Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders

Lysosomal Storage Disease Clinical Trial

Official title: Treatment of High Risk, Inherited Lysosomal and Peroxisomal Disorders by Reduced-Intensity Hematopoietic Cell Transplantation and Low-Dose Total Body Irradiation With Marrow Boosting by Volumetric-Modulated Arc Therapy (VMAT)

Status Completed

Clinical Trial 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.

Clinical Trial Description

The conditioning regimen consists of alemtuzumab (Campath-1H), clofarabine, melphalan, and VMAT-delivered low-dose TBI with boosted marrow irradiation. Additional graft-versus-host disease prophylaxis consists of mycophenolate mofetil and cyclosporine. ;

Related Conditions & MeSH terms

• Disease
• Lysosomal Storage Disease
• Lysosomal Storage Diseases
• Peroxisomal Disorder
• Peroxisomal Disorders

• NCT number: NCT01626092
• Study type: Interventional
• Source: Masonic Cancer Center, University of Minnesota
• Status: Completed
• Phase: N/A
• Start date: July 11, 2012
• Completion date: November 2013