The Efficacy and Safety of ZS802 in Chinese Hemophilia A Patients.

NCT05523128 | Recruiting

• 1 other identifier: IIT2022031
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief 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%.

Conditions

Hemophilia A

Keywords

Hemophilia A; Gene therapy; Adeno-associated virus

Outcome Measures

Primary Outcomes (4)

• Incidence of adverse events

  An adverse event (AE) is any medical occurrence, the event will not relate to the treatment.

  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.

  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.

  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.

  Baseline up to Week 52

Secondary Outcomes (4)

• Vector-derived FVIII:C Activity

  Baseline up to Week 52

• Vector-derived FVIII antigen levels

  Baseline up to Week 52

• Vector shedding of ZS802

  Baseline up to Week 52

• Antibody against AAV capsid protein

  Baseline up to Week 52

Other Outcomes (3)

• Annualized bleeding rate changes from baseline

  Baseline up to Week 52

• Annualized FVIII consumption changes from baseline

  Baseline up to Week 52

• Number of target joints

  Baseline up to Week 52

Study Arms (1)

ZS802

EXPERIMENTAL

Single intravenous (i.v.) infusion of ZS802 Intervention: Gene Therapy / Gene Transfer

Genetic: ZS802

Interventions

ZS802 GENETIC

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.

ZS802

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• %-2% (1-2 IU/dL) endogenous FVIII activity levels and \>10 bleeding events per year (in the last 52 weeks prior to screening); OR
• %-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.

You may not qualify if:

• 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;
• 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;

Sponsors & Collaborators

• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Institute of Hematology & Blood Diseases Hospital

Tianjin, China

RECRUITING

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Lei Zhang, MD

  Institute of Hematology & Blood Diseases Hospital, China

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Lei Zhang, MD

CONTACT

+86 022-23909240; zhanglei1@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Hemophilia A patients

Study Record Dates

• First Submitted: July 25, 2022
• First Posted: August 31, 2022
• Study Start: September 29, 2022
• Primary Completion: October 1, 2025
• Study Completion: October 1, 2025
• Last Updated: February 21, 2025

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL
• Time Frame: IPD will be shared with other researchers when ZS802 is fully approved.
• Access Criteria: IPD will be shared with other researchers when ZS802 is fully approved.

Locations

CN (1)