Clinical Trial: Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders

Brief 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.


Detailed Summary:

Primary Objective:

  • To estimate the proportion of patients with donor derived engraftment at day 100 post transplant as defined by 80% or greater donor cells in the CD3 (T cell) fraction

Secondary Objectives:

  • To determine the incidence and severity of graft-versus-host disease (GVHD) by day 100
  • To determine the incidence of peri-transplant mortality (death by day 100)
  • To monitor donor cell chimerism at various time points following allogeneic transplantation with this transplant regimen as determined at day 28, 42, 100, 6 months and yearly for 5 years.

Sponsor: Masonic Cancer Center, University of Minnesota

Current Primary Outcome: Number of Patients With Donor Derived Engraftment [ Time Frame: Day 100 Post Transplant ]

Donor derived engraftment is defined as 80 percent or greater donor cells in the recipient's bone marrow and blood cells.


Original Primary Outcome: Number of Patients With Donor Derived Engraftment [ Time Frame: Day 100 Post Transplant ]

Current Secondary Outcome:

  • Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD) [ Time Frame: Day 100 Post Transplant ]
  • Number of Patients With Grade 1 Graft-Versus-Host Disease (GVHD) [ Time Frame: Day 100 Post Transplant ]
  • Number of Patients With Grade 2 Graft-Versus-Host Disease (GVHD) [ Time Frame: Day 100 Post Transplant ]
  • Number of Patients With Grade 3 Graft-Versus-Host Disease (GVHD) [ Time Frame: Day 100 Post Transplant ]
  • Number of Patients With Grade 4 Graft-Versus-Host Disease (GVHD) [ Time Frame: Day 100 Post Transplant ]
  • Number of Patients Who Died Peri-Transplant [ Time Frame: By Day 100 Post Transplant ]
    Peri-transplant is defined as within 100 days of transplant.
  • Donor Cell Chimerism Following Transplant [ Time Frame: Day 28 ]
    Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin.
  • Donor Cell Chimerism Following Transplant [ Time Frame: Day 42 ]
    Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin.
  • Donor Cell Chimerism Following Transplant [ Time Frame: Day 100 ]
    Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin.
  • Donor Cell Chimerism Following Transplant [ Time Frame: 6 months ]
    Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin.
  • Donor Cell Chimerism Following Transplant [ Time Frame: One year ]
    Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin.


Original Secondary Outcome:

  • Number of Patients with Graft-Versus-Host Disease (GVHD) by Severity [ Time Frame: Day 100 Post Transplant ]
  • Number of Patients Died Peri-Transplant [ Time Frame: By Day 100 Post Transplant ]
  • Donor Cell Chimerism Following Transplant [ Time Frame: Day 28, Day 42, Day 100, Month 6, Yearly Post Transplant ]


Information By: Masonic Cancer Center, University of Minnesota

Dates:
Date Received: January 5, 2010
Date Started: December 2009
Date Completion: June 2017
Last Updated: March 31, 2017
Last Verified: March 2017