Hemophilia A
Conditions
Interventions
Low-dose group:ZS802 injection
High-dose group:ZS802 injection
Dose-extension group:ZS802 injection
Sponsors
Institute of Hematology & Blood Diseases Hospital
Eligibility
Sex/Gender
Male
Age
18 Years to 65 Years
Inclusion criteria
Inclusion criteria: 1. At the time of signing the informed consent form, 18 years old = age = 65 years old, male; 2. Clinical diagnosis of severe hemophilia A, endogenous FVIII activity level <1%, and =3 previous bleeds within 1 year; 3. History of exposure (ED) = 150 days for any recombinant and/or plasma-derived FVIII-containing product; 4. all subjects of childbearing age will voluntarily use effective contraception and refrain from sperm donation from the start of the screening period until 52 weeks after dosing (main study period); 5. Subjects volunteered to participate and were fully informed, fully understood the study and were able to comply with the requirements of the study protocol and had the desire to complete the study as planned, and volunteered to cooperate in providing biological samples for testing as required by the protocol.
Exclusion criteria
Exclusion criteria: 1. Hypersensitivity to any component of the study drug (including immunosuppressants) or a condition that can not use; 2. Inability to tolerate immunosuppressants or steroid drugs; 3. Subjects on long-term steroid therapy, defined as oral corticosteroids for=3 months in the last 12 months; 4. Previous history of FVIII inhibitor or positive FVIII inhibitor during screening; 5. Subjects with a history of serious illness or current serious illness: (1) Have a history of malignant tumors or are currently suffering from any malignant tumors; (2) Patients with active autoimmune diseases;(3) Serious heart disease;(4) Have a history of potentially serious liver disease or liver disease;(5) HBsAg positive or HCVAb positive at the screening stage, or currently receiving hepatitis B or hepatitis C antiviral treatment;(6) Have an acute/chronic infection or other chronic disease, and the investigator determines that participating in the study will increase the risk of the subject;(7) Subjects with diabetes that is difficult to control with medication;(8)High blood pressure that is difficult to control through drug treatment; 6. Subjects with significantly abnormal liver function, or HIV antibody positive or treponema pallidum antibody positive during the screening period; 7. Subjects with AAV5 capsid neutralizing antibody titer levels > 1:5; 8. Subjects who had previously received a clinical trial drug for gene therapy were screened; 9. Subjects scheduled for surgery during the main study period; 10.Those who lost more than 400 mL of blood within 3 months before screening; 11. Those with epilepsy, history of mental illness (such as schizophrenia, depression, mania or anxiety) or obvious mental disorder, incapacitated or incapacitated by other reasons; 12. Patients with a history of drug abuse or alcoholism; 13. Investigators believe that subjects have poor compliance or are expected to be less likely to complete follow-up; 14. There are clinically significant diseases or other reasons that the researcher and/or collaborators consider unsuitable to participate in this researcher.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Adverse event; | — |
Secondary
| Measure | Time frame |
|---|---|
| AAV neutralizing antibody;AAV Binding Antibody;FVIII activity;Annualized bleeding rate;Vector shedding;FVIII antigen;FVIII inhibitor;T cell response; | — |
Countries
CHINA
Contacts
Public ContactLei Zhang
Institute of Hematology & Blood Diseases Hospital
Outcome results
None listed