Adrenoleukodystrophy Clinical Trial
Official title:
Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Bone Marrow Transplantation
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.
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.
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