Allogeneic Hematopoietic Stem Cell Transplantation for Standard Risk Inherited Metabolic Disorders
21 Years
N/A
Both
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
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
serology
Type of Study:
Interventional
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:
No
Principal Investigator
Paul Orchard, MD
Investigator Role:
Principal Investigator
Investigator Affiliation:
Masonic Cancer Center, University of Minnesota
Authority:
United States: Institutional Review Board
Study ID:
2009LS088
NCT ID:
NCT01043640
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
Name | Location |
|---|---|
| Masonic Cancer Center, University of Minnesota | Minneapolis, Minnesota 55455 |
