A non-randomized, open-label, dose-escalation, phase I/II study to evaluate the safety, tolerability, kinetics and efficacy of a single intravenous infusion of ZS801 in hemophilia B subjects with endogenous FIX ≤2%.
Male ≥18 years of age;
Confirmed diagnosis of hemophilia B, and endogenous FIX ≤2%;
Have had ≥100 prior exposure days (EDs) to any recombinant and/or plasma-derived FIX protein products;
The subject had at least 3 or more bleeding events and/or chronic hemophilia arthritis in one or more joints in the previous 1 year requiring treatment with FIX;
Hypersensitivity to any component of the study drug (including immunosuppressants) or a condition that can not use;
Inability to tolerate immunosuppressants or steroid drugs;
Have FIX inhibitor as assessed by laboratory, or documented history of FIX inhibitor;
History of malignancy or current presence of any malignancy;
Single group
ZS801
GeneticA novel, bioengineered adeno-associated viral (AAV) vector carrying human factor IX variant. The dose levels are as follows: 2.0×10\^12vg/kg, 5.0×10\^12vg/kg, 1.0×10\^13vg/kg.
Incidence of adverse events
An adverse event (AE) is any medical occurrence, the event will not relate to the treatment.
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.
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.
Number of participants with clinical laboratory abnormalities
Findings were considered to be clinically significant based on the investigator's decision.
Vector-derived FIX activity levels
The vector-derived endogenous FIX activity levels will be measured by One-Stage clot (OS) assay, and characterized by post-treatment population mean and its change from baseline during each visit.
Vector-derived FIX antigen levels
The vector-derived endogenous FIX antigen levels will be characterized by post-treatment population mean, and its change from baseline during each visit.
Vector shedding of ZS801
Blood, saliva, urine and semen will be collected to assess clearance of vector genomes.
Annualized bleeding rate changes from baseline
The number of bleeding episodes per participant will be recorded, and the annualized number of bleeding episodes was calculated.
Annualized FIX consumption changes from baseline
The use of FIX replacement therapy will be recorded by dose (IU/kg) administered, and the annualized use of FIX replacement therapy will be calculated.
Number of target joints
The target joint is a minimum of three bleeds into a single joint within a consecutive 3-month period.
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