Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A
Status:
Not yet recruiting
Trial end date:
2039-04-01
Target enrollment:
Participant gender:
Summary
This is a first-in-human, non-randomized, open label, single treatment, Phase 1 study in
approximately 7 patients with severe hemophilia A. The study will evaluate gene therapy by
transplantation of autologous CD34+ hematopoietic stem cells transduced ex vivo with the
CD68-ET3 lentiviral vector.