Actively Recruiting
Safety and Tolerability of VGB-R04 in Patients With Haemophilia B
Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2025-02-21
3
Participants Needed
1
Research Sites
204 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
An Open-Label, Non-Randomized, uncontrolled, single-dose pilot study of VGB-R04 in subjects with Hemophilia B.
CONDITIONS
Official Title
Safety and Tolerability of VGB-R04 in Patients With Haemophilia B
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male aged 18 to 75 years
- Confirmed diagnosis of Hemophilia B with baseline factor IX activity 2% or less
- At least 100 days of exposure to factor IX treatment
- Currently receiving factor IX prophylaxis therapy or on-demand treatment
- Acceptable laboratory values: hemoglobin ≥110 g/L, platelets ≥100 x10^9 cells/L, AST, ALT, alkaline phosphatase ≤2 times upper limit of normal, bilirubin ≤3 times upper limit of normal, creatinine ≤1.5 times upper limit of normal
- No measurable factor IX inhibitor and no prior history of inhibitors to factor IX protein
- Agree to use reliable barrier contraception until three consecutive samples are negative for vector sequences
- Able to provide informed consent and comply with study requirements
You will not qualify if you...
- Significant underlying liver disease within 6 months prior to or at screening, including portal hypertension, splenomegaly, encephalopathy, reduced serum albumin, or significant liver fibrosis
- Anti-VGB-R04 neutralizing antibody titers of 1:5 or higher
- Severe infectious diseases such as HIV, syphilis, or tuberculosis
- Active hepatitis B virus infection (HBV-DNA >103 IU/ml) or hepatitis C virus infection (HCV antigen and RNA positive)
- Evidence or history of malignant tumors
- History of chronic infection or other chronic diseases posing unacceptable risk
- Any immunodeficiency
- Participation in a gene transfer trial within the last 52 weeks or a clinical trial with investigational drugs within the last 4 weeks
- Use of glucocorticoids, immunosuppressives, or antipsychotics within the last 3 months
- History of hypersensitivity or allergic reaction to any factor IX products or immunoglobulins
- Unable or unwilling to comply with study visit schedule and assessments
- Any other clinically significant major disease or condition making participation unsuitable according to the Investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Blood diseases hospital
Tianjin, Tianjin Municipality, China, 300020
Actively Recruiting
Research Team
L
Lei Zhang, Doctor
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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