Clinical Trials Logo
  1. Home
  2. Other
  3. NCT01372228

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
See also
  Status Clinical Trial Phase
Recruiting NCT02254863 - UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells Phase 1
Recruiting NCT04624789 - Registry Gangliosidoses
Completed NCT03759665 - N-Acetyl-L-Leucine for GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) Phase 2
Active, not recruiting NCT04221451 - A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2 Phase 3
Completed NCT04470713 - Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
Completed NCT00672022 - Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses Phase 3
Completed NCT00176904 - Stem Cell Transplant for Inborn Errors of Metabolism Phase 2/Phase 3
Active, not recruiting NCT04669535 - A Dose-escalation and Safety & Efficacy Study of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease Phase 1
Recruiting NCT03333200 - Longitudinal Study of Neurodegenerative Disorders
Active, not recruiting NCT05109793 - GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)
Terminated NCT02030015 - Synergistic Enteral Regimen for Treatment of the Gangliosidoses Phase 4
Recruiting NCT00668187 - A Natural History Study of the Gangliosidoses
Completed NCT01869270 - Gene Therapy for Tay-Sachs Disease N/A
Completed NCT01102686 - Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease) Phase 1/Phase 2