Stopped: A leukaemia case was reported in patient treated with a similar vector. For safety risk we stopped recruitment
Patient stem cells will be mobilized with induction chemotherapy (R)-ICE and G-CSF. If sufficient cells can be mobilized, patients will be treated with high-dose chemotherapy and a transplant of autologous CD34+ cells transduced with an antiviral vector (M87o). If autologous CD34+ yield is insufficient, allogeneic gene-modified cells will be given, if a compatible donor is available. To minimize risk of transplant failure, a second unmodified CD34+ cell transplant will be given one week after the first transplant.
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Adverse events, ECOG performance status and laboratory safety tests
Timeframe: five years after transplantation