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NCT Number: NCT06833983

To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A

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.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

18 year–65 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 3

Primary location

Anhui Provincial Hospital, Hefei, Anhui, China

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 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 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
  • Subject is willing and able to follow planned visits, treatment plans, and other study procedures.

Exclusion criteria

  • 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;
  • Active systemic immune disease.

Treatment and study plan

GS1191-0445 injection

Drug

A single intravenous administration of GS1191-0445 injection at a dose of 3E12 vg/kg

Primary outcomes

  1. Annualized Bleeding Rate (ABR)

    Time frame: Weeks 3 to 52 after infusion

    To evaluate the efficacy of a single intravenous administration of GS1191-0445 in subjects with hemophilia A

Secondary outcomes

  1. Incidence of adverse events (AE)

    Time frame: 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)

  2. Immunogenic response of AAV8 capsid protein and vector derived FVIII within 52 weeks after GS1191-0445 infusion

    Time frame: Within 52 weeks after infusion

    Evaluate the safety of a single intravenous infusion of GS1191-0445 injection in hemophilia A subjects

  3. Evaluate the long-term efficacy of GS1191-0445 injection after intravenous infusion for 2-5 years

    Time frame: 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

Study contacts

Contact information is provided by the study sponsor or research team.

Yanfang Li

CONTACT

[email protected]

+8613817454595

Sponsors and collaborators

Lead sponsor

Gritgen Therapeutics Co., Ltd.

Industry

Registry information

Official study 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

Important dates

Study start
2025
Primary completion
2026
Study completion
2030
First posted
Feb 19, 2025
Registry last updated
Dec 9, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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