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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.
Enrollment
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Inclusion criteria
Signed informed consent of the subject's legally authorized representative (in most cases, this will be the parent or parents),
Age 1 month to 4 years,
SCID, leaky SCID with <100 TRECs, or Omenn syndrome requiring stem cell transplant with conditioning therapy (patients with decreased T-cell numbers by flow cytometry, decreased TREC, and decreased in vitro responses to T cell mitogens will be eligible regardless of B-cell and/or natural killer (NK) cell function),
Identified donor (9 or 10/10 Human Leukocyte Antigen (HLA)-matched unrelated or haplocompatible relative),
Eligible patients must have adequate physical function to tolerate the conditioning regimen and hematopoietic stem cell transplantation (HSCT), as measured by:
Exclusion criteria
The following are NOT exclusion criteria:
Primary purpose
Allocation
Interventional model
Masking
8 participants in 1 patient group
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Central trial contact
Brian Turner, Dr.; Yosef Refaeli, Dr.
Data sourced from clinicaltrials.gov
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