Inherited Metabolic Storage Diseases Clinical Trial
Official title:
Anti-CD45 (YTH-24 & YTH 54) and ANTI-CD52 (CAMPATH-1H) Monoclonal Antibody Conditioning Regimen for Allogeneic Stem Cell Transplantation of Patients With Inherited Metabolic Storage Diseases
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.'
Fludarabine will be given as a daily IV (intravenous, by vein) infusion for a total of 5 days. CAMPATH-1H will be given as a daily 4-hour IV (intravenous, by vein) infusion for three days. Anti-CD45 will be given as a daily 6-hour IV infusion over the next 4 days. Then patients will have a one-day rest period before receiving the stem cell transplant. To help prevent the body from rejecting the transplant, patients will also receive the drug FK506, starting two days before the transplant and continuing for three months. If there is no GVHD, the amount of FK506 patients are taking will be reduced by 20% every 2 weeks until this medication is stopped. ;
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment