Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Status:
Completed
Trial end date:
2017-06-01
Target enrollment:
Participant gender:
Summary
Rationale: Chemotherapy administration before a donor stem cell transplant is necessary to
stop the patient's immune system from rejecting the donor's stem cells. When healthy stem
cells from a donor are infused into the patient, the donor white blood cells can provide the
missing enzyme that causes the metabolic disease. Sometimes the transplanted cells from a
donor can make an immune response against the body's normal cells. Giving a monoclonal
antibody, alemtuzumab, before transplant and cyclosporine and mycophenolate mofetil before
and after transplant may stop this from happening. This may be an effective treatment for
inherited metabolic disorders.
Purpose: The design of this study is to achieve donor cell engraftment in patients with
standard-risk inherited metabolic diseases with limited peri-transplant morbidity and
mortality. This will be achieved through the administration of the chemotherapy regimen
described. The intention is to follow transplanted patient for years after transplant
monitoring them for complications of their disease and assisting families with a multifaceted
interdisciplinary approach.