Hematopoietic Stem Cell Transplantation for Hurler Syndrome, Maroteaux Lamy Syndrome (MPS VI), and Alpha Mannosidase Deficiency (Mannosidosis)
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.
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Mean Percentage of Donor Cells in Study Population (Chimerism).
Donor-derived engraftment determined by restriction fragment length polymorphism (RFLP).
at 21 days, 42 days, 60 days, 100 days, 6 months, and 1 year
Paul Orchard, MD
Masonic Cancer Center, University of Minnesota
United States: Institutional Review Board
|Masonic Cancer Center, University of Minnesota||Minneapolis, Minnesota 55455|