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

NCT06833983 | Recruiting Phase 3

• 1 other identifier: GS1191-0445-GTHA-CN02
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 13

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.

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

• Annualized Bleeding Rate (ABR)

  To evaluate the efficacy of a single intravenous administration of GS1191-0445 in subjects with hemophilia A

  Weeks 3 to 52 after infusion

Secondary Outcomes (3)

• Incidence of adverse events (AE)

  Within 52 weeks after infusion

• Immunogenic response of AAV8 capsid protein and vector derived FVIII within 52 weeks after GS1191-0445 infusion

  Within 52 weeks after infusion

• Evaluate the long-term efficacy of GS1191-0445 injection after intravenous infusion for 2-5 years

  2-5 years after infusion

Study Arms (1)

3E12 vg/kg

EXPERIMENTAL

Drug: GS1191-0445 injection

Interventions

GS1191-0445 injection DRUG

A single intravenous administration of GS1191-0445 injection at a dose of 3E12 vg/kg

3E12 vg/kg

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Understand the purpose and risks of the study and provide informed consent in accordance with national and local privacy laws:
• Subject must be male, aged \>18 years old at the time of signing informed consent, and ≤65 years old:
• Participants with confirmed hemophilia A in their pre-admission history and based on clinical laboratory examination ;
• Subjects had used FVIII products for at least 150 exposure days (ED) before enrollment;
• Subject has no prior history of FVIII inhibitors;
• Subjects agree to use a reliable barrier contraceptive method from the date of signing the informed consent
• Subject is willing and able to follow planned visits, treatment plans, and other study procedures.

You may not qualify if:

• The subject has any hemorrhagic disorder not related to hemophilia A,
• Abnormal liver function test results of subjects during screening.
• Abnormal laboratory examination of subjects during screening
• 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;
• Active systemic immune disease.

Sponsors & Collaborators

• Gritgen Therapeutics Co., Ltd.: lead

Study Sites (13)

Anhui Provincial Hospital

Hefei, Anhui, 230001, China

NOT YET RECRUITING

Peking Union Medical College Hospital

Beijing, Beijing Municipality, 100005, China

NOT YET RECRUITING

First Hospital of Lanzhou University

Lanzhou, Gansu, 730099, China

NOT YET RECRUITING

Nanfang Hospital Southern Medical University

Guangzhou, Guangdong, 510515, China

NOT YET RECRUITING

North China University of Science and Technology Affiliated Hospital

Tangshan, Hebei, 063000, China

NOT YET RECRUITING

Henan Cancer Hospital

Zhengzhou, Henan, 450003, China

NOT YET RECRUITING

Union Hospital, Tongji Medical College, Huazhong University of Science and Technology

Wuhan, Hubei, 430022, China

NOT YET RECRUITING

Xiangya Hospital of Central South University

Changsha, Hunan, 410028, China

NOT YET RECRUITING

Nanjing Drum Tower Hospital, the Affiliated Hospital of Nanjing University Medical School

Nanjing, Jiangsu, 210000, China

NOT YET RECRUITING

Jiangxi Provincial People's Hospital

Nanchang, Jiangxi, 344001, China

NOT YET RECRUITING

Central Hospital Affiliated to Shandong First Medical University

Jinan, Shandong, 250013, China

RECRUITING

Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

The second Affiliated Hospital of Kunming Medical University

Kunming, Yunnan, 650101, China

NOT YET RECRUITING

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Yanfang Li

CONTACT

+8613817454595; yanfang.li@gritgen.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 28, 2025
• First Posted: February 19, 2025
• Study Start: March 31, 2025
• Primary Completion (Estimated): November 30, 2026
• Study Completion (Estimated): November 30, 2030
• Last Updated: December 9, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (13)