Safety and Efficacy Study of NGGT003 in Hemophilia A Patients
Status:
RECRUITING
Trial end date:
2030-01-31
Target enrollment:
Participant gender:
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.
Phase:
EARLY_PHASE1
Details
Lead Sponsor:
Institute of Hematology & Blood Diseases Hospital, China