RECRUITINGPhase 3INTERVENTIONAL
To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A
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
About This Trial
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.
Who May Be Eligible (Plain English)
Who May Qualify:
1. Understand the purpose and risks of the study and provide willing to sign a consent form in accordance with national and local privacy laws:
2. Subject must be male, aged \>18 years old at the time of signing willing to sign a consent form, 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 FVIII 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 willing to sign a consent form
7. Subject is willing and able to follow planned visits, treatment plans, and other study procedures.
Who Should NOT Join This Trial:
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.
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
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 FVIII 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.
Treatments Being Tested
DRUG
GS1191-0445 injection
A single intravenous administration of GS1191-0445 injection at a dose of 3E12 vg/kg
Locations (13)
Anhui Provincial Hospital
Hefei, Anhui, China
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China
First Hospital of Lanzhou University
Lanzhou, Gansu, China
Nanfang Hospital Southern Medical University
Guangzhou, Guangdong, China
North China University of Science and Technology Affiliated Hospital
Tangshan, Hebei, 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 Municipality, China
The second Affiliated Hospital of Kunming Medical University
Kunming, Yunnan, China