Clinical trial NCT01862367: Use of rFXIII in Treatment of Congenital FXIII Deficiency, a Prospective Multi-centre Observational Study
Official title: Use of rFXIII in Treatment of Congenital FXIII Deficiency, a Prospective Multi-centre Observational Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Congenital Bleeding Disorder
Congenital FXIII Deficiency
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2013-05-24
Enrollment: 30
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: This observational study will enroll patients with congenital FXIII A-subunit deficiency for whom the decision to treat with rFXIII has been made and who are willing to provide informed consent (or patient's legally acceptable representative (LAR) consent, if applicable).

Brief summary: This study is conducted globally. The aim of this observational study is to investigate the incidence of specific adverse drug reactions associated with the use of recombinant factor XIII (NovoThirteen®) in patients with congenital FXIII A-subunit deficiency (congenital FXIII deficiency), comprising FXIII antibodies, allergic reactions, embolic and thrombotic events and lack of therapeutic effect.

The study will aim at observing all patients exposed to NovoThirteen® in the EU, and additional patients from selected non-EU countries. Recombinant FXIII (rFXIII) is registered in EU and Switzerland as NovoThirteen® and in Canada as Tretten®.


Eligibility criteria: Inclusion Criteria:

* Informed consent obtained before any study-related activities. (Study-related activities are any procedure related to recording of data according to the protocol)
* Able and willing to provide signed informed consent (or patient's legally acceptable representative (LAR) consent, if applicable), as required by local ethics committee, governmental or regulatory authorities
* Congenital FXIII A-subunit deficiency
* Actual or planned exposure to rFXIII

Primary outcomes:
measure: Adverse drug reactions in patients with congenital FXIII A-subunit deficiency treated with rFXIII,comprising FXIII antibodies, allergic reactions, embolic and thrombotic events and lack of effect collected
timeFrame: During study period up to 6 years
Secondary outcomes:
measure: All serious adverse events collected
timeFrame: During study period up to 6 years
measure: All medical events of special interest collected
timeFrame: During study period up to 6 years
measure: All medication errors and near medication errors collected
timeFrame: During study period up to 6 years
measure: Use of rFXIII in patients with congenital FXIII A-subunit deficiency also for other uses than for prophylactic treatment collected
timeFrame: During study period up to 6 years
measure: Frequency of bleeding episodes collected
timeFrame: During study period up to 6 years
Other outcomes:
Locations:
city: Orange
country: United States
facility: Novo Nordisk Investigational Site
state: California
city: Tampa
country: United States
facility: Novo Nordisk Investigational Site
state: Florida
city: Atlanta
country: United States
facility: Novo Nordisk Investigational Site
state: Georgia
city: Detroit
country: United States
facility: Novo Nordisk Investigational Site
state: Michigan
city: Minneapolis
country: United States
facility: Novo Nordisk Investigational Site
state: Minnesota
city: Cleveland
country: United States
facility: Novo Nordisk Investigational Site
state: Ohio
city: Columbus
country: United States
facility: Novo Nordisk Investigational Site
state: Ohio
city: St. John's
country: Canada
facility: Novo Nordisk Investigational Site
state: Newfoundland and Labrador
city: Toronto
country: Canada
facility: Novo Nordisk Investigational Site
state: Ontario
city: Debrecen
country: Hungary
facility: Novo Nordisk Investigational Site
city: Chieti
country: Italy
facility: Novo Nordisk Investigational Site
city: Genova
country: Italy
facility: Novo Nordisk Investigational Site
city: Barcelona
country: Spain
facility: Novo Nordisk Investigational Site
city: Madrid
country: Spain
facility: Novo Nordisk Investigational Site
city: Málaga
country: Spain
facility: Novo Nordisk Investigational Site
city: Tortosa
country: Spain
facility: Novo Nordisk Investigational Site
city: Aberdeen
country: United Kingdom
facility: Novo Nordisk Investigational Site
Study officials:
affiliation: Novo Nordisk A/S
name: Global Clinical Registry (GCR, 1452)
role: STUDY_DIRECTOR
References: