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Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders

Phase 2
21 Years
Open (Enrolling)
Mucopolysaccharidosis, Hurler Syndrome, Hunter Syndrome, Maroteaux-Lamy Syndrome, Sly Syndrome, Alpha Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Adrenoleukodystrophy (ALD), Globoid Cell Leukodystrophy (GLD), Krabbe Disease, Metachromatic Leukodystrophy (MLD), Sphingolipidoses, Peroxisomal Disorders

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Trial Information

Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders

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.

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

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Number of Patients with Donor Derived Engraftment

Outcome Time Frame:

Day 100 Post Transplant

Safety Issue:


Principal Investigator

Paul Orchard, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Masonic Cancer Center, University of Minnesota


United States: Institutional Review Board

Study ID:




Start Date:

December 2009

Completion Date:

November 2016

Related Keywords:

  • Mucopolysaccharidosis
  • Hurler Syndrome
  • Hunter Syndrome
  • Maroteaux-Lamy Syndrome
  • Sly Syndrome
  • Alpha Mannosidosis
  • Fucosidosis
  • Aspartylglucosaminuria
  • Adrenoleukodystrophy (ALD)
  • Globoid Cell Leukodystrophy (GLD)
  • Krabbe Disease
  • Metachromatic Leukodystrophy (MLD)
  • Sphingolipidoses
  • Peroxisomal Disorders
  • inherited metabolic disorders
  • Fucosidosis
  • Leukodystrophy, Globoid Cell
  • Leukodystrophy, Metachromatic
  • Alpha-Mannosidosis
  • Mannosidase Deficiency Diseases
  • Metabolic Diseases
  • Mucopolysaccharidoses
  • Mucopolysaccharidosis II
  • Mucopolysaccharidosis VI
  • Sphingolipidoses
  • Mucopolysaccharidosis VII
  • Mucopolysaccharidosis I
  • Adrenoleukodystrophy
  • Aspartylglucosaminuria
  • Peroxisomal Disorders



Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota  55455