Congenital fibrinogen deficiency
Conditions
Interventions
Experimental group:Human fibrinogen
Sponsors
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
Eligibility
Sex/Gender
All
Age
12 Years to 65 Years
Inclusion criteria
Inclusion criteria: 1. 12 to 65 years of age ( 18 to 65 years old and without bleeding symptoms before administration for patients participants in the pharmacokinetic study), male or female; 2. Clinical diagnosis of congenital fibrinogen deficiency (family history or evidence of genetic testing), and fibrinogen <1.0g/L; 3. All subjects of reproductive age must take effective contraception from screening period until 3 months after the completion of the study; 4. Voluntarily and signs the informed consent form(ICF), if neither the subject nor the legal guardian can read and understand the ICF, it should be signed jointly with a third-party witness; ICF must be signed by the subject and his legal guardian or his legal representative for patients of 12 to less than 18 years old.
Exclusion criteria
Exclusion criteria: 1. Previous or current allergy to human fibrinogen or excipients (sodium citrate, sodium chloride, arginine hydrochloride) or other human plasma proteins; 2. Acquired fibrinogen deficiency; 3. Received treatment with human fibrinogen or blood products containing human fibrinogen within 4 weeks prior to enrollment; 4.Deep vein thrombosis or pulmonary embolism or arterial embolism within 1 year before signing the informed consent; 5. Severe heart disease, including myocardial infarction, chronic cardiac insufficiency (NYHA grades III and IV); 6. Liver and kidney function reached one of the following indicators: a. glutamate aminotransferase (ALT) or aspartate aminotransferase (AST) > 2 times the upper limit of normal range; b. Alkaline phosphatase or total bilirubin > 2 times the upper limit of normal range; c. Urea nitrogen or blood creatinine > 1.5 times the upper limit of normal range; 7. FDP > 1.5 times the upper limit of normal range; 8. Positive of HBsAg (or nucleic acid test) or HCV antibody (or nucleic acid test) or HIV antibody (or nucleic acid test) or treponema pallidum antibody; 9. Pregnant or lactating female or female of childbearing age or male subjects of childbearing age who do not wish to use contraception during the study period; 10. Patients who cannot be interrupted or need to use anticoagulant drugs during the trial; 11. Participated in other drug or medical device clinical investigators within 1 month before signing the informed consent; 12. Patients with mental illness, obvious mental disorders or epilepsy; Patients with acute medical conditions; Patients with current or previous malignant neoplasms; Persons with no behavioral or cognitive capacity; 13. History of esophageal varices bleeding; History of acute bleeding; Patients with end-stage liver disease; 14. Multiple injuries occurred within 1 year before signing the informed consent; 15. Alcoholic (drinking =2 times per week within 3 months before signing the ICF, and the average amount of each drink = 100ml of pure alcohol after conversion of different alcoholic drinks), drug addicts, drug abusers and addicts; Participants in the pharmacokinetic study smoked more than 5 cigarettes per day in the 3 months prior to signing the informed consent or during the trial period; 16. Patients with poor compliance or any condition deemed unsuitable for inclusion by other investigators (such as a projected survival of less than 3 months).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Recovery of fibrinogen activity at 1.5 hours after the starting of first infusion; | — |
Secondary
| Measure | Time frame |
|---|---|
| Changes of thrombin time (TT) at 1.5h and 24h after the start of first infusion compared with baseline.;Recovery of fibrinogen activity at 24 hours after the starting of first infusion;Evaluation of hemostasis after treatment for each bleeding event at 24 hours after the starting of infusion;Pharmacokinetics parameters; | — |
Countries
China
Contacts
Public ContactZhang Lei
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
Outcome results
None listed