieve arrest of bleeding
timeFrame: Time to achieve arrest of bleeding
measure: Treatment efficacy (of first and/or second treatment modality) evaluated after surgery: good, partially effective, not evaluable, or ineffective
timeFrame: After surgery
measure: Treatment efficacy (of first and/or second treatment modality) evaluated after surgery: good, partially effective, not evaluable, or ineffective
timeFrame: Evaluated after 30 days
measure: Estimated blood loss volume
timeFrame: During surgery/delivery
measure: Number of red blood cell units administered
timeFrame: During surgery
measure: Number of days spent in hospital
timeFrame: Until last data collection (20 Jan 2012)
measure: Number of re-bleeding episodes (associated with the surgery)
timeFrame: Within 5 days after surgery
measure: Prophylactic treatment efficacy evaluation: excellent, excellent, partially effective, or effective
timeFrame: 30 days after first prophylaxis dose
Secondary outcomes:
measure: Number of bleeding episodes during prophylaxis per year
timeFrame: Up to one year
measure: Number of intensive care unit (ICU) and/or the number of ward days
timeFrame: After first haemostatic product administration until day 30
measure: Mortality
timeFrame: Within a 30-day (follow-up) period
measure: Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen)
timeFrame: Prior to dosing
measure: Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen)
timeFrame: After 15 minutes
measure: Changes in laboratory parameters (prothombin time/international normalized ratio, activated partial thromboplastin time, FVII clotting activity, platelet count, fibrinogen)
timeFrame: After 30 days
measure: Presence of and/or de novo appearance of inhibiting antibodies to FVII
timeFrame: Prior to dosing
measure: Presence of and/or de novo appearance of inhibiting antibodies to FVII
timeFrame: After 30 days
measure: Number of Adverse Events
timeFrame: Until Day 5
measure: Number of Serious Adverse Events
timeFrame: Until Day 30
Other outcomes:
Locations:
city: Princeton
country: United States
facility: Novo Nordisk Investigational Site
state: New Jersey
city: Paris La défense cedex
country: France
facility: Novo Nordisk Investigational Site
city: Mainz
country: Germany
facility: Novo Nordisk Investigational Site
city: Vouliagment
country: Greece
facility: Novo Nordisk Investigational Site
city: Kowloon
country: Hong Kong
facility: Novo Nordisk Investigational Site
city: Bangalore
country: India
facility: Novo Nordisk Investigational Site
city: Teheran
country: Iran, Islamic Republic of
facility: Novo Nordisk Investigational Site
city: Kfar Saba
country: Israel
facility: Novo Nordisk Investigational Site
city: Rome
country: Italy
facility: Novo Nordisk Investigational Site
city: Karachi
country: Pakistan
facility: Novo Nordisk Investigational Site
city: Belgrade
country: Serbia
facility: Novo Nordisk Investigational Site
city: Bratislava
country: Slovakia
facility: Novo Nordisk Investigational Site
city: Madrid
country: Spain
facility: Novo Nordisk Investigational Site
city: Bangkok
country: Thailand
facility: Novo Nordisk Investigational Site
city: Istanbul
country: Turkey
facility: Novo Nordisk Investigational Site
city: Caracas
country: Venezuela
facility: Novo Nordisk Investigational Site
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References:
citation: Mariani G, Dolce A, Batorova A, Auerswald G, Schved JF, Siragusa S, Napolitano M, Knudsen JB, Ingerslev J; STER and the International Factor VII Deficiency Study Groups. Recombinant, activated factor VII for surgery in factor VII deficiency: a prospective evaluation - the surgical STER. Br J Haematol. 2011 Feb;152(3):340-6. doi: 10.1111/j.1365-2141.2010.08287.x. Epub 2010 Dec 16.
citation: Mariani G, Dolce A, Napolitano M, Ingerslev J, Giansily-Blaizot M, Di Minno MD, Auerswald G, De Saez AR, Tagliaferri A, Batorova A; STER (Seven Treatment Evaluation Registry). Invasive procedures and minor surgery in factor VII deficiency. Haemophilia. 2012 May;18(3):e63-5. doi: 10.1111/j.1365-2516.2012.02751.x. Epub 2012 Feb 22. No abstract available.