Hemophilia B
Conditions
Interventions
Experimental group:ANB-002
Sponsors
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College.
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Sign the informed consent form for participating in the research; 2. Men diagnosed with hemophilia B who were aged 18 or above at the time of informed consent; 3. Diagnosis of hemophilia B: · FIX activity =0.6 BU, it was considered to be present), and there was no related medical history; 5. FIX concentrate treatment for at least 100 exposure days; 6. For patients receiving FIX concentrate as preventive treatment, there is no need to increase the dose or frequency of FIX concentrate within 6 months prior to signing the informed consent form, except for intermittent dose increases before elective medical procedures and other events with bleeding risks. For patients receiving on-demand treatment, there is a history of bleeding (three or more spontaneous bleeding episodes) within 52 weeks before signing the informed consent form; 7. The subjects and their fertile sexual partners are willing to use reliable contraceptive methods until three consecutive sperm samples without DNA vectors are obtained. This requirement does not apply to patients undergoing surgical sterilization; 8. Fertile subjects refused to donate sperm until three consecutive sperm samples without DNA vectors were obtained; 9. The researchers believe that the subjects have the ability to follow the program procedures.
Exclusion criteria
Exclusion criteria: 1. A history of the use of any gene therapy drugs; 2. There are other blood or hematopoietic system diseases other than hemophilia B; 3. Anti-aav5 antibodies were detected by ELISA. If the titer of the anti-AAV5 antibody of the subjects is found to be greater than 1:5 during the screening, the detection of the anti-AAV5 antibody titer can be repeated. After repeated tests, subjects with an anti-AAV5 antibody titer of =35 kg/m2; 7. History of arterial or venous thrombosis events (any venous thrombosis, pulmonary thromboembolism, acute myocardial infarction, etc.); 8. Diagnosed with HIV infection. If the following conditions are met, patients with HIV-1 and HIV-2 antibodies who receive antiretroviral therapy can be included in the study: · CD4+ cell count > 200 cells /mm3, · Undetected viral load ( 1.5×ULN; Platelet count 1.5×ULN; · Total bilirubin > 1.5×ULN; · INR>=1.4; 14. Relevant liver diseases or conditions that may be symptoms of existing liver diseases, including but not limited to: · Liver cirrhosis; Based on the results of liver biopsy or non-invasive examination methods, subjects with grade 3-4 fibrosis evaluated according to the METAVIR scale (or equivalent grades if other scales are used) can be included in Gilbert syndrome or cholelithiasis; 15. Lymphoproliferative disorders or malignant tumors with a remission period of less than 5 years, except for cured cases of basal cell carcinoma; 16. History of malignant liver tumors; 17. Diseases that require regular use of systemic glucocorticoids. 18. Immunosuppressants (except glucocorticoids) were used within 30 days before administration of ANB-002; 19. A major operation is planned to be performed within 52 weeks after the administration of ANB-002; 20. Current signs of drug addiction or alcohol dependence, psychoactive substances or drug abuse (or their medical history), and alcohol/drug addiction or abuse are considered by researchers to be contraindications for the treatment of hemophilia B or may limit patients' treatment compliance; 21. Participate in other clinical studies simultaneously, and have participated in other clinical studies within 3 months before signing the informed consent form, and have participated in this study in the past. Exceptional case: The subjects withdrew from this study during the screening; 22. The last administration of any investigational/investigational drug within one month or five half-lives (whichever is longer) prior to the signing of the informed consent form.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The change of FIX activity from baseline in Week 52 (before the administration of the study drug ANB-002);Proportion of subjects with adverse reactions; | — |
Secondary
| Measure | Time frame |
|---|---|
| The changes in FIX activity at all planned evaluation time points compared to the baseline; | — |
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