NCT06833983招募中3 期
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.13 个研究点 分布在 1 个国家目标入组 50 人开始时间: 2025年3月31日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 发起方
- 入组人数
- 50
- 试验地点
- 13
- 主要终点
- Annualized Bleeding Rate (ABR)
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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.
研究组 & 干预措施
3E12 vg/kg
Experimental
干预措施: GS1191-0445 injection (Drug)
结局指标
主要结局
Annualized Bleeding Rate (ABR)
时间窗: Weeks 3 to 52 after infusion
To evaluate the efficacy of a single intravenous administration of GS1191-0445 in subjects with hemophilia A
次要结局
- Incidence of adverse events (AE)(Within 52 weeks after infusion)
- Immunogenic response of AAV8 capsid protein and vector derived FVIII within 52 weeks after GS1191-0445 infusion(Within 52 weeks after infusion)
- Evaluate the long-term efficacy of GS1191-0445 injection after intravenous infusion for 2-5 years(2-5 years after infusion)
研究者
研究点 (13)
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