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

Phase 3

Recruiting

• Conditions: Hemophilia A
• Interventions: Drug: GS1191-0445 injection

• Registration Number: NCT06833983
• Lead Sponsor: Gritgen Therapeutics Co., Ltd.

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2025-01-28
• Status Verified: 2025-02
• Study First Submitted QC: 2025-02-13
• Study First Posted: 2025-02-19
• Study Start: 2025-03-31
• Last Update Submitted: 2025-04-20
• Last Update Posted: 2025-04-24
• Primary Completion: 2026-11-30
• Study Completion: 2030-11-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: Male
• Target Recruitment: 50

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
3E12 vg/kg	GS1191-0445 injection	-

Primary Outcome Measures

Name	Time	Method
Annualized Bleeding Rate (ABR)	Weeks 3 to 52 after infusion	To evaluate the efficacy of a single intravenous administration of GS1191-0445 in subjects with hemophilia A

Secondary Outcome Measures

Name	Time	Method
Incidence of adverse events (AE)	Within 52 weeks after infusion	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)
Immunogenic response of AAV8 capsid protein and vector derived FVIII within 52 weeks after GS1191-0445 infusion	Within 52 weeks after infusion	Evaluate the safety of a single intravenous infusion of GS1191-0445 injection in hemophilia A subjects
Evaluate the long-term efficacy of GS1191-0445 injection after intravenous infusion for 2-5 years	2-5 years after infusion	Evaluate the changes in annualized bleeding rate (ABR) and FVIII activity from baseline during the 2nd to 5th year after GS1191-0445 injection

Trial Locations

Locations (12)

North China University of Science and Technology Affiliated Hospital; 🇨🇳 Tangshan, Hebei, China

Anhui Provincial Hospital; 🇨🇳 Hefei, Anhui, China

First Hospital of Lanzhou University; 🇨🇳 Lanzhou, Gansu, China

Nanfang Hospital Southern Medical University; 🇨🇳 Guangzhou, Guangdong, China

Henan Cancer Hospital; 🇨🇳 Zhengzhou, Henan, China

Union Hospital, Tongji Medical College, Huazhong University of Science and Technology; 🇨🇳 Wuhan, Hubei, China

Xiangya Hospital of Central South University; 🇨🇳 Changsha, Hunan, China

Nanjing Drum Tower Hospital, the Affiliated Hospital of Nanjing University Medical School; 🇨🇳 Nanjing, Jiangsu, China

Jiangxi Provincial People's Hospital; 🇨🇳 Nanchang, Jiangxi, China

Central Hospital Affiliated to Shandong First Medical University; 🇨🇳 Jinan, Shandong, China

Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College; 🇨🇳 Tianjin, Tianjin, China

The second Affiliated Hospital of Kunming Medical University; 🇨🇳 Kunming, Yunnan, China