Severe Combined Immunodeficiency Clinical Trial
Official title:
Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency
This is a study of stem cell transplantation with TBX-1400 in pediatric subjects with severe
combined immunodeficiency (SCID).
The donor cells are exposed to a protein that has been shown in the laboratory to improve the
ability of the donor cells to make blood and immune cells after transplant. Exposure of the
donor cells to this protein does not modify the genes in the cells in any way.
This study has two goals. The first goal is to find out if transplant with TBX-1400 is safe.
The second goal is to find out what effects TBX-1400 stem cells have on time to engraftment
in pediatric subjects with SCID. The study hypothesis is that TBX-1400 cells will shorten the
time to immune reconstitution after transplant.
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