Clinical trial NCT06833983: To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A
Official title: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Hemophilia A
Lead sponsor: Gritgen Therapeutics Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2025-02-19
Enrollment: 50
Sex: MALE
Minimum age (years): 18
Maximum age (years): 65
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


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 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.

Primary outcomes:
measure: Annualized Bleeding Rate (ABR)
timeFrame: Weeks 3 to 52 after infusion
Secondary outcomes:
measure: Incidence of adverse events (AE)
timeFrame: Within 52 weeks after infusion
measure: Immunogenic response of AAV8 capsid protein and vector derived FVIII within 52 weeks after GS1191-0445 infusion
timeFrame: Within 52 weeks after infusion
measure: Evaluate the long-term efficacy of GS1191-0445 injection after intravenous infusion for 2-5 years
timeFrame: 2-5 years after infusion
Other outcomes:
Locations:
city: Hefei
country: China
facility: Anhui Provincial Hospital
state: Anhui
city: Lanzhou
country: China
facility: First Hospital of Lanzhou University
state: Gansu
city: Guangzhou
country: China
facility: Nanfang Hospital Southern Medical University
state: Guangdong
city: Tangshan
country: China
facility: North China University of Science and Technology Affiliated Hospital
state: Hebei
city: Zhengzhou
country: China
facility: Henan Cancer Hospital
state: Henan
city: Wuhan
country: China
facility: Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
state: Hubei
city: Changsha
country: China
facility: Xiangya Hospital of Central South University
state: Hunan
city: Nanjing
country: China
facility: Nanjing Drum Tower Hospital, the Affiliated Hospital of Nanjing University Medical School
state: Jiangsu
city: Nanchang
country: China
facility: Jiangxi Provincial People's Hospital
state: Jiangxi
city: Jinan
country: China
facility: Central Hospital Affiliated to Shandong First Medical University
state: Shandong
city: Tianjin
country: China
facility: Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
state: Tianjin
city: Kunming
country: China
facility: The second Affiliated Hospital of Kunming Medical University
state: Yunnan
Study officials:
References: