Clinical Trial: Stem Cell Transplant for Immunologic or Histiocytic Disorders

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Allogeneic Hematopoietic Stem Cell Transplant for Patients With Immunologic or Histiocytic Disorders Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed Chimerism

Brief Summary:

This study tests the clinical outcomes of a preparative regimen of fludarabine (FLU), anti-thymocyte globulin (ATG)/or Campath, and melphalan; followed by hematopoietic stem cell transplant, and a post transplant regimen of Cyclosporin A (CsA) in patients with immunologic or histiocytic disorders. The researchers hypothesize that this regimen will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease (GVHD).

Patients will be randomized biologically into one of 3 arms based upon donor availability: (a) human leukocyte antigen (HLA) genotypic matched sibling donor, (b) HLA phenotypic matched unrelated peripheral blood stem cell (PBSC) donor, (c) two HLA 0-2 antigen mismatched unrelated cord blood donors (double cord).


Detailed Summary:

Prior to transplantation, subjects will receive Melphalan, Fludarabine and Anti-Thymocyte Globulin (ATG) or Campath. These three drugs are being given to subjects to help the new stem cells take and grow. On the day of transplantation, subjects will receive stem cells transfused via intravenous (IV) catheter.

After stem cell transplantation, subjects will be given Cyclosporin A (CsA) and mycophenolate mofetil (MMF) to reduce the risk of graft-versus-host disease, the complication that occurs when the donor's stem cells react against the patient.


Sponsor: Masonic Cancer Center, University of Minnesota

Current Primary Outcome: Number of Subjects With Mixed Chimerism [ Time Frame: Day 100 ]

>10% Donor Cells at Day 100


Original Primary Outcome: To demonstrate the safety and the ability to establish stable mixed chimerism (>10% donor cells at day 100) using a nonmyeloablative preparative regimen in a phase 2 pilot trial

Current Secondary Outcome:

  • Percentage of Donor Chimerism at 100 Days [ Time Frame: Day 100 ]
    The percent of recipient bone marrow and blood cells that are of donor origin.
  • Percentage of Donor Chimerism at 180 Days [ Time Frame: Day 180 ]
    The percent of recipient bone marrow and blood cells that are of donor origin.
  • Percentage of Donor Chimerism at 365 Days [ Time Frame: Day 365 ]
    The percent of recipient bone marrow and blood cells that are of donor origin.
  • Incidence of Grade 2-4 Acute Graft Versus Host Disease (aGVHD) [ Time Frame: Day 100 ]
    Acute graft versus host disease (aGVHD) is a reaction occurring within the first 100 days after transplant where the T- cells of the donor graft attacks the recipient's (host's) skin, GI tract, liver and other organs. The severity of aGVHD is graded on a scale of 1 - 4 with the highest number representing the most severe disease.
  • Incidence of Grade 3-4 Acute Graft Versus Host Disease (aGVHD) [ Time Frame: Day 100 ]
    Acute graft versus host disease (aGVHD) is a reaction occurring within the first 100 days after transplant where the T- cells of the donor graft attacks the recipient's (host's) skin, GI tract, liver and other organs. The severity of aGVHD is graded on a scale of 1 - 4 with the highest number representing the most severe disease.
  • Incidence of Chronic Graft Versus Host Disease (cGVHD) [ Time Frame: 6 months and 1 year ]
    Chronic graft versus host disease (cGVHD) is a reaction which typically develops 3 to 6 months after transplant where the T- cells of the donor graft attacks the recipient's (host's) skin, GI tract, liver and other organs.
  • Number of Subjects Alive at 100 Days [ Time Frame: Day 100 ]
  • Number of Subjects Alive at One Year [ Time Frame: Day 365 ]
  • Compare Quality of Life (QOL) [ Time Frame: Pretransplant, 1 year, 2 years and 5 years ]


Original Secondary Outcome:

  • Determine the incidence of chimerism at 100 days, 6 months and 1 year.
  • Determine the incidence of grade 2-4 and 3-4 acute GVHD at 100 days.
  • Determine the incidence of chronic GVHD at 6 months and 1 year.
  • Compare the quality of life (QOL) at 1, 2 and 5 years with the pre-transplant assessment.
  • Determine overall survival at 100 days and 1 year.


Information By: Masonic Cancer Center, University of Minnesota

Dates:
Date Received: September 12, 2005
Date Started: August 2002
Date Completion:
Last Updated: March 3, 2017
Last Verified: October 2014