Clinical Trial: Stem Cell Transplant for Inborn Errors of Metabolism

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

Official Title: Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Bone Marrow Transplantation

Brief Summary: The purpose of this study is to determine the safety and engraftment of donor hematopoietic cells using this conditioning regimen in patients undergoing a hematopoietic (blood forming) cell transplant for an inherited metabolic storage disease.

Detailed Summary:

Prior to transplantation, subjects will receive Busulfan intravenously (IV) via the Hickman line four times daily for 4 days, Cyclophosphamide intravenously via the Hickman line once a day for 4 days, and Anti-Thymocyte Globulin (ATG) intravenously (IV) via the Hickman line twice daily for three days before the transplant. These three drugs are being given to subjects to help the new marrow "take" and grow.

On the day of transplantation, the donor's hematopoietic cells will be transfused via central venous catheter.

After hematopoietic cell transplant, subjects will then receive two drugs, cyclosporin and either methylprednisolone or Mycophenolate Mofetil (MMF). Cyclosporin and methylprednisolone or MMF are given to help prevent the complication of graft-versus-host disease and to decrease the chance that the new donor cells will be rejected.


Sponsor: Masonic Cancer Center, University of Minnesota

Current Primary Outcome: Overall Survival [ Time Frame: 100 Days, 1 Year and 3 Years ]

Number of patients alive at designated timepoints after transplant.


Original Primary Outcome: We will evaluate whether patients treated by bone marrow, peripheral blood, or umbilical cord blood transplantation after March of 2001 have equivalent or better outcome than historical controls with BMT for these patients over the last 5 years.

Current Secondary Outcome:

  • Overall Donor Engraftment [ Time Frame: Day 100 ]
    Number of patients with full donor chimerism (state in bone marrow transplantation in which bone marrow and host cells exist compatibly without signs of graft-versus-host rejection disease) by Day 100 post-transplant of at least 90%.
  • Number of Patients With Grade II-IV Acute Graft-Versus-Host Disease [ Time Frame: Day 100 ]
    Number of patients who exhibited acute graft-versus-host disease by Day 100 post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Grade I=mild, Grade II=moderate, Grade III=severe, Grade IV=life threatening.
  • Number of Patients With Grade III-IV Acute Graft-Versus-Host Disease [ Time Frame: Day 100 ]
    Number of patients who exhibited acute graft-versus-host disease by Day 100 post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Grade I=mild, Grade II=moderate, Grade III=severe, Grade IV=life threatening.
  • Number of Patients With Chronic Graft-Versus-Host Disease [ Time Frame: 1 Year Post Transplant ]
    Number of patients who exhibited chronic graft-versus-host disease by 1 Year post transplant. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted material attacks the transplant recipient's body. Chronic GVHD is an extension of this syndrome.


Original Secondary Outcome:

  • estimate survival at 100 days, 1 year and 3 years.
  • estimate change in neuropsychometric function at 6 months, 1 year, 2 years and 3 years.
  • estimate the toxicity of hematopoietic cell transplant therapy:
  • estimate the rate of hematological donor cell engraftment.
  • estimate the incidence of graft-versus-host disease (GVHD).


Information By: Masonic Cancer Center, University of Minnesota

Dates:
Date Received: September 12, 2005
Date Started: January 1995
Date Completion:
Last Updated: November 6, 2012
Last Verified: November 2012