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RECRUITING
NCT06833983
PHASE3

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

Sponsor: Gritgen Therapeutics Co., Ltd.

View on ClinicalTrials.gov

Summary

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.

Official 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

Key Details

Gender

MALE

Age Range

18 Years - 65 Years

Study Type

INTERVENTIONAL

Enrollment

50

Start Date

2025-03-31

Completion Date

2030-11-30

Last Updated

2025-12-09

Healthy Volunteers

No

Conditions

Interventions

DRUG

GS1191-0445 injection

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

Locations (13)

Anhui Provincial Hospital

Hefei, Anhui, China

Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

First Hospital of Lanzhou University

Lanzhou, Gansu, China

Nanfang Hospital Southern Medical University

Guangzhou, Guangdong, China

North China University of Science and Technology Affiliated Hospital

Tangshan, Hebei, China

Henan Cancer Hospital

Zhengzhou, Henan, China

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

Wuhan, Hubei, China

Xiangya Hospital of Central South University

Changsha, Hunan, China

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

Nanjing, Jiangsu, China

Jiangxi Provincial People's Hospital

Nanchang, Jiangxi, China

Central Hospital Affiliated to Shandong First Medical University

Jinan, Shandong, China

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

Tianjin, Tianjin Municipality, China

The second Affiliated Hospital of Kunming Medical University

Kunming, Yunnan, China