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

Status: Recruiting
Location: See all (13) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 3
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.

Eligibility
Participation Requirements
Sex: Male
Minimum Age: 18
Maximum Age: 65
Healthy Volunteers: f
View:

• 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 FVII 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.

Locations
Other Locations
China
Peking Union Medical College Hospital
NOT_YET_RECRUITING
Beijing
Xiangya Hospital of Central South University
NOT_YET_RECRUITING
Changsha
Nanfang Hospital Southern Medical University
NOT_YET_RECRUITING
Guangzhou
Anhui Provincial Hospital
NOT_YET_RECRUITING
Hefei
Central Hospital Affiliated to Shandong First Medical University
RECRUITING
Jinan
The second Affiliated Hospital of Kunming Medical University
NOT_YET_RECRUITING
Kunming
First Hospital of Lanzhou University
NOT_YET_RECRUITING
Lanzhou
Jiangxi Provincial People's Hospital
NOT_YET_RECRUITING
Nanchang
Nanjing Drum Tower Hospital, the Affiliated Hospital of Nanjing University Medical School
NOT_YET_RECRUITING
Nanjing
North China University of Science and Technology Affiliated Hospital
NOT_YET_RECRUITING
Tangshan
Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
RECRUITING
Tianjin
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
NOT_YET_RECRUITING
Wuhan
Henan Cancer Hospital
NOT_YET_RECRUITING
Zhengzhou
Contact Information
Primary
Yanfang Li
yanfang.li@gritgen.com
+8613817454595
Time Frame
Start Date: 2025-03-31
Estimated Completion Date: 2030-11-30
Participants
Target number of participants: 50
Treatments
Experimental: 3E12 vg/kg
Related Therapeutic Areas
Sponsors
Leads: Gritgen Therapeutics Co., Ltd.

This content was sourced from clinicaltrials.gov