Stem Cell Transplant for Inborn Errors of Metabolism

Status Completed

Clinical Trial 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.

Clinical Trial Description

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. ;

Study Design

Related Conditions & MeSH terms

Adrenoleukodystrophy
Aphasia, Primary Progressive
Batten Disease
Frontotemporal Dementia
Fucosidosis
Gangliosidoses
Gangliosidosis, GM1
Gaucher Disease
Gaucher's Disease
Globoid Cell Leukodystrophy
GM1 Gangliosidosis
Leukodystrophy, Globoid Cell
Leukodystrophy, Metachromatic
Metabolism, Inborn Errors
Metachromatic Leukodystrophy
Neuronal Ceroid-Lipofuscinoses
Niemann-Pick Disease
Niemann-Pick Disease, Type C
Niemann-Pick Diseases
Pick Disease of the Brain
Sandhoff Disease
Tay Sachs Disease
Tay-Sachs Disease
Wolman Disease

Clinical Trial Details

NCT number	NCT00176904
Study type	Interventional
Source	Masonic Cancer Center, University of Minnesota
Contact
Status	Completed
Phase	Phase 2/Phase 3
Start date	January 1995
Completion date	June 2010

View Details

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