To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A
ConditionHemophilia A
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexMale
Age18-65
SponsorGritgen Therapeutics Co., Ltd.
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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
Disqualifiers
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;
Trial design
Design model
Single group
Treatments tested in this trial
GS1191-0445 injection
DrugA single intravenous administration of GS1191-0445 injection at a dose of 3E12 vg/kg
Treatment groups
50 Participants
are divided into 1 treatment groupGroup A: 3E12 vg/kgExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Annualized Bleeding Rate (ABR)
To evaluate the efficacy of a single intravenous administration of GS1191-0445 in subjects with hemophilia A
Time frame
Weeks 3 to 52 after infusionSecondary outcomes
1
Incidence of adverse events (AE)
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)
Time frame
Within 52 weeks after infusion2
Immunogenic response of AAV8 capsid protein and vector derived FVIII within 52 weeks after GS1191-0445 infusion
Evaluate the safety of a single intravenous infusion of GS1191-0445 injection in hemophilia A subjects
Time frame
Within 52 weeks after infusion3
Evaluate the long-term efficacy of GS1191-0445 injection after intravenous infusion for 2-5 years
Evaluate the changes in annualized bleeding rate (ABR) and FVIII activity from baseline during the 2nd to 5th year after GS1191-0445 injection
Time frame
2-5 years after infusionSponsors and contacts
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Source ClinicalTrials.gov