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

Официальное название

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

Обзор

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.

Вмешательства

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

Первичные конечные точки

• Annualized Bleeding Rate (ABR) [Срок оценки: Weeks 3 to 52 after infusion]

Вторичные конечные точки (3)

• Incidence of adverse events (AE) [Срок оценки: Within 52 weeks after infusion]
• Immunogenic response of AAV8 capsid protein and vector derived FVIII within 52 weeks after GS1191-0445 infusion [Срок оценки: Within 52 weeks after infusion]
• Evaluate the long-term efficacy of GS1191-0445 injection after intravenous infusion for 2-5 years [Срок оценки: 2-5 years after infusion]

Критерии участия

Критерии включения

• Understand the purpose and risks of the study and provide informed consent in accordance with national and local privacy laws:
• Subject must be male, aged >18 years old at the time of signing informed consent, and ≤65 years old:
• Participants with confirmed hemophilia A in their pre-admission history and based on clinical laboratory examination ;
• Subjects had used FVIII products for at least 150 exposure days (ED) before enrollment;
• Subject has no prior history of FVIII inhibitors;
• Subjects agree to use a reliable barrier contraceptive method from the date of signing the informed consent
• Subject is willing and able to follow planned visits, treatment plans, and other study procedures.

Критерии исключения

• The subject has any hemorrhagic disorder not related to hemophilia A,
• Abnormal liver function test results of subjects during screening.
• Abnormal laboratory examination of subjects during screening
• 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;
• Active systemic immune disease.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Китай · 13 центров

• Anhui Provincial Hospital — Хэфэй
• Peking Union Medical College Hospital — Пекин
• First Hospital of Lanzhou University — Lanzhou
• Nanfang Hospital Southern Medical University — Гуанчжоу
• North China University of Science and Technology Affiliated Hospital — Tangshan
• Henan Cancer Hospital — Чжэнчжоу
• Union Hospital, Tongji Medical College, Huazhong University of Science and Technology — Ухань
• Xiangya Hospital of Central South University — Чанша
• … и ещё 5 центров

Идентификаторы

NCT: NCT06833983 · GS1191-0445-GTHA-CN02

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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