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Tay Sachs disease

Tay-Sachs Disease
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Clinical Trials

This information is provided by ClinicalTrials.gov

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Displaying 1-7 of 7 results.
A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2
Status: Not yet recruiting
Last Changed: Jan 09, 2020
First Received: Jan 09, 2020
Disease(s): Tay-Sachs Disease Sandhoff Disease
Intervention(s): venglustat GZ402671, placebo
N-Acetyl-L-Leucine for GM2 Gangliosdisosis (Tay-Sachs and Sandhoff Disease)
Status: Recruiting
Last Changed: Oct 22, 2019
First Received: Nov 30, 2018
Disease(s): GM2 Gangliosidosis, Tay-Sachs Disease, Sandhoff Disease
Intervention(s): IB1001
Locations: University of California - Los Angeles, Los Angeles, California, United States
Mayo Clinic, Rochester, Minnesota, United States
NYU Langone School of Medicine, New York, New York, United States
University of Giessen, Gießen, Germany
Ludwig Maximilian University of Munich, München, Germany
... and 4 other locations.
Effects of Miglustat Therapy on Infantile Type of Sandhoff and Taysachs Diseases (EMTISTD)
Status: Recruiting
Last Changed: Jan 31, 2019
First Received: Jan 30, 2019
Disease(s): GM2 Gangliosidosis, Supportive Care
Intervention(s): Miglustat
Locations: Kashan University Of Medical Sciences, Kashan, Isfahan, Iran, Islamic Republic of
Mashhad University Of Medical Sciences, Mashhad, Khorasan, Iran, Islamic Republic of
Tehran University Of Medical Sciences, Tehran, Iran, Islamic Republic of
Synergistic Enteral Regimen for Treatment of the Gangliosidoses
Status: Recruiting
Last Changed: May 29, 2019
First Received: Jan 08, 2014
Disease(s): GM1 Gangliosidoses, GM2 Gangliosidoses, Tay-Sachs Disease, Sandhoff Disease
Intervention(s): miglustat, Ketogenic Diet
Locations: University of Minnesota, Minneapolis, Minnesota, United States
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Status: Recruiting
Last Changed: Dec 03, 2019
First Received: Oct 02, 2014
Disease(s): Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Neimann Pick Disease, Pelizaeus-Merzbacher Disease, Sandhoff Disease, Tay-Sachs Disease, Brain Diseases, Metabolic, Inborn, Alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses
Intervention(s): DUOC-01
Locations: Duke University Medical Center, Durham, North Carolina, United States
Longitudinal Study of Neurodegenerative Disorders
Status: Recruiting
Last Changed: Nov 05, 2019
First Received: Nov 06, 2017
Disease(s): MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
Intervention(s): Palliative Care, Hematopoetic Stem Cell Transplantation
Locations: The Program for the Study of Neurodevelopment in Rare Disorders, Children's Hospital of Pittsburgh of UPMC, Pittsburgh, Pennsylvania, United States
Nervous System Degeneration in Glycosphingolipid Storage Disorders
Status: Recruiting
Last Changed: Feb 20, 2020
First Received: Jan 28, 2002
Disease(s): Neurological Regression, Myoclonus, Cherry Red Spot, Brain Atrophy
Locations: National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda, Maryland, United States