Fabry disease
Conditions
Interventions
Sponsors
West China Hospital, Sichuan University
Eligibility
Sex/Gender
All
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Age >=18 at the time of signing the informed consent. 2. Clinically diagnosed with Fabry disease. 3. AAV capsid neutralizing antibody negative. 4. Agree to use effective contraception after entering the screening period until 52 weeks after administration. 5. Subjects voluntarily participate and are fully informed, fully understand the research and can comply with the requirements of the research protocol, are willing to complete the research as planned, and voluntarily cooperate with the provision of biological samples for testing.
Exclusion criteria
Exclusion criteria: 1. Hypersensitivity to any component of the study drug or a condition that can not use. 2. Subjects on long-term steroid therapy. 3. Have a history of malignant tumors or currently suffer from any malignant tumors. 4. Active autoimmune disease. 5. Have a history of severe heart disease or surgery. 6. Have severe liver disease or a history of liver disease. 7. The subjects have received kidney transplantation or are undergoing hemodialysis or peritoneal dialysis. 8. Uncontrolled hypertension or diabetes at screening period. 9. Plan to participate in other clinical trials during the study period. 10. Have received live attenuated vaccine within 12 weeks prior to screening or plan to receive live attenuated vaccine in the study. 11. Have a history of epilepsy, mental illness or obvious mental disorders. 12. Patients with a history of drug abuse or drug abuse or alcoholism. 13. Patients with poor compliance and low likelihood of expected completion of treatment and follow-up. 14. There is a clinically significant illness or other cause for which the investigator and/or sponsor deems it inappropriate to participate in the Investigator.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety;Tolerability; | — |
Secondary
| Measure | Time frame |
|---|---|
| PK;Immunogenicity;Efficacy; | — |
Countries
China
Contacts
Public ContactYucheng Chen
West China Hospital, Sichuan University
Outcome results
None listed