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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Treatment of High Risk, Inherited Lysosomal and Peroxisomal Disorders by Reduced-Intensity Hematopoietic Cell Transplantation and Low-Dose Total Body Irradiation With Marr

Brief 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.

Detailed Summary: 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.
Sponsor: Masonic Cancer Center, University of Minnesota

Current Primary Outcome: Donor (Allogeneic) Hematopoietic Engraftment [ Time Frame: Day 100 Following Hematopoietic Cell Transplant (HCT) ]

Number of patients who achieve hematopoietic engraftment - assessment of nucleated peripheral blood cells for donor (allogeneic) chimerism following this reduced-intensity HCT.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Transplant-Related Mortality [ Time Frame: Day 100 following HCT ]
    Incidence of death due to complications of HCT following this reduced-intensity conditioning regimen.
  • Neurologic Outcomes [ Time Frame: Changes from Baseline, Days 30, 60, 100, Year 1, Year 2, Year 3 Following HCT ]
    Depending upon underlying primary disease, a combination of evaluative tools (e.g. brain magnetic resonance imaging (MRI), clinical neurologic exam, neuropsychologic testing, electromyography) will be applied for assessment of neurologic function and how it may be affected by this reduced-intensity HCT regimen.


Original Secondary Outcome: Same as current

Information By: Masonic Cancer Center, University of Minnesota

Dates:
Date Received: June 20, 2012
Date Started: July 11, 2012
Date Completion:
Last Updated: February 17, 2017
Last Verified: February 2017