Clinical Trial: Stem Cell Transplantation for Fanconi Anemia

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

Official Title: A Study of Thymic Shielding in Recipients of Total Body Irradiation, Cyclophosphamide, and Fludarabine Followed by Alternate Donor Hematopoietic Stem Cell Transplantation in Patients With

Brief Summary: The purpose of this study is to determine whether thymic shielding during total body irradiation can be given and whether it will reduce the risk of infections in Fanconi Anemia patients undergoing alternate donor (not a matched sibling) stem cell transplants.

Detailed Summary: All subjects will be given the same treatment regimen of total body irradiation (TBI), Fludarabine, Cyclophosphamide, and anti-thymocyte globulin (ATG), followed by an alternate donor stem cell transplant. Since this treatment regimen has been given before, without thymic shielding, we will compare the outcomes of these patients with the historical data from subjects who did not receive thymic shielding.
Sponsor: Masonic Cancer Center, University of Minnesota

Current Primary Outcome: Number of Patients Who Exhibited Hematopoietic Recovery and Engraftment [ Time Frame: Day 42 after hematopoietic cell transplant ]

Original Primary Outcome: incidence of hematopoietic recovery and engraftment at day 42 after HCT

Current Secondary Outcome:

  • Number of Patients Who Exhibited Secondary Graft Failure [ Time Frame: Day 100 after hematopoietic cell transplant ]
  • Number of Patients With Acute Graft Versus-Host Disease (aGVHD) [ Time Frame: Day 100 after hematopoietic cell transplant ]
  • Number of Patients With Chronic Graft Versus-Host Disease (GVHD) [ Time Frame: 1 year after hematopoietic cell transplant ]
  • Number of Patients Who Exhibited Regimen-related Toxicity (RRT) [ Time Frame: 1 year after hematopoietic cell transplant ]
  • Immune Reconstitution - Mean Value (1 Year) [ Time Frame: 1 year post-transplant. ]
  • Immune Reconstitution - Mean Value (2 Years) [ Time Frame: at 2 years after transplant ]
  • Number of Patients Alive at 1 Year [ Time Frame: 1 year after transplant ]
  • Number of Patients Alive at 2 Years [ Time Frame: 2 years after transplant ]


Original Secondary Outcome:

  • - Incidence of secondary graft failure at day 100 after HCT
  • - Incidence of acute graft-versus-host disease (GVHD) at 100 days after HCT
  • - Incidence of chronic GVHD at 1 year after HCT
  • - Incidence of regimen-related toxicity (RRT) at 1 year after HCT
  • - Immune reconstitution over the first two years after HCT
  • 2.2.6 Probability of survival at 1 and 2 years after HCT


Information By: Masonic Cancer Center, University of Minnesota

Dates:
Date Received: September 9, 2005
Date Started: March 2004
Date Completion:
Last Updated: November 30, 2009
Last Verified: November 2009