Congenital Bleeding Disorder, Congenital FXIII Deficiency
Conditions
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®.
Interventions
DRUGcatridecacog
No treatment given. All patients enrolled in this observational study will receive their medication through usual commercial channels.
Sponsors
Novo Nordisk A/S
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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 | During study period up to 6 years |
Secondary
| Measure | Time frame |
|---|---|
| All serious adverse events collected | During study period up to 6 years |
| All medical events of special interest collected | During study period up to 6 years |
| All medication errors and near medication errors collected | During study period up to 6 years |
| Use of rFXIII in patients with congenital FXIII A-subunit deficiency also for other uses than for prophylactic treatment collected | During study period up to 6 years |
| Frequency of bleeding episodes collected | During study period up to 6 years |
Countries
Canada, Hungary, Italy, Spain, United Kingdom, United States
Outcome results
None listed