Safety and Tolerability of VGB-R04 in Patients With Haemophilia B

This is an interventional treatment trial for Hemophilia B focused on measuring Hemophilia, AAV, safety, gene therapy Read More

Inclusion Criteria:

1. Male ≥18 years and ≤75years of age;
2. Confirmed diagnosis of hemophilia B (baseline FIX activity ≤ 2% of normal or documented history of FIX activity ≤2%);
3. At least 100 days exposure history to FIX;
4. Currently receiving FIX Prophylaxis therapy or on-demand treatment to prevent bleeding;

5. Have acceptable laboratory values:

   1. Hemoglobin ≥110 g/L;
   2. Platelets ≥100×10'9 cells/L;
   3. AST, ALT, alkaline phosphatase ≤2×upper limit of normal (ULN) at the testing laboratory;
   4. Bilirubin ≤3× ULN ;
   5. Creatinine ≤1.5× ULN.
6. No measurable factor IX inhibitor as assessed by the central laboratory and have no prior history of inhibitors to factor IX protein;
7. Agree to use reliable barrier contraception until 3 consecutive samples are negative for vector sequences;
8. Able to provide informed consent and comply with the requirements of the study.

Exclusion Criteria:

1. Have significant underlying liver disease within the past 6 months prior to or at Screening, including but not limited to:

   1. Preexisting diagnosis of portal hypertension;
   2. Splenomegaly;
   3. Encephalopathy;
   4. Reduction of serum albumin;
   5. Evidence of significant liver fibrosis;
2. Have anti-VGB-R04 neutralizing antibody titers ≥1:5;
3. Evidence of severe infection disease, i.e., human immunodeficiency virus (HIV) infection, syphilis, tuberculosis, etc.;
4. Evidence of active hepatitis B virus infection (HBV-DNA >103 IU/ml) or hepatitis C virus infection (HCV antigen and HCV-RNA positive);
5. Evidence of malignant tumours or those with a previous history of malignant tumours;
6. Have a history of chronic infection or other chronic diseases that the Investigator considers to constitute an unacceptable risk;
7. Any immunodeficiency;
8. Participated in a gene transfer trial within the last 52 weeks or in a clinical trial with an investigational drug within the last 4 weeks;
9. Have used glucocorticoids, immunosuppressive drugs, or antipsychotics within the last 3 months;
10. Previous history of hypersensitivity or allergic reaction to any FIX products or any immunoglobulin;
11. Unable or unwilling to comply with the schedule of visits and study assessments described in the clinical protocol;
12. Any concurrent clinically significant major disease or any other condition that, in the opinion of the Investigator, makes the subject unsuitable for participation in the study.