Clinical Trials in China / NCT05641610
Recruiting
Phase 1/2
A Study to Evaluate the Safety and Efficacy of ZS801 in Adult Hemophilia B Patients
NCT05641610 · tracked via the Priya Life Science China tracker
Condition(s) studied
Hemophilia B
Investigational drug(s) / intervention(s)
ZS801
ZS801: A novel, bioengineered adeno-associated viral (AAV) vector carrying human factor IX variant. The dose levels are as follows: 2.0×10\^12vg/kg, 5.0×10\^12vg/kg, 1.0×10\^13vg/kg.
Study summary
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%.
Eligibility
Inclusion Criteria:
1. Male ≥18 years of age;
2. Confirmed diagnosis of hemophilia B, and endogenous FIX ≤2%;
3. Have had ≥100 prior exposure days (EDs) to any recombinant and/or plasma-derived FIX protein products;
4. The subject had at least 3 or more bleeding events and/or chronic hemophilia arthritis in one or more joints in the previous 1 year requiring treatment with FIX;
5. Agree to use reliable barrier contraception and prohibition of sperm donation until 52 weeks after the administration of ZS801.
6. Subjects voluntarily participate and are fully informed, fully understand the research and can comply with the requirements of the research protocol, are willing to complete the research as planned, and voluntarily cooperate with the provision of biological samples for testing.
Exclusion Criteria:
1. Hypersensitivity to any component of the study drug (including immunosuppressants) or a condition that can not use;
2. Inability to tolerate immunosuppressants or steroid drugs;
3. Have FIX inhibitor as assessed by laboratory, or documented history of FIX inhibitor;
4. Who have a history or are currently suffering from any of the following serious clinical diseases:
1. History of malignancy or current presence of any malignancy;
2. Have active autoimmune disease;
3. Severe heart disease, including angina pectoris, myocardial infarction, heart failure, clinically significant congenital heart disease, heart valve disease, arrhythmia and atrioventricular block, etc.;
4. Have underlying liver disease or history of liver disease (such as portal hypertension, ascites, splenomegaly, esophageal varices, hepatic encephalopathy or hepatic fibrosis);
5. Have active hepatitis B infection (HBsAg positive) or active hepatitis C infection (HCVAb positive), or are currently receiving hepatitis B or hepatitis C antiviral therapy;
6. Diabetes mellitus that is poorly controlled after drug treatment;
7. Uncontrolled hypertension or hypotension;
5. laboratory values:
1. Hemoglobin\<110g/L;
2. Platelets\<100×10\^9/L;
3. AST, ALT, alkaline phosphatase\>2×ULN;
4. Total bilirubin\>1.5×ULN;
5. Creatinine\>ULN;
6. Albumin\<LLN;
7. HIV antibody positive or Treponema pallidum antibody positive.
6. Have AAV5 capsid neutralizing antibody titers \>1:5;
7. Those who have received clinical trials of gene therapy before screening, or have used FIX clinical trial drugs within 1 month, or participated in other drug/device clinical trials within 3 months, or plan to participate in other clinical trials during this study;
8. Those who have planned surgery within 52 weeks after the infusion;
9. Those who lost more than 400 mL of blood within 3 months before screening;
10. Those with epilepsy, history of mental illness (such as schizophrenia, depression, mania or anxiety) or obvious mental disorder, incapacitated or incapacitated by other reasons;
11. Patients with a history of drug abuse or alcoholism;
12. Investigators believe that subjects have poor compliance or are expected to be less likely to complete follow-up;
13. There are clinically significant diseases or other reasons that the researcher and/or collaborators consider unsuitable to participate in this researcher.
Primary outcome measure(s)
- Incidence of adverse events — Baseline up to Week 52
An adverse event (AE) is any medical occurrence, the event will not relate to the treatment. - Number of participants with clinically significant change from baseline in vital signs — Baseline up to Week 52
Vital signs will be obtained with participants in the seated position, after having sat calmly for at least 5 minutes. The clinical significance of vital signs will be determined at the investigator's discretion. - Number of participants with clinically significant change from baseline in physical examination findings — Time Frame: Baseline up to Week 52
Findings will be considered to be clinically significant based on the investigator's decision. - Number of participants with clinical laboratory abnormalities — Baseline up to Week 52
Findings were considered to be clinically significant based on the investigator's decision. - Antibody against AAV capsid protein — Baseline up to Week 52
Immune response against AAV capsid will be evaluated by measurement of the binding antibody and neutralizing antibody against AAV capsid protein in plasma samples.
Trial sites (1)
| Facility | City | Region | Status |
|---|---|---|---|
| Institute of Hematology & Blood Diseases Hospital | Tianjin | Tianjin Municipality | Recruiting |
More Institute of Hematology & Blood Diseases Hospital, China trials in China
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Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05641610 on ClinicalTrials.gov ↗ ← All trials in China