Hemophilia A Clinical Trial
Official title:
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.
Hemophilia A (HA) is an X-linked recessive genetic disease caused by mutations in the FVIII gene on the X chromosome, leading to abnormal coagulation function. In the male population, the incidence rate of hemophilia A was about 1/5000, and female patients with hemophilia A were extremely rare. Type A hemophilia patients mainly exhibit a tendency for bleeding, with a wide range of bleeding sites and frequent recurrence, which can form hematoma and joint deformation. This is an early phase 1, open-label, single-center, dose-escalation pilot trial to evaluate the safety and efficacy of a single intravenous infusion of NGGT003 in hemophilia A patients. 4-6 subjects will be enrolled and divided into 3 groups according to the principle of dose escalation, respectively administered intravenous infusion of NGGT003 at low dose (4e11vg/kg), medium dose (1e12vg/kg) and high dose (2.5e12vg/kg). All subjects will undergo 52 weeks of treatment observation and further 260 weeks of long-term follow-up. ;
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