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ET3-201 Gene Therapy for Severe Hemophilia A
NCT04418414Phase 1Not yet recruiting
- Sponsor
- Expression Therapeutics
- Start
- Sep 2024
- Primary completion
- Aug 2029
- Modality
- Gene Editing, Gene Silencing, Lentiviral Vectors
01
Overview
Phase 1, open-label, single-arm trial of autologous CD34+ peripheral blood stem cells transduced with a lentiviral vector encoding a novel coagulation factor VIII transgene in hemophilia A, sponsored by Expression Therapeutics. Primary endpoints: Number of study participants experiencing serious adverse events at 12 weeks, Severity of serious adverse events at 12 weeks and 1 more. Planned start Sep 2024; status: not yet recruiting.
02
Drugs
autologous CD34+ peripheral blood stem cells transduced with a lentiviral vector encoding a novel coagulation factor VIII transgene
03
Conditions
hemophilia A
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