Clinical Trial: T-Cell Depletion and Stem Cell Transplant for Immune Deficiencies and Histiocytic Disorders

Study Status: Completed
Recruit Status: Unknown status
Study Type: Interventional

Official Title: In-vivo T-cell Depletion and Hematopoietic Stem Cell Transplantation for Life-Threatening Immune Deficiencies and Histiocytic Disorders

Brief Summary: The hypothesis is to determine if a preparative regimen of busulfan, cyclophosphamide, and antithymocyte globulin (ATG) plus allogeneic stem cell transplantation will be effective in the treatment of immune deficiencies and histiocytic disorders.

Detailed Summary:

Subjects will begin chemotherapy as a preparative regimen, which is intended to completely eliminate their defective immune system and bone marrow. The preparative regimen consists of the chemotherapy drugs (busulfan, cyclophosphamide, and antithymocyte globulin (ATG)).

Transplantation: subjects will then have a source of blood stem cells (bone marrow) from their donor administered into their catheter. Medication will be given to help prevent Graft-Versus Host Disease (GVHD). The ATG will help to deplete the donor stem cells of the type of cells that can cause GVHD and will also help to promote engraftment of the new stem cells.

Recovery Phase: The second phase of treatment consists of a period after transplantation during which we wait for the return of bone marrow function. This usually takes two to four weeks. Subjects will be given a blood cell growth factor, G-CSF, to help speed recovery of the white blood cells and potentially decrease the risk of infection and decrease the time until the bone marrow recovers.


Sponsor: Masonic Cancer Center, University of Minnesota

Current Primary Outcome: Time to Transplant Engraftment [ Time Frame: Day 100 Post Transplant ]

Original Primary Outcome:

  • Time to engraftment and incidence of graft failure
  • Incidence of grade II-IV graft vs. host disease


Current Secondary Outcome:

  • Number of Patients With Treatment Related Mortality. [ Time Frame: Day 100 Post Transplant ]
  • Number of Patients Surviving (Disease-free) [ Time Frame: 1 year ]
  • Number of Patients With Grade II-IV Graft-Versus-Host Disease (GVHD) [ Time Frame: Day 100 Post Transplant ]
  • Number of Patients With Graft Failure [ Time Frame: Day 100 Post transplant ]
  • Number of Patients With III-IV Graft-Versus-Host Disease (GVHD) [ Time Frame: Day 100 Post Transplant ]
  • Number of Patients Surviving (Disease-free) [ Time Frame: 3 years ]


Original Secondary Outcome:

  • Incidence of grade 3 and 4 regimen related toxicity
  • Estimate of disease-free survival


Information By: Masonic Cancer Center, University of Minnesota

Dates:
Date Received: September 12, 2005
Date Started: September 2000
Date Completion: July 2015
Last Updated: April 22, 2015
Last Verified: April 2015