MT2013-31:Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis following Conditioning with Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
The purpose of this study is to determine the safety and effectiveness of allogeneic hematopoietic cell transplant in persons with an inherited metabolic disorder or osteopetrosis and if it is effective in reducing or slowing the symptoms associated with the genetic error. The study uses a chemotherapy conditioning regimen that prepares the body to accept the donor hematopoietic cells.
• up to 55 years old
• diagnosis of an Inherited Metabolic Disorders (IMD)
• see link to clinicaltrials.gov for complete Inclusion and Exclusion criteria
• uncontrolled bacterial, fungal or viral infections including HIV
• women who are pregnant
Drug: IMD Preparative Regimen, Drug: Osteopetrosis Haploidentical Only Preparative Regimen, Drug: Osteopetrosis Only Preparative Regimen, Biological: Stem Cell Transplantation, Drug: cALD HR-D (High-Risk, Regimen C), Drug: cALD HR-D (High-Risk, Regimen D), Drug: cALD SR-A (Standard-Risk, Regimen A), Drug: cALD SR-B (Standard-Risk, Regimen B)
Rare Diseases
Clinics and Surgery Center (CSC), Allogeneic Hematopoietic Cell Transplantation, IMD, Inherited metabolic disorders, osteopetrosis