NCT05630651

The Efficacy and Safety of ZS801 in Chinese Hemophilia B Patients.

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%.

• Status: Recruiting
• Phase: N/A
• Study type: Interventional
• Enrollment: 6 (estimated)
• Sponsor: Institute of Hematology & Blood Diseases Hospital, China · Academic / Other
• Sex: Male
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

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%.

Detailed description

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

Conditions

• Hemophilia B

Interventions

Type	Name	Description
GENETIC	ZS801	A novel, bioengineered adeno-associated viral (AAV) vector carrying human factor IX variant. The dose level is 5.0×10\^12vg/kg.

Timeline

• Start date: 2023-04-19
• Primary completion: 2025-12-01
• Completion: 2028-12-01
• First posted: 2022-11-30
• Last updated: 2025-02-24

Locations

1 site across 1 country: China