A non-randomized, open-label study to evaluate the safety, kinetics and efficacy of a single intravenous infusion of ZS801 in hemophilia B subjects with endogenous FIX ≤2%.
This study will seek to determine the safety, kinetics and efficacy of a single IV infusion of ZS801. The dose level is 5.0×10\^12vg/kg; Dose addition may occur based on the safety and FIX activity on steady state. Subjects will provide informed consent and then undergo screening assessments up to 6 weeks prior administration of ZS801. All subjects will undergo 52 weeks safety and efficacy observation. Then subjects
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
6
A novel, bioengineered adeno-associated viral (AAV) vector carrying human factor IX variant. The dose level is 5.0×10\^12vg/kg.
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, China
RECRUITINGIncidence of adverse events
An adverse event (AE) is any medical occurrence, the event will not relate to the treatment.
Time frame: Baseline up to Week 52
Number of participants with clinically significant change from baseline in vital signs
Vital signs will be obtained with participants in the seated position, after having sat calmly for at least 5 minutes. The clinical significance of vital signs will be determined at the investigator's discretion.
Time frame: Baseline up to Week 52
Number of participants with clinically significant change from baseline in physical examination findings
Findings will be considered to be clinically significant based on the investigator's decision.
Time frame: Time Frame: Baseline up to Week 52
Number of participants with clinical laboratory abnormalities
Findings were considered to be clinically significant based on the investigator's decision.
Time frame: Baseline up to Week 52
Antibody against AAV capsid protein
Immune response against AAV capsid will be evaluated by measurement of the binding antibody and neutralizing antibody against AAV capsid protein in plasma samples.
Time frame: Baseline up to Week 52
Vector-derived FIX:C activity levels
The vector-derived endogenous FIX:C activity levels will be characterized by post-treatment population mean, and its change from baseline during each visit.
Time frame: Baseline up to Week 52
Vector-derived FIX antigen levels
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
The vector-derived endogenous FIX antigen levels will be characterized by post-treatment population mean, and its change from baseline during each visit.
Time frame: Baseline up to Week 52
Vector shedding of ZS801
Blood, saliva, urine and semen will be collected to assess clearance of vector genomes.
Time frame: Baseline up to Week 52