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To Evaluate the Safety and Efficacy of GS1191-0445 Injection in the Treatment of Severe Hemophilia A
Sponsor: Gritgen Therapeutics Co., Ltd.
Summary
This study is a single-arm, open-label study evaluating the safety and efficacy of GS1191-0445 injection as a single dose in Chinese subjects with severe hemophilia A. GS1191-0445 is an adeno-associated virus 8 (AAV8)-delivered gene therapy 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 gene expression cassette, which transfects hepatocytes and facilitates the specific expression and secretion of FVIII into the blood.
Official title: A Single-arm, Open-label Study Evaluating the Safety and Efficacy of a Single Dose of GS1191-0445 Injection in Chinese Subjects With Severe Hemophilia A
Key Details
Gender
MALE
Age Range
18 Years - 65 Years
Study Type
INTERVENTIONAL
Enrollment
7
Start Date
2023-07-10
Completion Date
2029-12-23
Last Updated
2026-05-06
Healthy Volunteers
No
Conditions
Interventions
GS1191-0445 injection
A single intravenous administration of GS1191-0445 injection at a dose of 3E12 vg/kg
Locations (2)
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wuhan, Hubei, China
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
Tianjin, Tianjin Municipality, China