Actively Recruiting

Phase 1
Phase 2
Age: 18Years +
MALE
NCT05641610

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

Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2025-02-24

21

Participants Needed

1

Research Sites

354 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

A non-randomized, open-label, dose-escalation, phase I/II study to evaluate the safety, tolerability, kinetics and efficacy of a single intravenous infusion of ZS801 in hemophilia B subjects with endogenous FIX ≤2%.

CONDITIONS

Official Title

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

Who Can Participate

Age: 18Years +
MALE

Eligibility Criteria

Eligible

You may qualify if you...

  • Male aged 18 years or older
  • Confirmed diagnosis of hemophilia B with endogenous FIX level 2% or less
  • At least 100 prior exposure days to recombinant or plasma-derived FIX products
  • At least 3 bleeding events or chronic hemophilic arthritis in the past year requiring FIX treatment
  • Agree to use reliable barrier contraception and avoid sperm donation until 52 weeks after ZS801 administration
  • Willing and able to provide informed consent, understand the study, comply with protocol, and provide biological samples
Not Eligible

You will not qualify if you...

  • Hypersensitivity to any study drug components including immunosuppressants
  • Unable to tolerate immunosuppressants or steroid drugs
  • Presence or history of FIX inhibitor
  • History or presence of malignancy
  • Active autoimmune disease
  • Severe heart disease including angina, myocardial infarction, heart failure, congenital heart disease, valve disease, arrhythmia, or atrioventricular block
  • Underlying or history of liver disease such as portal hypertension, ascites, splenomegaly, esophageal varices, hepatic encephalopathy, or fibrosis
  • Active hepatitis B or C infection or receiving antiviral therapy for these
  • Poorly controlled diabetes mellitus
  • Uncontrolled high or low blood pressure
  • Laboratory abnormalities including hemoglobin <110g/L, platelets <100 x 10^9/L, elevated liver enzymes >2x upper limit, total bilirubin >1.5x upper limit, elevated creatinine, low albumin
  • Positive HIV or syphilis antibody tests
  • AAV5 capsid neutralizing antibody titers greater than 1:5
  • Participation in prior gene therapy trials or use of FIX clinical trial drugs within 1 month or other drug/device trials within 3 months, or plans to join other trials during this study
  • Planned surgery within 52 weeks after infusion
  • Recent blood loss over 400 mL within 3 months before screening
  • History of epilepsy, mental illness, or cognitive incapacity
  • History of drug abuse or alcoholism
  • Expected poor compliance or inability to complete follow-up
  • Any other significant clinical conditions deemed unsuitable by investigators

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, China, 300020

Actively Recruiting

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Research Team

Z

Zhang Lei, 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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