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Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Sandhoff Disease Clinical Trial

Official title:
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Clinical Trial Summary

The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.

Clinical Trial Description

The objective for the study is to establish chimerism following reduced intensity conditioning with no grade III/IV GVHD. The primary endpoint we will follow is production of the missing enzyme at ≥ 10% of the normal level at day 180 post-transplant in > 90% of patients. ;

Study Design

Related Conditions & MeSH terms

  • Adrenoleukodystrophy
  • Adrenoleukodystrophy (ALD and AMN)
  • Alpha-mannosidosis
  • Hunter Syndrome (MPS II)
  • Hurler Syndrome (MPS I)
  • Hurler-Scheie Syndrome
  • Krabbe Disease (Globoid Leukodystrophy)
  • Leukodystrophy, Globoid Cell
  • Leukodystrophy, Metachromatic
  • Metabolic Diseases
  • Metachromatic Leukodystrophy (MLD)
  • Mucopolysaccharidosis I
  • Mucopolysaccharidosis II
  • Mucopolysaccharidosis III
  • Niemann-Pick Disease
  • Niemann-Pick Disease, Type C
  • Niemann-Pick Diseases
  • Pelizaeus Merzbacher (PMD)
  • Pick Disease of the Brain
  • Sandhoff Disease
  • Sanfilippo Syndrome (MPS III)
  • Syndrome
  • Tay Sachs Disease
  • Tay-Sachs Disease
Clinical Trial Details
NCT number NCT01372228
Study type Interventional
Source Talaris Therapeutics Inc.
Contact
Status Terminated
Phase Phase 1/Phase 2
Start date April 2011
Completion date April 2016
View Details
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