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Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

University of Minnesota (UMN) logo

University of Minnesota (UMN)

Status and phase

Completed
Phase 2

Conditions

Hunter Syndrome
Sly Syndrome
Aspartylglucosaminuria
Maroteaux-Lamy Syndrome
Metachromatic Leukodystrophy (MLD)
Alpha Mannosidosis
Sphingolipidoses
Mucopolysaccharidosis
Peroxisomal Disorders
Adrenoleukodystrophy (ALD)
Fucosidosis
Hurler Syndrome
Krabbe Disease

Treatments

Drug: Cyclophosphamide
Drug: Busulfan
Procedure: Allogeneic stem cell transplantation
Drug: Campath-1H
Drug: Mycophenolate Mofetil
Drug: Cyclosporine A

Study type

Interventional

Funder types

Other

Identifiers

NCT01043640
2009LS088
MT2009-19 (Other Identifier)

Details and patient eligibility

About

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.

Full 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.

Enrollment

46 patients

Sex

All

Ages

Under 21 years old

Volunteers

No Healthy Volunteers

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

Trial design

Primary purpose

Treatment

Allocation

N/A

Interventional model

Single Group Assignment

Masking

None (Open label)

46 participants in 1 patient group

Transplant Patients
Experimental group
Description:
Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.
Treatment:
Drug: Cyclosporine A
Drug: Busulfan
Drug: Campath-1H
Procedure: Allogeneic stem cell transplantation
Drug: Cyclophosphamide
Drug: Mycophenolate Mofetil

Trial contacts and locations

1

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Data sourced from clinicaltrials.gov

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