Monoclonal Antibody Conditioning for Allogeneic Stem Cell Transplantation of Patients With Inherited Metabolic Storage Diseases

• Status: Terminated
• Trial end date: 2003-06-01

Summary

This study treats patients with an inherited disease that prevents the body from making a specific protein or enzyme needed for the body's metabolism. Lack of this enzyme causes accumulation of harmful or toxic substances in the body, which leads to deterioration and failure of organs such as the brain or the heart. This disease can be fatal.

Some patients with inherited metabolic storage disease may benefit from an allogeneic stem cell transplant ('allogeneic' means that the stem cells come from another person). Stem cells are created in the bone marrow. They mature into different types of blood cells that are needed including red blood cells, white blood cells, and platelets. Stem cells, when transplanted, can make a new blood system. Donor stem cells can make the protein or enzyme patients with this disease cells cannot. The donor cells may prevent further accumulation of toxic substances. It is hoped that the donor cells can prevent or stop the disease from progressing.

This research study uses a new pre-treatment combination of two drugs, Anti-CD45 and CAMPATH-1H. Anti-CD45 and CAMPATH-1H are antibodies against certain types of blood cells. CAMPATH-1H is particularly important because it stays active in the body for a long time after infusion, which means it may work longer at preventing GVHD symptoms. In addition to antibodies, patients will receive Fludarabine, which is a chemotherapy drug. Fludarabine kills bone marrow cells and is given to reduce the bone marrow cells so that donor stem cells may 'take.'

• Phase: Phase 1/Phase 2

Details

• Lead Sponsor: Baylor College of Medicine
• Collaborators: Texas Children's Hospital; The Methodist Hospital Research Institute; The Methodist Hospital System
• Treatments: Alemtuzumab; Antibodies; Antibodies, Monoclonal; Fludarabine; Fludarabine phosphate; Tacrolimus