Mucopolysaccharidosis I Clinical Trial
Official title:
Hematopoietic Stem Cell Transplantation for Hurler Syndrome, Maroteaux Lamy Syndrome (MPS VI), and Alpha Mannosidase Deficiency (Mannosidosis)
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.
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.
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