In this article
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
π¬ 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.
π― 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
π§ͺ 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
π¨ββοΈ Eligibility Criteria
Inclusion:
- Males aged 18β75 with confirmed Hemophilia B (FIX β€2%)
- β₯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
π Trial Location
Blood Diseases Hospital, Tianjin, China
π§ Contact: Dr. Lei Zhang β [email protected]
π +86 182 1759 2149
π 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.
βοΈ 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.
π© Reach out to CancerFax.com for expert assistance connecting with this and other cutting-edge trials in China.
How CancerFax Helps
CancerFax is a specialist cancer access and patient-navigation platform. We help patients and families understand their options, organise medical records, coordinate hospital communication, and support cross-border treatment planning where appropriate.
We help collect and organise reports, scans, pathology, biomarker results, and treatment history for structured case review.
We communicate with hospitals or trial teams to assess whether a case may be suitable for further screening.
We support appointment coordination, document submission, translation, and direct communication with international departments.
For international patients, we help with practical coordination β travel planning, hospital admission guidance, and local support.
If this option is not suitable, we help explore other relevant treatments, clinical trials, or advanced care pathways.
From inquiry through to follow-up, our coordinators provide a single point of contact for the family.
CancerFax does not guarantee treatment access, eligibility, or clinical outcome. Our role is to help patients access accurate information, structured review, and appropriate specialist pathways.
About Sai Sree
β Reviewed for medical accuracy by the CancerFax review panel.
Medical Disclaimer
This article is for educational purposes only and should not replace medical advice, diagnosis, or treatment from a qualified oncology specialist. Every patient's case is different. Treatment decisions should always be made after a review of complete medical records by the treating medical team.
Treatment availability, eligibility, timelines, and access can change. Any clinical trial participation depends on detailed review and approval by the trial hospital or investigator.