Evaluating GS1191-0445 Injection for Hemophilia A Treatment

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

This study is testing a new gene therapy injection for hemophilia A to see if it can safely help men aged 18 to 65 by improving their blood clotting.

Quick facts

• Phase: PHASE3
• Study type: Interventional
• Enrollment: 50 (estimated)
• Ages: 18 Years to 65 Years
• Sex: Male
• Sponsor: Gritgen Therapeutics Co., Ltd. (industry)
• Locations: 13 sites (Hefei, Anhui and 12 other locations)
• Trial ID: NCT06833983 on ClinicalTrials.gov

What this trial studies

This clinical trial is a single-arm, open-label, multicenter evaluation of GS1191-0445 injection, a gene therapy designed to treat hemophilia A in male subjects aged 18 to 65. The study aims to assess the efficacy and safety of a single intravenous dose of GS1191-0445, which utilizes an AAV8-based vector to express human factor VIII in the liver. Participants will be monitored for their response to the treatment and any potential side effects.

Who should consider this trial

Good fit: Ideal candidates for this study are male adults aged 18 to 65 with confirmed hemophilia A who have previously used FVIII products.

Not a fit: Patients with other hemorrhagic disorders or those with abnormal liver function tests will not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the management of hemophilia A by providing a long-lasting solution for patients.

How similar studies have performed: Other studies involving gene therapy for hemophilia have shown promising results, indicating potential success for this approach.

Eligibility criteria

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.

Where this trial is running

Hefei, Anhui and 12 other locations

• Anhui Provincial Hospital — Hefei, Anhui, China (NOT_YET_RECRUITING)
• Peking Union Medical College Hospital — Beijing, Beijing Municipality, China (NOT_YET_RECRUITING)
• First Hospital of Lanzhou University — Lanzhou, Gansu, China (NOT_YET_RECRUITING)
• Nanfang Hospital Southern Medical University — Guangzhou, Guangdong, China (NOT_YET_RECRUITING)
• North China University of Science and Technology Affiliated Hospital — Tangshan, Hebei, China (NOT_YET_RECRUITING)
• Henan Cancer Hospital — Zhengzhou, Henan, China (NOT_YET_RECRUITING)
• Union Hospital, Tongji Medical College, Huazhong University of Science and Technology — Wuhan, Hubei, China (NOT_YET_RECRUITING)
• Xiangya Hospital of Central South University — Changsha, Hunan, China (NOT_YET_RECRUITING)
• Nanjing Drum Tower Hospital, the Affiliated Hospital of Nanjing University Medical School — Nanjing, Jiangsu, China (NOT_YET_RECRUITING)
• Jiangxi Provincial People's Hospital — Nanchang, Jiangxi, China (NOT_YET_RECRUITING)
• Central Hospital Affiliated to Shandong First Medical University — Jinan, Shandong, China (RECRUITING)
• Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences & Peking Union Medical College — Tianjin, Tianjin Municipality, China (RECRUITING)
• The second Affiliated Hospital of Kunming Medical University — Kunming, Yunnan, China (NOT_YET_RECRUITING)

Study contacts

• Study coordinator: Yanfang Li

Conditions: Hemophilia A