Clinical Study on the Safety and Efficacy of an Intravenous Infusion of NGGT003 in the Treatment of Hemophilia A
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.
• Voluntarily sign the informed consent form;
• Male, age ≥18 years old;
• Diagnosed with hemophilia A according to the Guidelines for Diagnosis and Treatment of Hemophilia A (2022 Edition), and the endogenous FVIII activity level was \<1 IU/dL (\<1%);
• The exposure days (EDs) of treatment with any recombinant or plasma-derived FVIII product were ≥150 days;
• Anti-AAV neutralizing antibody titer ≤1:5, binding antibody titer ≤1:100;
• Bleeding events and/or FVIII product injections have occurred within 12 weeks before screening;
• No history of allergy to FVIII products;
• FVIII inhibitor titer﹤0.6BU/mL;
• Commitment to use other drugs during the study requires the consent of the investigator;
⁃ Willing and able to comply with study procedures and requirements;
⁃ Willing to use effective contraceptive methods within 52 weeks after administration.