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Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism

Niemann-Pick Disease, Type C Clinical Trial

Official title:
Treatment of Lysosomal and Peroxisomal Inborn Errors of Metabolism by Hematopoietic Cell Transplantation

Clinical Trial Summary

The primary objective of this clinical trial is to evaluate the ability to achieve and sustain donor engraftment in patients with lysosomal and peroxisomal inborn errors of metabolism undergoing hematopoietic stem cell transplantation (HCT).

Clinical Trial Description

This has been an ongoing area of interest by our group at the Univ. of Minnesota, but this is a new protocol to take the place of several older protocols. While survival has been very good on the prior protocols over the past decade, incomplete engraftment has remained somewhat problematic. Therefore, we have modified the preparative regimen somewhat to increase engraftment by replacing anti-thymocyte globulin (ATG) with Campath-1H, a drug that is more immune suppressive. In addition, we have modified the supportive care regimen. Based on this, we will monitor levels of an anti-oxidant therapy (N-acetylcysteine) and biomarkers of inflammation and oxidative stress for the families that consent to these research studies. ;

Study Design

Related Conditions & MeSH terms

  • Alpha Mannosidosis
  • alpha-Mannosidosis
  • Aphasia, Primary Progressive
  • Aspartylglucosaminuria
  • Frontotemporal Dementia
  • Fucosidosis
  • Hurler's Syndrome
  • Krabbe Disease
  • Leukodystrophy, Globoid Cell
  • Maroteaux-Lamy Syndrome
  • Metabolism, Inborn Errors
  • Mucopolysaccharidosis I
  • Mucopolysaccharidosis VI
  • Mucopolysaccharidosis VII
  • Niemann-Pick Disease Type B
  • Niemann-Pick Disease, Type C
  • Niemann-Pick Diseases
  • Pick Disease of the Brain
  • Sly Syndrome
  • Sphingolipidoses
  • Syndrome
  • Wolman Disease
  • Wolman's Disease
Clinical Trial Details
NCT number NCT00668564
Study type Interventional
Source Masonic Cancer Center, University of Minnesota
Contact
Status Terminated
Phase Phase 2
Start date March 2008
Completion date February 2010
View Details
See also
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