Hemophilia A
Conditions
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.
Interventions
A single intravenous administration of GS1191-0445 injection at a dose of 3E12 vg/kg
Sponsors
Gritgen Therapeutics Co., Ltd.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
18 Years to 65 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
| 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 |
Countries
China
Contacts
Primary ContactYanfang Li
Outcome results
None listed