Skip to main content
OpenTrials
Active, Not Recruiting

NCT Number: NCT07548411

To Evaluate the Safety and Efficacy of GS1191-0445 Injection in the Treatment of Severe Hemophilia A

This study is a single-arm, open-label study evaluating the safety and efficacy of GS1191-0445 injection as a single dose in Chinese subjects with severe hemophilia A.

GS1191-0445 is an adeno-associated virus 8 (AAV8)-delivered gene therapy 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 gene expression cassette, which transfects hepatocytes and facilitates the specific expression and secretion of FVIII into the blood.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Notify Me

Key information

Conditions

Severe 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 Hemophilia A Hemorrhagic Disorders

Age range

18 year–65 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, Hubei, China

Loading trial locations.

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:
  • Participants with confirmed severe 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 received continuous prophylactic treatment with exogenous FVIII for one year prior to enrollment or has been treated with exogenous FVIII on demand;
  • Subject has no history of hypersensitivity or allergic reactions related to the administration of FVIII agents;
  • Subject has no 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. Number of participants with Adverse Events (AE) as assessed by CTCAE v5.0, including Adverse Event of Special Interests (AESI) and Serious Adverse Events (SAE);

    Time frame: Five years after infusion

  2. Number of Participants with Thrombosis Risk: In subjects with >150% FⅧ:C post-GS1191-0445 infusion, VTE risk will be assessed via Caprini model, coagulation function, D-dimer, FDP, and TAT;

    Time frame: Five years after infusion

  3. The shedding of GS1191-0445 viral vector: Viral vector titers in serum, saliva, urine, semen and fecal will be monitored;

    Time frame: Five years after infusion

  4. Total FⅧ Antibody Levels: Total FⅧ antibody levels will be measured to determine the immunogenicity of FⅧ expression protein;

    Time frame: Five years after infusion

  5. FVIII inhibitor: Factor Ⅷ inhibitor will be measured to determine the immunogenicity of FⅧ expression protein;

    Time frame: Five years after infusion

  6. The number of dose-limiting toxicity (DLT) events will be determined by the Safety Review Committee (SRC), at least 12 weeks after GS1191-0445 infusion.

    Time frame: Five years after infusion

  7. The Change of Laboratory Values: Change in serum chemistry values including liver function tests, hematology, and urinalysis;

    Time frame: Five years after infusion

  8. Changes for vital signs: Includes sitting blood pressure (mmHg), respiratory rate (breaths/min), body temperature (°C), and pulse rate (beats/min);

    Time frame: Five years after infusion

  9. Changes for physical examination: Includes skin, mucous membranes, lymph nodes, head and neck, chest (heart, lungs), abdomen, muscles, nervous system, spine/extremities;

    Time frame: Five years after infusion

  10. The immunogenicity of AAV capsid protein: Collection of Peripheral Blood Mononuclear Cell (PBMC) and serum samples for vector shedding detection;

    Time frame: Five years after infusion

Secondary outcomes

  1. Vector-derived FⅧ Activity Level: Validated methods will be used to measure vector-derived FⅧ activity, including peak and steady state following GS1191-0445 infusion；

    Time frame: Day 4 to Week 52 after infusion

  2. Total Consumption of Exogenous FⅧ Infusion;

    Time frame: Weeks 3 to 52 and five years after infusion

  3. Annualized Consumption of FⅧ Infusion；

    Time frame: Weeks 3 to 52 and five years after infusion

  4. Number of bleeding events requiring exogenous FⅧ infusion: To assess the number of bleeding events requiring exogenous FⅧ infusion after administration;

    Time frame: Weeks 3 to 52 and five years after infusion

  5. Number of bleeding events: To assess bleeding events, including spontaneous, traumatic and untreated bleeding events after administration

    Time frame: Weeks 3 to 52 and five years after infusion

  6. Number of joint bleeding events: To assess joint bleeding events after administration

    Time frame: Weeks 3 to 52 and five years after infusion

Sponsors and collaborators

Lead sponsor

Gritgen Therapeutics Co., Ltd.

Industry

Registry information

Official study title

A Single-arm, Open-label Study Evaluating the Safety and Efficacy of a Single Dose of GS1191-0445 Injection in Chinese Subjects With Severe Hemophilia A

Important dates

Study start
2023
Primary completion
2024
Study completion
2029
First posted
Apr 23, 2026
Registry last updated
May 6, 2026

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.

Related clinical trials

Published trials that share one or more normalized conditions with this study.