Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
Status:
Completed
Trial end date:
2013-11-01
Target enrollment:
Participant gender:
Summary
This study is designed to test the ability to achieve donor hematopoietic engraftment while
maintaining low rates of transplant-related mortality (TRM) in patients with high-risk
lysosomal and peroxisomal disorders using a novel conditioning regimen for hematopoietic cell
transplantation (HCT). After a reduced-intensity conditioning regimen using
volumetric-modulated arc therapy (VMAT)-delivered low-dose total body irradiation (TBI) with
highly conformal marrow boosting, patients will be transplanted using either a related or
unrelated allograft. The cell source may be marrow, peripheral blood or cord blood based on
donor availability.