Status:
UNKNOWN
A Study to Evaluate the Safety and Efficacy of VGB-R04 in Adult Hemophilia B Patients
Lead Sponsor:
Shanghai Vitalgen BioPharma Co., Ltd.
Conditions:
Hemophilia B
Eligibility:
MALE
18-65 years
Phase:
PHASE1
PHASE2
Brief Summary
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.
Detailed Description
Hemophilia B is a genetic bleeding disorder caused by pathogenic variants (eg, mutations, deletion) in the FIX gene. HB patients have frequent and potentially life-threatening bleeding and often devel...
Eligibility Criteria
Inclusion
- 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;
Exclusion
- Have significant underlying liver disease within the past 6 months prior to or at Screening, including but not limited to:
- Preexisting diagnosis of portal hypertension;
- Splenomegaly;
- Encephalopathy;
- Reduction of serum albumin;
- Evidence of significant liver fibrosis;
- Have anti-VGB-R04 neutralizing antibody titers ≥1:5;
- Evidence of severe infection disease, i.e., human immunodeficiency virus (HIV) infection, syphilis, tuberculosis, etc.;
- Novel coronavirus infection occurred in the 6 weeks prior to entry into the group
- Evidence of active hepatitis B virus infection (HBsAg positive) or hepatitis C virus infection (HCV-RNA positive);
- Evidence of malignant tumours or those with a previous history of malignant tumours;
- Have a history of chronic infection or other chronic diseases that the Investigator considers to constitute an unacceptable risk;
- Any immunodeficiency;
- planned surgery may be required within one year;
- Past thromboembolic events (arterial or venous thromboembolic events);
- Hypertensive patients with poor blood pressure control (systolic blood pressure ≥150 mmHg or diastolic blood pressure ≥90mmHg after antihypertensive drug treatment);
Key Trial Info
Start Date :
July 1 2022
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
January 1 2025
Estimated Enrollment :
26 Patients enrolled
Trial Details
Trial ID
NCT05441553
Start Date
July 1 2022
End Date
January 1 2025
Last Update
July 1 2022
Active Locations (1)
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1
Shanghai Vitalgen Biopharma Co.,Ltd.
Shanghai, China