3
Participants
Start Date
March 31, 2016
Primary Completion Date
June 30, 2034
Study Completion Date
June 30, 2034
X vivo gene therapy
Transplantation of patient's autologous CD34+ cells transduced with lentiviral vector containing XCGD gene. The investigational product is patient-specific and corresponds to autologous CD34+ cells transduced ex vivo with the G1XCGD vector. These transduced cells will be cryopreserved until safety testing and infusion into the patient.
Hôpital Necker Enfants Malades, Paris
Lead Sponsor
Genethon
OTHER