Clinical Trial: Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiencies

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiencies

Brief Summary:

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.


Detailed Summary:
Sponsor: Taiga Biotechnologies, Inc.

Current Primary Outcome: Adverse Events following transplant with TBX-1400 [ Time Frame: Two years ]

Adverse events from subject or parent reporting or other assessments


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Transplant Engraftment [ Time Frame: Up to Day 180 ]
    Assessment of transplant engraftment will include analysis of T-cells , B-cells and Natural Killer cells
  • Chimerism [ Time Frame: Up to Day 180 ]
    Assessment of chimerism will include analysis of T-cells , B-cells and Natural Killer cells
  • Absolute numbers of T-cells [ Time Frame: Days 30 to 360 ]
  • T-cell receptor excision circles (TREC) [ Time Frame: Days 30 to 360. ]
  • Kappa-deleting recombination excision circles (KREC) [ Time Frame: Days 30 to 360. ]
  • Immunoglobulin (Ig) levels [ Time Frame: Days 30 to 360. ]
  • Immunoglobulin G (IgG) titers to pneumococcal antigens in 13-valent vaccine [ Time Frame: Sixty days after final immunization ]
  • T-cell responses to anti-CD3 and phytohemagglutinin (PHA) [ Time Frame: Days 30, 60, 90, 120, and 180. ]
  • Number of infections following transplant [ Time Frame: Two years ]
  • Number of days granulocyte colony stimulating factor (G-CSF) was administered [ Time Frame: Up to 1 year ]
  • Number of days to specific cell counts and last packed red blood cell (PRBC) transfusion [ Time Frame: Up to 2 years ]


Original Secondary Outcome: Same as current

Information By: Taiga Biotechnologies, Inc.

Dates:
Date Received: July 26, 2016
Date Started: September 2016
Date Completion: March 2020
Last Updated: August 22, 2016
Last Verified: August 2016