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

December 5, 2025 updated by: Gritgen Therapeutics Co., Ltd.

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

50

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • China
    • Anhui
      • Hefei, Anhui, China, 230001
        • Not yet recruiting
        • Anhui Provincial Hospital
        • Contact:
    • Beijing Municipality
      • Beijing, Beijing Municipality, China, 100005
        • Not yet recruiting
        • Peking Union Medical College Hospital
        • Contact:
          • Tienan Zhu, Doctor
          • Phone Number: +8618611743292
          • Email: Zhutn@pumch.cn
    • Gansu
      • Lanzhou, Gansu, China, 730099
        • Not yet recruiting
        • First Hospital of Lanzhou University
        • Contact:
    • Guangdong
      • Guangzhou, Guangdong, China, 510515
        • Not yet recruiting
        • Nanfang Hospital Southern Medical University
        • Contact:
    • Hebei
      • Tangshan, Hebei, China, 063000
        • Not yet recruiting
        • North China University of Science and Technology Affiliated Hospital
        • Contact:
    • Henan
      • Zhengzhou, Henan, China, 450003
        • Not yet recruiting
        • Henan Cancer Hospital
        • Contact:
    • Hubei
      • Wuhan, Hubei, China, 430022
        • Not yet recruiting
        • Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
        • Contact:
    • Hunan
      • Changsha, Hunan, China, 410028
        • Not yet recruiting
        • Xiangya Hospital of Central South University
        • Contact:
    • Jiangsu
      • Nanjing, Jiangsu, China, 210000
        • Not yet recruiting
        • Nanjing Drum Tower Hospital, the Affiliated Hospital of Nanjing University Medical School
        • Contact:
    • Jiangxi
      • Nanchang, Jiangxi, China, 344001
        • Not yet recruiting
        • Jiangxi Provincial People's Hospital
        • Contact:
    • Shandong
      • Jinan, Shandong, China, 250013
        • Recruiting
        • Central Hospital Affiliated to Shandong First Medical University
        • Contact:
    • Tianjin Municipality
      • Tianjin, Tianjin Municipality, China, 300020
        • Recruiting
        • Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College
        • Contact:
    • Yunnan
      • Kunming, Yunnan, China, 650101
        • Not yet recruiting
        • The Second Affiliated Hospital of Kunming Medical University
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 3E12 vg/kg
Drug: GS1191-0445 injection
A single intravenous administration of GS1191-0445 injection at a dose of 3E12 vg/kg

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Weeks 3 to 52 after infusion

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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)
Within 52 weeks after infusion
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
Within 52 weeks after infusion
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
2-5 years after infusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Gritgen Therapeutics Co., Ltd.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 31, 2025

Primary Completion (Estimated)

November 30, 2026

Study Completion (Estimated)

November 30, 2030

Study Registration Dates

First Submitted

January 28, 2025

First Submitted That Met QC Criteria

February 13, 2025

First Posted (Actual)

February 19, 2025

Study Record Updates

Last Update Posted (Estimated)

December 9, 2025

Last Update Submitted That Met QC Criteria

December 5, 2025

Last Verified

September 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GS1191-0445-GTHA-CN02

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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