Fabry disease
Conditions
Interventions
Sponsors
West China Hospital,Sichuan University
Eligibility
Sex/Gender
Male
Age
18 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Males=18 years old at the time of signing the informed consent; 2. Clinically diagnosed with classical Fabry disease; 3. Agree to use reliable barrier contraception and prohibition of sperm donation until 52 weeks after the administration of ZS805; 4. 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 (including immunosuppressants) or a condition that can not use; 2.Inability to tolerate immunosuppressants or steroid drugs; 3.Those who used enzyme replacement therapy (ERT) within 3 months before screening; 4.Subjects on long-term steroid therapy; 5.Subjects with positive anti-a-gal A antibody; 6.Subjects with a history of serious illness or current serious illness; 7.Subjects with significantly abnormal liver function, or HIV antibody positive or treponema pallidum antibody positive during the screening period; 8.Subjects with AAV5 capsid neutralizing antibody titer levels > 1:5; 9.Subjects who had previously received a clinical trial drug for gene therapy were screened; 10.Subjects scheduled for surgery during the main study period; 11.Subjects who had received a live attenuated vaccine within 12 weeks before screening, or were scheduled to receive a live attenuated vaccine during the course of the study; 12.Those who lost more than 400 mL of blood within 3 months before screening; 13.Those with epilepsy, history of mental illness (such as schizophrenia, depression, mania or anxiety) or obvious mental disorder, incapacitated or incapacitated by other reasons; 14.Patients with a history of drug abuse or alcoholism; 15.Investigators believe that subjects have poor compliance or are expected to be less likely to complete follow-up; 16.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;Vital signs;Physical examination;Laboratory examination; | — |
Secondary
| Measure | Time frame |
|---|---|
| Vector shedding;AAV Neutralizing Antibody;AAV Binding Antibody; | — |
Countries
CHINA
Contacts
Public ContactYucheng Chen
West China Hospital,Sichuan University
Outcome results
None listed