A Phase 1/2 Study to Evaluate the Safety and Efficacy of VGB-R04 in Adult Hemophilia B Patients
A multicenter, open, non-randomized, phase I/II, two-phase clinical study. The dose exploration phase was phase I, and the dose extension phase was phase II.
• Male ≥18 years and ≤65years of age;
• Confirmed diagnosis of hemophilia B (baseline FIX activity ≤ 2% of normal);
• At least 100 days exposure history to FIX;
• Currently receiving FIX Prophylaxis therapy or on-demand treatment to prevent bleeding;
• Have acceptable laboratory values:
• Hemoglobin ≥110 g/L;
• Platelets ≥100×109 /L;
• AST, ALT, alkaline phosphatase ≤2×upper limit of normal (ULN) at the testing laboratory;
• Bilirubin ≤3× ULN ;
• Creatinine ≤1.5× ULN.
• No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein;
• Agree to use reliable contraception until 2 consecutive samples are negative for vector sequences;