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

Gene Therapy Trial VGB-R04 for Hemophilia B Patients in China

Trial ID: NCT05152732
Study Title: Safety and Tolerability of VGB-R04 in Patients With Haemophilia B
Sponsor: Institute of Hematology & Blood Diseases Hospital, China
Principal Investigator: Dr. Lei Zhang

🔬 Overview

This pilot gene therapy study is evaluating VGB-R04, a novel adeno-associated virus (AAV) vector carrying a high-specific activity Factor IX variant for treating Hemophilia B. Hemophilia B patients typically require lifelong FIX infusions to prevent bleeding events. This study explores a one-time gene infusion aimed at long-term FIX expression and clinical benefit.

🎯 Objectives

Primary Endpoints:

• Incidence of adverse events (AEs) and serious adverse events (SAEs)

• Clinical significance of changes in vital signs, lab results, and physical findings

Secondary Endpoints:

• FIX:C activity and antigen levels post-treatment

• Changes in annualized bleeding rate and FIX usage

• Reduction in target joints and evaluation of gene vector shedding

🧪 Study Design

• Type: Interventional (Gene Therapy)

• Phase: Early Phase I

• Model: Single Group Assignment (Open-Label)

• Enrollment: Estimated 3 participants

Treatment:
A single intravenous infusion of VGB-R04 with a 52-week safety observation period. Patients are encouraged to join an extension study for long-term safety monitoring up to 5 years.

Study Period:

• Start: December 28, 2021

• Estimated Primary Completion: March 2025

• Estimated Study Completion: December 2025

👨‍⚕️ Eligibility Criteria

Inclusion:

• Males aged 18–75 with confirmed Hemophilia B (FIX ≤2%)

• ≥100 exposure days to FIX therapy

• No history of FIX inhibitors

• Stable liver and kidney function, no HIV, HBV, HCV, or cancer

Exclusion:

• Underlying liver conditions or active viral infections

• High neutralizing antibodies to VGB-R04

• Recent participation in gene therapy or clinical trials

• Use of immunosuppressants in the past 3 months

📍 Trial Location

Blood Diseases Hospital, Tianjin, China
📧 Contact: Dr. Lei Zhang — info@cancerfax.com
📞 +86 182 1759 2149

🔄 Data Sharing & IPD

The investigators intend to share Individual Participant Data (IPD) with other researchers once VGB-R04 is approved, including study protocols and analysis plans.

✍️ Connect with us

This gene therapy breakthrough offers Hemophilia B patients the possibility of long-term relief from bleeding and the burdens of regular treatment. If you or a loved one is eligible, this trial may be a gateway to lasting freedom from FIX infusions.

📩 Reach out to CancerFax.com for expert assistance connecting with this and other cutting-edge trials in China.