Safety and Efficacy Study of NGGT003 in Hemophilia A Patients

NCTID	NCT06238908 (View at clinicaltrials.gov)
Description	This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of an intravenous infusion of NGGT003 in hemophilia A patients. NGGT003 uses adeno-associated virus (AAV) as a vector, carrying a liver specific promoter and codon optimized human FVIII gene B domain deletion mutant (hFVIII BDD), and expresses human FVIII protein in the liver through intravenous injection. (Show More)
Development Status	Active
Indication	Hemophilia A
Disease Ontology Term	DOID:12134
Compound Name	NGGT003
Sponsor	Institute of Hematology & Blood Diseases Hospital, China
Funder Type	Other
Recruitment Status	Recruiting
Enrollment Count	6
Results Posted	Not Available

Target Gene/Variant	F8
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	AAV8
Editor Type	none
Dose 1	4E11 vg/kg
Dose 2	1E12 vg/kg
Dose 3	2.5E12 vg/kg
Dose 4
Dose 5

No.of Trial Sites	1
Locations	China