A fibrinogen concentrate study in paediatric patients with inherited fibrinogen deficiency

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 16

Inclusion Criteria

1. Signed and dated informed consent form by parents or a legal representative
2. Age 12 years old
3. Patients with inherited afibrinogenaemia (fibrinogen antigen levels 0.2 g/L and unclottable PT, aPTT and TT) or severe hypofibrinogenaemia defined as fibrinogen antigen levels 0.5 g/L and activity to antigen ratio ˜ 1
4. Negative results on HCG-based pregnancy test at the selection visit, before PK/PD part and before each event to be treated for females of childbearing potential (presence of menstruation)
5. Covered by healthcare insurance in accordance with local requirements

Are the trial subjects under 18? yes
Number of subjects for this age range: 12
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Dysfibrinogenaemia
2. Acquired fibrinogen deficiency
3. Suspected present or past anticoagulation inhibitor
4. Personal history of venous or arterial thrombosis or thromboembolic event
5. Co-morbidity with other/unrelated coagulopathies
6. Administration of any fibrinogen concentrate or fibrinogen containing blood product during the last 15 days (applicable only if fibrinogen levels to be measured at inclusion)
7. Platelet count less than 100 x 109/L
8. Serum creatinine > 2 times the upper limit of normal
9. AST and / or ALT > 5 times the upper limit of normal
10. Known history of hypersensitivity or other severe reaction to any component of the investigational medicinal product
11. Any other condition which, in the opinion of the investigator, could interfere with the study results or would not be in the best interest of the patient.
12. Participation in another drug clinical trial at the same time or within the previous 30 days
13. Anticipated poor compliance with study procedures
14. Legal incapacity or limited capacity
15. Permanent treatment with antithrombotic or anti-platelet agents such as heparins, anti-IIa or anti-Xa agents, aspirin, clopidogrel and NSAIDs.
Additional exclusion criteria specific to PK/PD Part
1. Patients with hypofibrinogenaemia defined as fibrinogen antigen levels 0.5 g/L and activity to antigen ratio ˜ 1
2. Significant bleeding(s) within 7 days prior to the start of the PK/PD part that could have an impact on the patient's plasma volume
3. Administration of any fibrinogen concentrate or fibrinogen containing blood product during the last 15 days prior to the start of the PK/PD part
4. Treatment with antithrombotic or anti-platelet agents such as heparins, anti-IIa or anti-Xa agents, aspirin, clopidogrel and NSAIDs within 10 days prior to the start of the PK/PD part.
5. Elective surgery within 14 days after administration of FGTW for PK/PD part

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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