Safety and Tolerability of VGB-R04 in Patients With Haemophilia B
An Open-Label, Non-Randomized, uncontrolled, single-dose pilot study of VGB-R04 in subjects with Hemophilia B.
• Male ≥18 years and ≤75years of age;
• Confirmed diagnosis of hemophilia B (baseline FIX activity ≤ 2% of normal or documented history of FIX activity ≤2%);
• 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 ≥100×10'9 cells/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 barrier contraception until 3 consecutive samples are negative for vector sequences;
• Able to provide informed consent and comply with the requirements of the study.