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drugset / Trial / NCT05641610

A Study to Evaluate the Safety and Efficacy of ZS801 in Adult Hemophilia B Patients

NCT05641610 ↗

Phase 1/2 Recruiting 21 enrolled Institute of Hematology & Blood Diseases Hospital, China
NaSingle-groupOpen-labelTreatment

Summary

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%.

Timeline

Start
2022-02-16
Primary completion
2025-12
Completion
2028-12

Drugs

EvaluationDrugModalityDoseRoute
Subject ZS801 Gene therapy (AAV / viral vector) 1 vg/kg Intravenous
Subject ZS801 Gene therapy (AAV / viral vector) 2 vg/kg Intravenous
Subject ZS801 Gene therapy (AAV / viral vector) 5 vg/kg Intravenous

Indications

hemophilia B