Clinical Trial: Stem Cell Transplantation for Hurler

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

Official Title: Hematopoietic Stem Cell Transplantation for Hurler Syndrome, Maroteaux Lamy Syndrome (MPS VI), and Alpha Mannosidase Deficiency (Mannosidosis)

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 Hurler syndrome, Maroteaux Lamy syndrome, Mannosidosis, or I-cell 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 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: Mean Percentage of Donor Cells in Study Population (Chimerism). [ Time Frame: at 21 days, 42 days, 60 days, 100 days, 6 months, and 1 year ]

Donor-derived engraftment determined by restriction fragment length polymorphism (RFLP).


Original Primary Outcome: estimate donor-derived engraftment by RFLP and a-L-iduronidase enzyme (Hurler syndrome), arylsulfatase B (Maroteaux Lamy syndrome), or Alpha Mannosidase (Mannosidosis) enzyme activity levels at 21 days, 42 days, 60 days, 100 days, 6 months, and 1 year

Current Secondary Outcome:

  • Number of Patients Surviving on Study [ Time Frame: at 100 days, 1 year, and 3 years post transplant ]
    Number of patients surviving (alive) at specified timepoints.
  • Number of Patients Who Failed Engraftment. [ Time Frame: Day 42 Post Transplant ]
    Toxicity (undesireable effect) of hematologic donor cell engraftment is determined by failure to engraft at Day 42.
  • Number of Patients With Grade III-IV Acute Graft-versus-host Disease (aGVHD). [ Time Frame: Day 100 Post Transplant ]
    Toxicity (undesireable effect) of this stem cell transplant preparative regimen due to acute graft-versus-host disease.


Original Secondary Outcome: estimate survival at 100 days, 1 year, and 3 years post transplant.

Information By: Masonic Cancer Center, University of Minnesota

Dates:
Date Received: September 12, 2005
Date Started: May 1999
Date Completion:
Last Updated: November 6, 2012
Last Verified: November 2012