Hemophilia ANCT05523128
The Efficacy and Safety of ZS802 in Chinese Hemophilia A Patients.
A study testing ZS802 in people with Hemophilia A, run by Institute of Hematology & Blood Diseases Hospital, China at 1 site in China.
Trial summary
A non-randomized, open-label, dose-escalation study to evaluate the safety, tolerability, kinetics and efficacy of a single intravenous infusion of ZS802 in hemophilia A subjects with endogenous FVIII ≤2%. Read more about this study
- Who is it for?
- Ages 18 to 65 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- ZS802
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteChina
- Study dates
- 29 Sep 2022to Oct 2025
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 to 65 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male ≥18 years and ≤65years of age;
- Confirmed diagnosis of hemophilia A, and endogenous FVIII ≤2%:
- <1% (<1 IU/dL) endogenous FVIII activity levels as historically documented by a certified laboratory or screening data results; OR
- 1%-2% (1-2 IU/dL) endogenous FVIII activity levels and >10 bleeding events per year (in the last 52 weeks prior to screening); OR
- 1%-2% (1-2 IU/dL) endogenous FVIII activity levels and on prophylaxis;
- Have had ≥150 prior exposure days (EDs) to any recombinant and/or plasma-derived FVIII protein products.
- Agree to use reliable barrier contraception and prohibition of sperm donation until 52 weeks after the administration of ZS802.
- 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 no measurable FVIII inhibitor as assessed by laboratory; or documented no prior history of FVIII 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 HBV-DNA positive) or active hepatitis C infection (HCVAb positive), or are currently receiving hepatitis B or hepatitis C antiviral therapy;
- Have history of chronic infection or other chronic disease that the Investigator considers to constitute an unacceptable risk;
- 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 FVIII 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 donated or 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.
China1 site, 1 recruiting
- Recruiting
Institute of Hematology & Blood Diseases Hospital
Tianjin
Lei Zhang, MD, +86 022-23909240, zhanglei1@ihcams.ac.cn
Lei Zhang, MD
About this study
Official title: A Non-randomized, Open-label, Dose-escalation Study to Evaluate the Safety, Tolerability, Kinetics and Efficacy of a Single Intravenous Infusion of ZS802 in Hemophilia A Subjects With Endogenous FVIII ≤2%.
A non-randomized, open-label, dose-escalation study to evaluate the safety, tolerability, kinetics and efficacy of a single intravenous infusion of ZS802 in hemophilia A subjects with endogenous FVIII ≤2%.
Read the detailed description
This study will seek to determine the safety, tolerability, kinetics and efficacy of a single IV infusion of ZS802.
Hemophilia A is a genetic bleeding disorder resulting in the lack of ability to produce blood-clotting factor VIII (FVIII). Individuals with hemophilia A 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 FVIII protein products, either prophylactically or in response to bleeding. ZS802 is an adeno-associated viral (AAV) vector designed to drive expression of the human factor VIII (hFVIII) transgene and raise circulating levels of endogenous FVIII.
6 patients will be enrolled sequentially every 3 weeks or more between cohorts and administered with single infusion of ZS802. Dose escalation may occur based on the safety and FVIII activity on steady state. The dose levels are as follows: 1. 2.0×10^13vg/kg; 2. 6.0×10^13vg/kg. Subjects will provide informed consent and then undergo screening assessments up to 6-8weeks prior administration of ZS802. All subjects will undergo 52 weeks safety and efficacy observation.
- Conditions
- Hemophilia A
- Being tested
- ZS802
- Keywords
- Hemophilia A, Gene therapy, Adeno-associated virus
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ZS802Genetic
A novel, bioengineered adeno-associated viral (AAV) vector carrying human factor VIII variant. The dose levels are as follows: 1. 2.0×10^13vg/kg; 2. 6.0×10^13vg/kg.
| Study group | Role | What they receive |
|---|---|---|
| ZS802 | Experimental | Single intravenous (i.v.) infusion of ZS802 Intervention: Gene Therapy / Gene Transfer Receives: Genetic: ZS802 |
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: 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
Secondary outcomes (4)
Vector-derived FVIII:C Activity
Peak and steady-state vector-derived circulating FVIII activity levels, and changes of FVIII activity levels after treatment compared with baseline.
Measured: Baseline up to Week 52
Vector-derived FVIII antigen levels
Peak and steady-state vector-derived circulating FVIII antigen levels, and changes of FVIII antigen levels after treatment compared with baseline.
Measured: Baseline up to Week 52
Vector shedding of ZS802
Blood, saliva, urine and semen will be collected to assess clearance of vector genomes.
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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Aug 2022
- Study start29 Sep 2022
- Primary completionOct 2025 (estimated)
- Study completionOct 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Hemophilia A patients
- Masking
- None (open label)
- Enrolment
- 6 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
- NCT05523128
- Sponsor's protocol ID
- IIT2022031
- Status last verified
- May 2024
- First submitted
- 25 Jul 2022
- Last updated
- 21 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 ZS802 is fully approved.
Study at a glance
From the registry record, last updated by the sponsor on 21 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Non-randomized, Open-label, Dose-escalation Study to Evaluate the Safety, Tolerability, Kinetics and Efficacy of a Single Intravenous Infusion of ZS802 in Hemophilia A Subjects With Endogenous FVIII ≤2%.
- ClinicalTrials.gov ID
- NCT05523128
- Acronym / sponsor ID
- IIT2022031
- Conditions
- Hemophilia A
- Keywords
- Hemophilia A, Gene therapy, Adeno-associated virus
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Institute of Hematology & Blood Diseases Hospital, ChinaUniversity, hospital or other
- Study start
- 29 Sep 2022 (actual)
- Primary completion
- Oct 2025 (estimated)
- Study completion
- Oct 2025 (estimated)
- First posted
- 31 Aug 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 study to evaluate the safety, tolerability, kinetics and efficacy of a single intravenous infusion of ZS802 in hemophilia A subjects with endogenous FVIII ≤2%.
Detailed description
This study will seek to determine the safety, tolerability, kinetics and efficacy of a single IV infusion of ZS802.
Hemophilia A is a genetic bleeding disorder resulting in the lack of ability to produce blood-clotting factor VIII (FVIII). Individuals with hemophilia A 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 FVIII protein products, either prophylactically or in response to bleeding. ZS802 is an adeno-associated viral (AAV) vector designed to drive expression of the human factor VIII (hFVIII) transgene and raise circulating levels of endogenous FVIII.
6 patients will be enrolled sequentially every 3 weeks or more between cohorts and administered with single infusion of ZS802. Dose escalation may occur based on the safety and FVIII activity on steady state. The dose levels are as follows: 1. 2.0×10^13vg/kg; 2. 6.0×10^13vg/kg. Subjects will provide informed consent and then undergo screening assessments up to 6-8weeks prior administration of ZS802. 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 A patients
| Arm | Type | Receives |
|---|---|---|
| ZS802 | Experimental | Genetic: ZS802Single intravenous (i.v.) infusion of ZS802 Intervention: Gene Therapy / Gene Transfer |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Male ≥18 years and ≤65years of age;
- Confirmed diagnosis of hemophilia A, and endogenous FVIII ≤2%:
- Have had ≥150 prior exposure days (EDs) to any recombinant and/or plasma-derived FVIII protein products.
- Agree to use reliable barrier contraception and prohibition of sperm donation until 52 weeks after the administration of ZS802.
- 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.
Key exclusion criteria (28 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 no measurable FVIII inhibitor as assessed by laboratory; or documented no prior history of FVIII 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 |
|---|---|---|
| ZS802A novel, bioengineered adeno-associated viral (AAV) vector carrying human factor VIII variant. The dose levels are as follows: 1. 2.0×10^13vg/kg; 2. 6.0×10^13vg/kg. | Genetic | ZS802 |
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: 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.
4 secondary outcomes
Vector-derived FVIII:C Activity
Time frame: Baseline up to Week 52
Vector-derived FVIII antigen levels
Time frame: Baseline up to Week 52
Vector shedding of ZS802
Time frame: Baseline up to Week 52
Antibody against AAV capsid protein
Time frame: Baseline up to Week 52
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Feb 2025.
- Individual participant dataSharing planned: Yes. IPD will be shared with other researchers when ZS802 is fully approved.
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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. This study is not recruiting at the moment.
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 study testing ZS802 in people with Hemophilia A, 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 8 inclusion and 28 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: status not confirmed, as of the sponsor's last update on 21 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.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Lei Zhang, 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 4 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 21 Feb 2025.