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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
50
A single intravenous administration of GS1191-0445 injection at a dose of 3E12 vg/kg
Anhui Provincial Hospital
Hefei, Anhui, China
NOT_YET_RECRUITINGPeking Union Medical College Hospital
Beijing, Beijing Municipality, China
NOT_YET_RECRUITINGFirst Hospital of Lanzhou University
Lanzhou, Gansu, China
NOT_YET_RECRUITINGNanfang Hospital Southern Medical University
Guangzhou, Guangdong, China
NOT_YET_RECRUITINGNorth China University of Science and Technology Affiliated Hospital
Tangshan, Hebei, China
NOT_YET_RECRUITINGHenan Cancer Hospital
Zhengzhou, Henan, China
NOT_YET_RECRUITINGUnion Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wuhan, Hubei, China
NOT_YET_RECRUITINGXiangya Hospital of Central South University
Changsha, Hunan, China
NOT_YET_RECRUITINGNanjing Drum Tower Hospital, the Affiliated Hospital of Nanjing University Medical School
Nanjing, Jiangsu, China
NOT_YET_RECRUITINGJiangxi Provincial People's Hospital
Nanchang, Jiangxi, China
NOT_YET_RECRUITING...and 3 more locations
Annualized Bleeding Rate (ABR)
To evaluate the efficacy of a single intravenous administration of GS1191-0445 in subjects with hemophilia A
Time frame: Weeks 3 to 52 after infusion
Incidence of adverse events (AE)
The incidence of adverse events (AE) within 52 weeks after GS1191-0445 infusion, including serious adverse events (SAE) and adverse events of special concern (AESI)
Time frame: Within 52 weeks after infusion
Immunogenic response of AAV8 capsid protein and vector derived FVIII within 52 weeks after GS1191-0445 infusion
Evaluate the safety of a single intravenous infusion of GS1191-0445 injection in hemophilia A subjects
Time frame: Within 52 weeks after infusion
Evaluate the long-term efficacy of GS1191-0445 injection after intravenous infusion for 2-5 years
Evaluate the changes in annualized bleeding rate (ABR) and FVIII activity from baseline during the 2nd to 5th year after GS1191-0445 injection
Time frame: 2-5 years after infusion
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