drugset / Trial / NCT03217032

Lentiviral FVIII Gene Therapy

NCT03217032 ↗

Phase 1 Recruiting 10 enrolled Shenzhen Geno-Immune Medical Institute
Non-randomizedSingle-groupOpen-labelTreatment

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.

Timeline

Start
2025-06-01
Primary completion
2027-05-31
Completion
2028-06-01

Drugs

EvaluationDrugModalityDoseRoute
Subject YUVA-GT-F801 Unknown — —

Indications