To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A

NCTID	NCT06833983 (View at clinicaltrials.gov)
Description	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. (Show More)
Development Status	Active
Indication	Hemophilia A
Disease Ontology Term	DOID:12134
Compound Name	GS1191-0445
Compound Alias	GS001
Compound Description	AAV8-BBD F8
Sponsor	Gritgen Therapeutics Co., Ltd.
Funder Type	Industry
Recruitment Status	Recruiting (Click here for study contact information)
Enrollment Count	50 (ESTIMATED)
Results Posted	Not Available

Target Gene/Variant	F8
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell	Hepatocyte
Delivery System	Viral transduction
Vector Type	AAV8
Dose 1	2E12 vg/kg
Dose 2	4E12 vg/kg
Dose 3	Up to 2E13 vg/kg

Eligible Age	18 Years - 65 Years
Standard Ages	Adult, Older adult
Sexes Eligible for Study	MALE
Eligibility Criteria	Inclusion Criteria: 1. Understand the purpose and risks of the study and provide informed consent in accordance with national and local privacy laws: 2. Subject must be male, aged \>18 years old at the time of signing informed consent, and ≤65 years old: 3. Participants with confirmed hemophilia A in their pre-admission history and based on clinical laboratory examination ; 4. Subjects had used FVIII products for at least 150 exposure days (ED) before enrollment; 5. Subject has no prior history of FVIII inhibitors; 6. Subjects agree to use a reliable barrier contraceptive method from the date of signing the informed consent 7. Subject is willing and able to follow planned visits, treatment plans, and other study procedures. Exclusion Criteria: 1. The subject has any hemorrhagic disorder not related to hemophilia A, 2. Abnormal liver function test results of subjects during screening. 3. Abnormal laboratory examination of subjects during screening 4. 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; 5. Active systemic immune disease. View Inclusion and Exclusion Criteria at ClinicalTrials.gov

No.of Trial Sites	13
Locations	China

Has US IND	False
FDA Designations
Recent Updates	First patient dosed November 2023