Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

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Brief Title

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

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 Description

      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.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Number of Patients With Donor Derived Engraftment

Secondary Outcome

 Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD)

Condition

Mucopolysaccharidosis

Intervention

Campath-1H

Study Arms / Comparison Groups

 Transplant Patients
Description:  Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

46

Start Date

December 2009

Completion Date

June 2017

Primary Completion Date

June 2015

Eligibility Criteria

        Inclusion Criteria:

          -  Must have diagnosis of one of the following: mucopolysaccharidosis disorder,
             glycoprotein metabolic disorder, sphingolipidoses or inherited leukodystrophy,
             peroxisomal disorder or other inherited diseases of metabolism

          -  Must have an acceptable graft source as defined by University of Minnesota criteria

          -  Adequate organ function

        Exclusion Criteria:

          -  Pregnant - menstruating females must have a negative serum pregnancy test within 14
             days of treatment start

          -  Evidence of human immunodeficiency virus (HIV) infection or known HIV positive
             serology
      

Gender

All

Ages

N/A - 21 Years

Accepts Healthy Volunteers

No

Contacts

Paul Orchard, MD, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT01043640

Organization ID

2009LS088

Secondary IDs

MT2009-19

Responsible Party

Sponsor

Study Sponsor

Masonic Cancer Center, University of Minnesota


Study Sponsor

Paul Orchard, MD, Principal Investigator, Masonic Cancer Center, University of Minnesota


Verification Date

September 2017