To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A
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
Gritgen Therapeutics Co., Ltd.
50 participants
Mar 31, 2025
INTERVENTIONAL
Conditions
Summary
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.
Eligibility
Inclusion Criteria7
- 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 Criteria5
- 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.
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Interventions
A single intravenous administration of GS1191-0445 injection at a dose of 3E12 vg/kg
Locations(13)
View Full Details on ClinicalTrials.gov
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NCT06833983