Actively Recruiting
A Non-randomized, Open-label, Dose-escalation Study to Evaluate Safety and Efficacy of a Single Intravenous Infusion of ZS801 Gene Therapy in Adult Males With Hemophilia B and Low Endogenous Factor IX
Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2025-02-24
21
Participants Needed
1
Research Sites
156 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, kinetics, and effectiveness of a single intravenous infusion of ZS801 in adult males with hemophilia B who have low levels of endogenous factor IX (FIX) 64;2%. Hemophilia B is a genetic disorder that reduces the ability of blood to clot, leading to repeated bleeding events, chronic joint disease, and potential life-threatening complications. ZS801 is a gene therapy using an adeno-associated viral vector to promote the production of human FIX to help improve clotting function. The study includes a dose-escalation phase where 16 participants receive increasing doses of ZS801 every 3 weeks or more between cohorts, with doses ranging from 2.010^12 to 1.010^13 vector genomes per kilogram. Following this, a dose-expansion phase will enroll 5 additional patients to receive the therapy. Treatment consists of a single intravenous infusion of ZS801, with careful monitoring of patients throughout the study period. Participants will undergo screening up to 6 to 8 weeks before receiving the infusion and will be observed for safety and efficacy for 52 weeks after treatment. Assessments include monitoring for adverse events, changes in vital signs and physical exams, laboratory abnormalities, and immune responses to the viral vector. The study also measures factor IX activity and antigen levels, as well as vector shedding. This comprehensive follow-up aims to evaluate how the gene therapy performs and its safety profile over one year.
CONDITIONS
Brief Title
A Study to Evaluate the Safety and Efficacy of ZS801 in Adult Hemophilia B Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male aged 18 years or older
- Confirmed diagnosis of hemophilia B with endogenous factor IX 64;2%
- At least 100 prior exposure days to any recombinant and/or plasma-derived factor IX products
- History of 3 or more bleeding events and/or chronic hemophilia arthritis in one or more joints in the past year requiring factor IX treatment
- Agree to use reliable barrier contraception and avoid sperm donation until 52 weeks after ZS801 administration
- Willing and able to provide informed consent, comply with protocol, and provide biological samples
You will not qualify if you...
- Hypersensitivity to any study drug component or inability to use study drugs including immunosuppressants
- Cannot tolerate immunosuppressants or steroids
- Presence or history of factor IX inhibitor
- History or presence of serious clinical diseases including malignancy, autoimmune disease, severe heart disease, liver disease, active hepatitis B or C infection, poorly controlled diabetes, or uncontrolled blood pressure
- Laboratory abnormalities such as hemoglobin <110 g/L, platelets <100x10^9/L, elevated liver enzymes >2xULN, total bilirubin >1.5xULN, creatinine above normal, low albumin
- HIV or syphilis antibody positive
- High neutralizing antibody titers to AAV5 capsid (>1:5)
- Participation in other gene therapy or clinical trials within specified timeframes
- Planned surgery within 52 weeks after infusion
- Significant recent blood loss (>400 mL within 3 months)
- History of epilepsy, mental illness, drug abuse, or alcoholism
- Poor compliance or expected inability to complete follow-up
- Other significant conditions deemed unsuitable by researchers
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 6 to 8 weeks
Participants are screened for eligibility to participate in the trial.
1 screening and enrollment visit
Duration - Single day infusion with 52 weeks observation
Participants receive a single intravenous infusion of ZS801 gene therapy and are monitored for safety and efficacy.
Regular visits during 52 weeks post-infusion for safety and efficacy assessments
Trial Site Locations
Total: 1 location
1
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, China, 300020
Actively Recruiting
Research Team
Z
Zhang Lei, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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