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

NCT07548411 | Active Not Recruiting Phase 1

• 1 other identifier: GS1191-0445-GTHA-CN01
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Severe Hemophilia A

Outcome Measures

Primary Outcomes (10)

• 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);

  Five years after infusion

• 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;

  Five years after infusion

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

  Five years after infusion

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

  Five years after infusion

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

  Five years after infusion

• 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.

  Five years after infusion

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

  Five years after infusion

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

  Five years after infusion

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

  Five years after infusion

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

  Five years after infusion

Secondary Outcomes (6)

• 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；

  Day 4 to Week 52 after infusion

• Total Consumption of Exogenous FⅧ Infusion;

  Weeks 3 to 52 and five years after infusion

• Annualized Consumption of FⅧ Infusion；

  Weeks 3 to 52 and five years after infusion

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

  Weeks 3 to 52 and five years after infusion

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

  Weeks 3 to 52 and five years after infusion

Study Arms (1)

3E12 vg/kg

EXPERIMENTAL

Drug: GS1191-0445 injection

Interventions

GS1191-0445 injection DRUG

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

3E12 vg/kg

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Gritgen Therapeutics Co., Ltd.: lead

Study Sites (2)

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

Wuhan, Hubei, 430022, China

Location

Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College

Tianjin, Tianjin Municipality, 300020, China

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

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

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 26, 2026
• First Posted: April 23, 2026
• Study Start: July 10, 2023
• Primary Completion: December 31, 2024
• Study Completion (Estimated): December 23, 2029
• Last Updated: May 6, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (2)