Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
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.
Timeline
- Start
- 2009-12
- Primary completion
- 2015-06
- Completion
- 2017-06
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Background | Alemtuzumab | Monoclonal antibody | 0.3 mg/kg | Subcutaneous |
| Background | Busulfan | Small molecule | 0.8 mg/kg | Intravenous |
| Background | Busulfan | Small molecule | 1.1 mg/kg | Intravenous |
| Background | Cyclophosphamide | Other / unclassified | 50 mg/kg | Intravenous |
| Background | Cyclosporine | Peptide | 2.5 mg/kg | Intravenous |
| Background | Mycophenolate Mofetil | Small molecule | 15 mg/kg | Intravenous |