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

NCTID	NCT05641610 (View at clinicaltrials.gov)
Description	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%. (Show More)
Development Status	Active
Indication	Hemophilia B
Disease Ontology Term	DOID:12259
Compound Name	ZS801
Sponsor	Institute of Hematology & Blood Diseases Hospital, China
Funder Type	Other
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	21 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	F9
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV
Editor Type	none
Dose 1	2.0E12 vg/kg
Dose 2	5.0E12 vg/kg
Dose 3	1.0E13 vg/kg
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China