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RECRUITING
NCT06238908
EARLY_PHASE1

Safety and Efficacy Study of NGGT003 in Hemophilia A Patients

Sponsor: Institute of Hematology & Blood Diseases Hospital, China

View on ClinicalTrials.gov

Summary

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.

Official title: Clinical Study on the Safety and Efficacy of an Intravenous Infusion of NGGT003 in the Treatment of Hemophilia A

Key Details

Gender

MALE

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

6

Start Date

2024-01-17

Completion Date

2030-01-31

Last Updated

2025-02-21

Healthy Volunteers

No

Conditions

Interventions

DRUG

NGGT003

Single intravenous infusion of NGGT003 at low dose (4e11vg/kg), medium dose (1e12vg/kg) and high dose (2.5e12vg/kg)

Locations (1)

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, China