Lentiviral FVIII Gene Therapy
NCT03217032 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 10
Last updated 2026-04-24
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.
Conditions
Interventions
- BIOLOGICAL
-
YUVA-GT-F801
Lentiviral factor VIII gene modified autologous hematopoeitic stem cells and mesenchymal stem cells
Sponsors & Collaborators
-
Shenzhen Geno-Immune Medical Institute
lead OTHER
Principal Investigators
-
Lung-Ji Chang · Shenzhen Geno-Immune Medical Institute
Study Design
- Allocation
- NON_RANDOMIZED
- Purpose
- TREATMENT
- Masking
- NONE
- Model
- SINGLE_GROUP
Eligibility
- Min Age
- 2 Years
- Max Age
- 65 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
- Start
- 2025-06-01
- Primary Completion
- 2027-05-31
- Completion
- 2028-06-01
Countries
- China
Study Locations
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