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ACTIVE NOT RECRUITING
NCT07548411
PHASE1/PHASE2

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

Sponsor: Gritgen Therapeutics Co., Ltd.

View on ClinicalTrials.gov

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.

Official 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

Key Details

Gender

MALE

Age Range

18 Years - 65 Years

Study Type

INTERVENTIONAL

Enrollment

7

Start Date

2023-07-10

Completion Date

2029-12-23

Last Updated

2026-05-06

Healthy Volunteers

No

Interventions

DRUG

GS1191-0445 injection

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

Locations (2)

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

Wuhan, Hubei, China

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

Tianjin, Tianjin Municipality, China