Gene Therapy Clinical Trial for Hemophilia B: ZS801 in China
Trial ID: NCT05641610
Study Title: A Non-Randomized, Open-Label, Dose-Escalation, Phase I/II Study to Evaluate the Safety, Tolerability, Kinetics and Efficacy of ZS801 in Hemophilia B Patients
Sponsor: Institute of Hematology & Blood Diseases Hospital, China
Principal Investigator: Dr. Lei Zhang
🔬 Overview
This ongoing gene therapy trial in Tianjin, China, is evaluating ZS801, a novel adeno-associated virus (AAV)-based gene therapy for adult patients with Hemophilia B, a rare genetic bleeding disorder caused by a deficiency of clotting Factor IX (FIX).
ZS801 is designed to deliver the human FIX gene into the liver through a single intravenous infusion, potentially enabling the body to produce FIX naturally.
🎯 Objectives
Primary Endpoints:
- Assess the safety profile and adverse events
- Monitor changes in vital signs, lab abnormalities, and immune responses to AAV capsid
Secondary Endpoints:
- Measure FIX activity and antigen levels
- Evaluate gene vector shedding (in blood, saliva, urine, semen)
- Track changes in annual bleeding rate and FIX usage
🧪 Study Design
- Type: Interventional (Gene Therapy)
- Phases: Phase I/II (Dose Escalation & Expansion)
- Model: Single Group Assignment (Open Label)
- Enrollment: Estimated 21 participants
Dosage Levels:
- 2.0×10¹² vg/kg
- 5.0×10¹² vg/kg
- 1.0×10¹³ vg/kg
Duration:
- Start: February 16, 2022
- Estimated Primary Completion: December 2025
- Final Study Completion: December 2028
👨⚕️ Eligibility Criteria
Inclusion:
- Males ≥18 years with FIX activity ≤2%
- ≥100 prior exposure days to FIX products
- ≥3 bleeding episodes or hemophilic arthropathy in past year
Exclusion:
- FIX inhibitors, liver disease, active hepatitis, uncontrolled chronic conditions
- Positive HIV/Treponema antibodies or prior gene therapy
- Participation in other clinical trials within 3 months
- Planned surgery or mental health conditions
📍 Trial Site
Institute of Hematology & Blood Diseases Hospital, Tianjin, China
📧 Contact: [email protected]
📞 +86 022-23909240
🔄 Data Sharing & IPD
The study will share individual participant data (IPD) with other researchers after regulatory approval of ZS801.
✍️ Connect with us
ZS801 represents a potential paradigm shift in the treatment of Hemophilia B—aiming to reduce or eliminate the need for frequent FIX infusions. If you or a loved one meets the eligibility criteria, this trial could be life-changing.
📩 Reach out to CancerFax.com to connect with this pioneering gene therapy study in China.
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About Dr. Nishant Mittal
Dr. Nishant Mittal is a highly accomplished researcher with over 13 years of experience in the fields of cardiovascular biology and cancer research. Significant contributions to stem cell biology, developmental biology, and innovative research techniques mark his career. Research Highlights Dr. Mittal's research has focused on several key areas: 1) Cardio…
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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.