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

Gritgen Therapeutics Co., Ltd.

Status and phase: Enrolling; Phase 3

Conditions: Hemophilia A

Treatments: Drug: GS1191-0445 injection

Study type: Interventional

Funder types: Industry

Identifiers

NCT06833983

GS1191-0445-GTHA-CN02

Details and patient eligibility

About

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.

Enrollment: 50 estimated patients

Sex: Male

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

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 FVII 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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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50 participants in 1 patient group

3E12 vg/kg

Experimental group

Treatment:

Drug: GS1191-0445 injection