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

NCT ID: NCT06833983

Last Updated: 2025-12-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE3

Total Enrollment

50 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-03-31

Study Completion Date

2030-11-30

Brief Summary

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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.

Detailed Description

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Conditions

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Hemophilia A

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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3E12 vg/kg

Group Type EXPERIMENTAL

GS1191-0445 injection

Intervention Type DRUG

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

Interventions

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GS1191-0445 injection

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

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Gritgen Therapeutics Co., Ltd.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Anhui Provincial Hospital

Hefei, Anhui, China

Site Status NOT_YET_RECRUITING

Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

Site Status NOT_YET_RECRUITING

First Hospital of Lanzhou University

Lanzhou, Gansu, China

Site Status NOT_YET_RECRUITING

Nanfang Hospital Southern Medical University

Guangzhou, Guangdong, China

Site Status NOT_YET_RECRUITING

North China University of Science and Technology Affiliated Hospital

Tangshan, Hebei, China

Site Status NOT_YET_RECRUITING

Henan Cancer Hospital

Zhengzhou, Henan, China

Site Status NOT_YET_RECRUITING

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

Wuhan, Hubei, China

Site Status NOT_YET_RECRUITING

Xiangya Hospital of Central South University

Changsha, Hunan, China

Site Status NOT_YET_RECRUITING

Nanjing Drum Tower Hospital, the Affiliated Hospital of Nanjing University Medical School

Nanjing, Jiangsu, China

Site Status NOT_YET_RECRUITING

Jiangxi Provincial People's Hospital

Nanchang, Jiangxi, China

Site Status NOT_YET_RECRUITING

Central Hospital Affiliated to Shandong First Medical University

Jinan, Shandong, China

Site Status RECRUITING

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

Tianjin, Tianjin Municipality, China

Site Status RECRUITING

The second Affiliated Hospital of Kunming Medical University

Kunming, Yunnan, China

Site Status NOT_YET_RECRUITING

Countries

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China

Central Contacts

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Yanfang Li

Role: CONTACT

+8613817454595

Facility Contacts

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Changcheng Zheng

Role: primary

+8613956961162

Tienan Zhu, Doctor

Role: primary

+8618611743292

Yaming Xi

Role: primary

+8613919110815

Jing Sun

Role: primary

+8613316202696

Zhenyu Yan

Role: primary

+8618831508262

Hu Zhou

Role: primary

+8613939068863

Yu Hu

Role: primary

+8613986183871

Xielan Zhao

Role: primary

+8613707489198

Rongfu Zhou

Role: primary

+8613605142342

Chenghao Jin

Role: primary

+8613699500207

Yun Chen

Role: primary

+8613370582720

Lei Zhang, Doctor

Role: primary

+8613502118379

Zeping Zhou

Role: primary

+8618788571605

Other Identifiers

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GS1191-0445-GTHA-CN02

Identifier Type: -

Identifier Source: org_study_id

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