Stopped: voluntary hold
SCID-X1 is a genetic disorder of blood cells caused by DNA changes in a gene that is required for the normal development of the human immune system. The purpose of this study is to determine if a new method, called lentiviral gene transfer, can be used to treat SCID-X1. This method involves transferring a normal copy of the common gamma chain gene into the participant's bone marrow stem cells. The investigators want to determine if the procedure is safe, whether it can be done according to the methods they have developed, and whether the procedure will provide a normal immune system for the patient. It is hoped that this type of gene transfer may offer a new way to treat children with SCID-X1 that do not have a brother or sister who can be used as a donor for stem cell transplantation.
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Number of patients with adequate cell collection and processing
Timeframe: Day 0
Number of patients with adequate neutrophil count recovery after busulfan conditioning
Timeframe: Day 42 post gene transfer
Number of patients without Grade 4 adverse event (AE)
Timeframe: 42 days post gene transfer
Number of patients with successful reconstitution
Timeframe: 42 days post gene transfer
Number of patients with treatment failure
Timeframe: 42 days post gene transfer