To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: GS1191-0445 injection.
- Who it may be relevant to
- Registry conditions: Hemophilia A. Basic parameters: 18 years — 65 years · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
A Single-arm, Open-label, Multicenter Study Evaluating the Efficacy and Safety of GS1191-0445 Injection as a Single Dose in Chinese Subjects With Hemophilia A
Overview
This study is a single-arm, open-label, multicenter study evaluating the efficacy and safety of GS1191-0445 injection as a single dose in Chinese subjects with hemophilia A. GS1191-0445 is an AAV8-based gene therapy vector designed to express B-domain deleted human factor VIII (FVIII) under the regulation of a human liver-specific promoter. Following a single intravenous administration, AAV8 targets hepatocytes and facilitates the specific expression and secretion of FVIII into the bloodstream.
Interventions
- Drug GS1191-0445 injection
A single intravenous administration of GS1191-0445 injection at a dose of 3E12 vg/kg
Primary outcome measures
- Annualized Bleeding Rate (ABR) [Time frame: Weeks 3 to 52 after infusion]
Secondary outcome measures (3)
- Incidence of adverse events (AE) [Time frame: Within 52 weeks after infusion]
- Immunogenic response of AAV8 capsid protein and vector derived FVIII within 52 weeks after GS1191-0445 infusion [Time frame: Within 52 weeks after infusion]
- Evaluate the long-term efficacy of GS1191-0445 injection after intravenous infusion for 2-5 years [Time frame: 2-5 years after infusion]
Eligibility criteria
Inclusion criteria
- Understand the purpose and risks of the study and provide informed consent in accordance with national and local privacy laws:
- Subject must be male, aged >18 years old at the time of signing informed consent, and ≤65 years old:
- Participants with confirmed hemophilia A in their pre-admission history and based on clinical laboratory examination ;
- Subjects had used FVIII products for at least 150 exposure days (ED) before enrollment;
- Subject has no prior history of FVIII inhibitors;
- Subjects agree to use a reliable barrier contraceptive method from the date of signing the informed consent
- Subject is willing and able to follow planned visits, treatment plans, and other study procedures.
Exclusion criteria
- The subject has any hemorrhagic disorder not related to hemophilia A,
- Abnormal liver function test results of subjects during screening.
- Abnormal laboratory examination of subjects during screening
- The subject has acute or chronic hepatitis B virus (HBV) infection or chronic hepatitis C virus (HCV) infection; Or are receiving antiviral treatment for hepatitis B and C;
- Active systemic immune disease.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 13 centers
- Anhui Provincial Hospital — Hefei
- Peking Union Medical College Hospital — Beijing
- First Hospital of Lanzhou University — Lanzhou
- Nanfang Hospital Southern Medical University — Guangzhou
- North China University of Science and Technology Affiliated Hospital — Tangshan
- Henan Cancer Hospital — Zhengzhou
- Union Hospital, Tongji Medical College, Huazhong University of Science and Technology — Wuhan
- Xiangya Hospital of Central South University — Changsha
- … and 5 more centers
Identifiers
NCT: NCT06833983 · GS1191-0445-GTHA-CN02