Hemophilia BPhase 1/2NCT05641610
A Study to Evaluate the Safety and Efficacy of ZS801 in Adult Hemophilia B Patients
A Phase 1/2 study testing ZS801 in people with Hemophilia B, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial 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%. Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia B
- What is being tested?
- ZS801
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 1 siteChina
- Study dates
- 16 Feb 2022to Dec 2028
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male ≥18 years of age;
- Confirmed diagnosis of hemophilia B, and endogenous FIX ≤2%;
- Have had ≥100 prior exposure days (EDs) to any recombinant and/or plasma-derived FIX protein products;
- 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;
- Agree to use reliable barrier contraception and prohibition of sperm donation until 52 weeks after the administration of ZS801.
- 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.
Who cannot join
- Hypersensitivity to any component of the study drug (including immunosuppressants) or a condition that can not use;
- Inability to tolerate immunosuppressants or steroid drugs;
- Have FIX inhibitor as assessed by laboratory, or documented history of FIX inhibitor;
- Who have a history or are currently suffering from any of the following serious clinical diseases:
- History of malignancy or current presence of any malignancy;
- Have active autoimmune disease;
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- Severe heart disease, including angina pectoris, myocardial infarction, heart failure, clinically significant congenital heart disease, heart valve disease, arrhythmia and atrioventricular block, etc.;
- Have underlying liver disease or history of liver disease (such as portal hypertension, ascites, splenomegaly, esophageal varices, hepatic encephalopathy or hepatic fibrosis);
- 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;
- Diabetes mellitus that is poorly controlled after drug treatment;
- Uncontrolled hypertension or hypotension;
- laboratory values:
- Hemoglobin<110g/L;
- Platelets<100×10^9/L;
- AST, ALT, alkaline phosphatase>2×ULN;
- Total bilirubin>1.5×ULN;
- Creatinine>ULN;
- Albumin<LLN;
- HIV antibody positive or Treponema pallidum antibody positive.
- Have AAV5 capsid neutralizing antibody titers >1:5;
- 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;
- Those who have planned surgery within 52 weeks after the infusion;
- Those who lost more than 400 mL of blood within 3 months before screening;
- Those with epilepsy, history of mental illness (such as schizophrenia, depression, mania or anxiety) or obvious mental disorder, incapacitated or incapacitated by other reasons;
- Patients with a history of drug abuse or alcoholism;
- Investigators believe that subjects have poor compliance or are expected to be less likely to complete follow-up;
- There are clinically significant diseases or other reasons that the researcher and/or collaborators consider unsuitable to participate in this researcher.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Institute of Hematology & Blood Diseases Hospital
Tianjin, Tianjin Municipality, 300020
Lei Zhang, MD, zhanglei1@ihcams.ac.cn
About this study
Official title: 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%.
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%.
Read the detailed description
This study will seek to determine the safety, tolerability, kinetics and efficacy of a single IV infusion of ZS801.
Hemophilia B is a genetic bleeding disorder resulting in the lack of ability to produce blood-clotting factor IX (FIX). Individuals with hemophilia B suffer repeated bleeding events, which can cause chronic joint disease and sometimes leads to death due to the inability for blood to clot efficiently. The current treatment is intravenous infusion of FIX protein products, either prophylactically or in response to bleeding.
ZS801 is an adeno-associated viral (AAV) vector designed to drive expression of the human factor IX (hFIX) transgene and raise circulating levels of endogenous FIX.
Dose-escalation phase: 16 patients will be enrolled sequentially every 3 weeks or more between cohorts and administered with single infusion of ZS801. Dose escalation may occur based on the safety and FIX activity on steady state. The dose levels are as follows: 2.0×10^12vg/kg, 5.0×10^12vg/kg, 1.0×10^13vg/kg.
Dose-expansion phase: 5 patients will be enrolled and be administrated of ZS801.
Subjects will provide informed consent and then undergo screening assessments up to 6-8 weeks prior administration of ZS801. All subjects will undergo 52 weeks safety and efficacy observation.
- Conditions
- Hemophilia B
- Being tested
- ZS801
- Keywords
- Hemophilia B, Gene therapy
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ZS801Genetic
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 group | Role | What they receive |
|---|---|---|
| ZS801 | Experimental | Single intravenous (i.v.) infusion of ZS801 Intervention: Gene Therapy / Gene Transfer Receives: Genetic: ZS801 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of adverse events
An adverse event (AE) is any medical occurrence, the event will not relate to the treatment.
Measured: Baseline up to Week 52
Number of participants with clinically significant change from baseline in vital signs
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.
Measured: Baseline up to Week 52
Number of participants with clinically significant change from baseline in physical examination findings
Findings will be considered to be clinically significant based on the investigator's decision.
Measured: Time Frame: Baseline up to Week 52
Number of participants with clinical laboratory abnormalities
Findings were considered to be clinically significant based on the investigator's decision.
Measured: Baseline up to Week 52
Antibody against AAV capsid protein
Immune response against AAV capsid will be evaluated by measurement of the binding antibody and neutralizing antibody against AAV capsid protein in plasma samples.
Measured: Baseline up to Week 52
Secondary outcomes (3)
Vector-derived FIX activity levels
The vector-derived endogenous FIX activity levels will be measured by One-Stage clot (OS) assay, and characterized by post-treatment population mean and its change from baseline during each visit.
Measured: Baseline up to Week 52
Vector-derived FIX antigen levels
The vector-derived endogenous FIX antigen levels will be characterized by post-treatment population mean, and its change from baseline during each visit.
Measured: Baseline up to Week 52
Vector shedding of ZS801
Blood, saliva, urine and semen will be collected to assess clearance of vector genomes.
Measured: Baseline up to Week 52
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Dec 2022
- Study start16 Feb 2022
- Primary completionDec 2025 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia BStudies of the same condition, open and under way.
- More trials run by Institute of Hematology & Blood Diseases Hospital, ChinaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Hemophilia B patients
- Masking
- None (open label)
- Enrolment
- 21 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Lei Zhang, MD, Principal investigator, Institute of Hematology & Blood Diseases Hospital, China
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05641610
- Sponsor's protocol ID
- IIT2021057（ZS801-01）
- Status last verified
- May 2024
- First submitted
- 25 Nov 2022
- Last updated
- 24 Feb 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: IPD will be shared with other researchers when ZS801 is fully approved
Study at a glance
From the registry record, last updated by the sponsor on 24 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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%.
- ClinicalTrials.gov ID
- NCT05641610
- Acronym / sponsor ID
- IIT2021057（ZS801-01）
- Conditions
- Hemophilia B
- Keywords
- Hemophilia B, Gene therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 21 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 16 Feb 2022 (actual)
- Primary completion
- Dec 2025 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 7 Dec 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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%.
Detailed description
This study will seek to determine the safety, tolerability, kinetics and efficacy of a single IV infusion of ZS801.
Hemophilia B is a genetic bleeding disorder resulting in the lack of ability to produce blood-clotting factor IX (FIX). Individuals with hemophilia B suffer repeated bleeding events, which can cause chronic joint disease and sometimes leads to death due to the inability for blood to clot efficiently. The current treatment is intravenous infusion of FIX protein products, either prophylactically or in response to bleeding.
ZS801 is an adeno-associated viral (AAV) vector designed to drive expression of the human factor IX (hFIX) transgene and raise circulating levels of endogenous FIX.
Dose-escalation phase: 16 patients will be enrolled sequentially every 3 weeks or more between cohorts and administered with single infusion of ZS801. Dose escalation may occur based on the safety and FIX activity on steady state. The dose levels are as follows: 2.0×10^12vg/kg, 5.0×10^12vg/kg, 1.0×10^13vg/kg.
Dose-expansion phase: 5 patients will be enrolled and be administrated of ZS801.
Subjects will provide informed consent and then undergo screening assessments up to 6-8 weeks prior administration of ZS801. All subjects will undergo 52 weeks safety and efficacy observation.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Hemophilia B patients
| Arm | Type | Receives |
|---|---|---|
| ZS801 | Experimental | Genetic: ZS801Single intravenous (i.v.) infusion of ZS801 Intervention: Gene Therapy / Gene Transfer |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Male ≥18 years of age;
- Confirmed diagnosis of hemophilia B, and endogenous FIX ≤2%;
- Have had ≥100 prior exposure days (EDs) to any recombinant and/or plasma-derived FIX protein products;
- 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;
- Agree to use reliable barrier contraception and prohibition of sperm donation until 52 weeks after the administration of ZS801.
Key exclusion criteria (27 listed)
- Hypersensitivity to any component of the study drug (including immunosuppressants) or a condition that can not use;
- Inability to tolerate immunosuppressants or steroid drugs;
- Have FIX inhibitor as assessed by laboratory, or documented history of FIX inhibitor;
- Who have a history or are currently suffering from any of the following serious clinical diseases:
- laboratory values:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ZS801A 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. | Genetic | ZS801 |
Primary outcome
Incidence of adverse events
Time frame: 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
Time frame: 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: 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
Time frame: Baseline up to Week 52
Findings were considered to be clinically significant based on the investigator's decision.
Antibody against AAV capsid protein
Time frame: 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.
3 secondary outcomes
Vector-derived FIX activity levels
Time frame: Baseline up to Week 52
Vector-derived FIX antigen levels
Time frame: Baseline up to Week 52
Vector shedding of ZS801
Time frame: Baseline up to Week 52
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Feb 2025.
- Individual participant dataSharing planned: Yes. IPD will be shared with other researchers when ZS801 is fully approved
Related trials for Hemophilia B
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
Not yet recruitingPhase 1
Lentiviral FIX Gene Therapy
Hemophilia B
RecruitingPhase 3
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
Hemophilia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Lei Zhang, MD principal investigator
Institute of Hematology & Blood Diseases Hospital, China
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ZS801 in people with Hemophilia B, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 27 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 24 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Zhang Lei, MD (+86 022-23909240, zhanglei1@ihcams.ac.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events (time frame: Baseline up to Week 52). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Feb 2025.