Congenital hemophilia
Conditions
Interventions
Test group:Recombinant Human Coagulation Factor ?a for Injection
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 years = 5Bu/ml; (2) screening period F?/F? inhibitor level = 0.6Bu/ml, but with a high response to injectable coagulation factor VIII or IX (i.e., patients with a have a history of positive F?/F? inhibitor and an inhibitor level >= 5Bu/ml after reinfusion of F?/F?). 3. Subjects participating in the pharmacokinetic trial must have no significant active bleeding prior to the first dose; 4. All subjects of childbearing age will voluntarily use effective contraception from the time of entry into the Screening Period until 3 months after the last dose of the trial; 5. Subjects and/or guardians fully understand and are able to comply with the requirements of the trial protocol and have the willingness to complete the study as planned, and voluntarily cooperate in providing biological samples for testing in accordance with the requirements of the protocol; 6. Able to understand the procedures and methods of this clinical trial, after fully informed consent, the subject voluntarily participates in the study and the informed consent form is signed by the subject himself/herself and/or his/her guardian.
Exclusion criteria
Exclusion criteria: 1. those with a known history of hypersensitivity to recombinant human coagulation factor VIIa preparation and any of its components, or with a known history of hypersensitivity to mouse-derived proteins; 2. Screening period F? inhibitor positivity or history of F? inhibitor positivity; 3. Platelet count = 2.5 times the upper limit of normal (ULN) or clinical renal function tests (blood creatinine [Cr]) >= 1.5 times the upper limit of normal (ULN); 5. Those who are severely anemic and require blood transfusion; 6. Persons with a clinical diagnosis of hepatitis C or persons with AIDS; 7. Persons with significant abnormalities in coagulation indices due to other disease causes (e.g. disseminated intravascular coagulopathy or platelet disorders, etc.), except for hemophilia A or B; 8. Those with severe heart disease, including myocardial infarction and chronic cardiac insufficiency (NYHA class III and IV); 9. those who have a history of thrombosis such as arterial or deep vein thrombosis, pulmonary embolism, a history of pulmonary edema or a history of disseminated intravascular coagulation within 1 year of signing the informed consent form 10. those who have had intracranial hemorrhage in the past; 11. having received any product (plasma-derived or recombinant) containing F? or F?a within 48 hours prior to the first dose; 12. have received any product containing F? (plasma source or recombinant) within 72 hours prior to the first dose or any product containing FIX (plasma source or recombinant) within 96 hours prior to the first dose; 13. Any anticoagulants, antifibrinolytics, or medications affecting platelet function, including non-steroidal anti-inflammatory drugs (NSAIDs) such as aspirin, that have been used within 1 week prior to the first dose or that are required during PK blood collection; 14. Those who have received immunomodulators (e.g., gammaglobulin, alpha-interferon, and prednisone >10 mg/d [and >7 days] or similar medications, except antiretroviral medications, within 2 weeks prior to the first dose; 15. Those who received whole blood or plasma within 2 weeks prior to first dose; 16. who have had a highly invasive surgical procedure (e.g., orthopedic surgery, abdominal surgery) within 1 month prior to the first dose, except for non-invasive or minimally invasive procedures; 17. those who have participated in other clinical trials within 1 month prior to the first administration of the drug 18. Those with a history of drug abuse or alcoholism (Alcoholism Criteria: a history of long-term alcohol consumption for more than 5 years, with a converted ethanol amount >= 40g/d for men and >= 20g/d for women, or a history of heavy alcohol consumption within 2 weeks, with a converted ethanol amount >80g/d. The formula for converting the amount of ethanol (g) = the amount of alcohol consumed (mL)*the amount of ethanol content (%) × 0.8); 19. People with mental illness, significant mental disorders, other causes of incapacitation or cognitive incapacity, including those who, in the opinion of the investigator, have poor compliance and will not be able to evaluate the efficacy of the treatment or who are expected to have a low likelihood of completing the treatment course and follow-up visits; 20. history of miscarriage or pregnancy termination within 3 months prior to signing the informed consent form, pregnant women and la
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of all bleeding events successfully stopped after treatment; | — |
Secondary
| Measure | Time frame |
|---|---|
| Proportion of all bleeding episodes treated with "excellent", "good" and "partially effective" hemostatic results;Infusion efficiency values at 10 min post-dose;Changes in coagulation indices (PT, APTT) at 10 min after drug administration;Number of injections per bleeding event for each bleeding event, dose per injection, total dose administered per bleeding event;Time to "good" or "excellent" response of the subject after treatment of a hemorrhagic event;Pharmacokinetics parameters; | — |
Countries
China
Contacts
Public ContactRenchi Yang
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
Outcome results
None listed