Lentiviral FVIII Gene Therapy
Lentiviral FVIII Gene Therapy for Hemophilia A
Sponsor: Shenzhen Geno-Immune Medical Institute
This observational or N/A phase trial investigates Hemophilia A and is currently actively recruiting participants. Shenzhen Geno-Immune Medical Institute leads this study, which shows 8 recorded versions since 2025 — indicating limited longitudinal coverage. The change history captured here reflects the iterative nature of clinical trial conduct.
Study Description(click to expand)Hemophilia A is a genetic bleeding disorder caused by the lack of ability to produce blood-clotting factor VIII (FVIII). Individuals with hemophilia A suffer repeated bleeding episodes, which can cause chronic joint disease and sometimes even death due to the inability for blood to clot efficiently. The current treatment is intravenous infusion of clotting factor concentrates, either prophylactically or in response to bleeding. The procedure is life time long and expensive while still cannot achieve a cure.Gene therapy is a novel technology that has been successfully demonstrated in a number of clinical studies for diseases such as cancer and genetic diseases. In this study, an advanced lentiviral vector system NHP/TYF will be used to deliver a functional FVIII gene to overcome human clotting FVIII gene defect in patients with hemophilia A. This study is a Phase I trial evaluating the safety and efficacy for infusion of gene modified autologous stem cells in patients with hemophilia A.
Hemophilia A is a genetic bleeding disorder caused by the lack of ability to produce blood-clotting factor VIII (FVIII). Individuals with hemophilia A suffer repeated bleeding episodes, which can cause chronic joint disease and sometimes even death due to the inability for blood to clot efficiently. The current treatment is intravenous infusion of clotting factor concentrates, either prophylactically or in response to bleeding. The procedure is life time long and expensive while still cannot achieve a cure.Gene therapy is a novel technology that has been successfully demonstrated in a number of clinical studies for diseases such as cancer and genetic diseases. In this study, an advanced lentiviral vector system NHP/TYF will be used to deliver a functional FVIII gene to overcome human clotting FVIII gene defect in patients with hemophilia A. This study is a Phase I trial evaluating the safety and efficacy for infusion of gene modified autologous stem cells in patients with hemophilia A.
Status Flow
Change History
8 versions recorded-
Apr 28, 2026 — Present [daily]
Recruiting
Status: Unknown → Recruiting · Phase: PHASE1 → None
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Sep 2024 — Apr 2026 [monthly]
Unknown PHASE1
-
Jul 2024 — Sep 2024 [monthly]
Unknown PHASE1
Status: Unknown Status → Unknown
-
Jul 2022 — Jul 2024 [monthly]
Unknown Status PHASE1
Status: Not Yet Recruiting → Unknown Status
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Jan 2021 — Jul 2022 [monthly]
Not Yet Recruiting PHASE1
▶ Show 3 earlier versions
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Jun 2019 — Jan 2021 [monthly]
Not Yet Recruiting PHASE1
Phase: PHASE1_PHASE2 → PHASE1
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Jun 2018 — Jun 2019 [monthly]
Not Yet Recruiting PHASE1_PHASE2
Status: Enrolling By Invitation → Not Yet Recruiting
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Aug 2017 — Jun 2018 [monthly]
Enrolling By Invitation PHASE1_PHASE2
First recorded
Eligibility Summary
This study is a Phase I trial using an advanced lentiviral vector to deliver a functional gene for human clotting factor VIII into patients with hemophilia A, to evaluate the safety and efficacy of infusion of lentiviral gene modified autologous stem cells in patients.
Contact Information
- Shenzhen Geno-Immune Medical Institute
For direct contact, visit the study record on ClinicalTrials.gov .