A Study to Evaluate the Safety and Efficacy of ZS801 in Adult Hemophilia B Patients

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: info@cancerfax.com
📞 +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.