Actively Recruiting
The Efficacy and Safety of ZS801 in Chinese Hemophilia B Patients.
Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2025-02-24
6
Participants Needed
1
Research Sites
293 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
A non-randomized, open-label study to evaluate the safety, kinetics and efficacy of a single intravenous infusion of ZS801 in hemophilia B subjects with endogenous FIX ≤2%.
CONDITIONS
Official Title
The Efficacy and Safety of ZS801 in Chinese Hemophilia B Patients.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male aged between 18 and 65 years
- Confirmed diagnosis of hemophilia B with endogenous factor IX level less than or equal to 2%
- At least 100 prior exposure days to any recombinant and/or plasma-derived factor IX products
- History of three or more bleeding events or chronic hemophilia arthritis in one or more joints in the past year requiring treatment with factor IX agents
- Agree to use reliable barrier contraception and not donate sperm until 52 weeks after receiving ZS801
- Willing to participate voluntarily, understand the study, comply with protocol requirements, complete the study as planned, and provide biological samples for testing
You will not qualify if you...
- Hypersensitivity to any component of the study drug or immunosuppressants, or conditions preventing their use
- Inability to tolerate immunosuppressants or steroid drugs
- Presence or history of factor IX inhibitor as confirmed by laboratory or documented history
- History or current serious clinical diseases including malignancy, active autoimmune disease, severe heart disease, underlying or history of liver disease, hepatitis B or C infection or treatment, poorly controlled diabetes, uncontrolled blood pressure
- Laboratory values outside specified limits: hemoglobin below 110 g/L, platelets below 100 x10^9/L, liver enzymes greater than twice upper limit of normal, total bilirubin above 1.5 times upper limit of normal, creatinine above normal limit, albumin below lower limit of normal, positive HIV or Treponema pallidum antibody
- Neutralizing antibody titers against AAV5 capsid above 1:640
- Prior gene therapy trials before screening, use of factor IX clinical trial drugs within 1 month, participation in other clinical trials within 3 months, or planned participation during this study
- Planned surgery within 52 weeks after infusion
- Loss of more than 400 mL of blood within 3 months before screening
- History of epilepsy, mental illness, or obvious mental disorder or incapacitation
- History of drug abuse or alcoholism
- Anticipated poor compliance or unlikely to complete follow-up as judged by investigators
- Other clinically significant diseases or reasons judged unsuitable by researchers
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, China, 300020
Actively Recruiting
Research Team
L
Lei Zhang, MD
CONTACT
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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