Afibrinogenemia, Congenital, Hypofibrinogenemia, Congenital
Conditions
Brief summary
The aim of the study is to evaluate clinical pharmacology, efficacy and safety of FGTW in pediatric patients with congenital fibrinogen deficiency.
Interventions
DRUGbiological: human fibrinogen concentrate
Sponsors
Laboratoire français de Fractionnement et de Biotechnologies
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
No minimum to 12 Years
Healthy volunteers
No
Inclusion criteria
* Signed and dated informed consent form by parents or a legal representative * Age less or equal to 12 years old * Patients with inherited afibrinogenemia or severe inherited hypofibrinogenemia * Negative results on HCG-based pregnancy test for females of childbearing potential (presence of menstruation)
Exclusion criteria
* Dysfibrinogenemia * Acquired fibrinogen deficiency * Suspected present or past anticoagulation inhibitor * Personal history of venous or arterial thrombosis or thromboembolic event * Co-morbidity with other/unrelated coagulopathies * Administration of any fibrinogen concentrate or fibrinogen containing blood product during the last 15 days * Permanent treatment with antithrombotic or anti-platelet agents such as heparins, anti-IIa or anti-Xa agents, aspirin, clopidogrel and NSAIDs.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Investigator's overall assessment of efficacy of FGTW on hemostasis using a 4-point scale at the end of each bleeding or surgical episode. | 6 hours or up to 5 days |
| Terminal half life for Fibrinogen antigen and activity | 5 days |
Secondary
| Measure | Time frame |
|---|---|
| Adverse Events | Participants will be followed for the duration of their participation in the study, an expected average of 1 year |
Countries
France, Lebanon, Morocco, Turkey (Türkiye)
Outcome results
None listed