NCT06833983

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

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

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 50 (estimated)
• Sponsor: Gritgen Therapeutics Co., Ltd. · Industry
• Sex: Male
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Hemophilia A

Interventions

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

Timeline

• Start date: 2025-03-31
• Primary completion: 2026-11-30
• Completion: 2030-11-30
• First posted: 2025-02-19
• Last updated: 2025-12-09

Locations

13 sites across 1 country: China