Tay-Sachs Disease



A Natural History of Late Onset Tay-Sachs Disease


Condition:   GM2 Gangliosidosis
Intervention:  
Sponsor:   Massachusetts General Hospital
Recruiting - verified May 2017


Biomarker for GM1/GM2 - Gangliosidoses


Conditions:   Gangliosidosis;   GM1-Gangliosidosis;   GM2-Gangliosidosis;   Hexosaminidase Activator Deficiency;   Tay-Sachs Disease, AB Variant;   Hexosaminidase A and B Deficiency;   Sandhoff Disease
Intervention:  
Sponsors:   University of Rostock;   Centogene AG Rostock
Recruiting - verified September 2016


UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells


Conditions:   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
Intervention:   Biological: DUOC-01
Sponsors:   Joanne Kurtzberg, MD;   The Marcus Foundation
Recruiting - verified April 2017


Synergistic Enteral Regimen for Treatment of the Gangliosidoses


Conditions:   GM1 Gangliosidoses;   GM2 Gangliosidoses;   Tay-Sachs Disease;   Sandhoff Disease
Interventions:   Drug: miglustat;   Other: Ketogenic Diet
Sponsors:   University of Minnesota - Clinical and Translational Science Institute;   Rare Diseases Clinical Research Network;   National Center for Advancing Translational Science (NCATS);   National Institute of Neurological Disorders and Stroke (NINDS);   National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);   Lysosomal Disease Network
Recruiting - verified December 2016


Efficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent.


Conditions:   Tay Sachs Disease;   Canavan Disease;   Familial Dysautonomia
Intervention:   Other: Online pre-test genetic education tool
Sponsor:   McGill University Health Center
Active, not recruiting - verified September 2016


Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders


Conditions:   Lysosomal Storage Disease;   Peroxisomal Disorder
Interventions:   Drug: Campath-1H;   Drug: Clofarabine;   Drug: Melphalan;   Radiation: Total Body Irradiation with Marrow Boosting;   Biological: Hematopoietic stem cell transplantation;   Drug: Cyclosporine A;   Drug: Mycophenolate mofetil
Sponsor:   Masonic Cancer Center, University of Minnesota
Completed - verified February 2017


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


Conditions:   Hurler Syndrome (MPS I);   Hurler-Scheie Syndrome With Early Neurologic Involvement and/or Sensitization to Enzyme Replacement Therapy (ERT);   Hunter Syndrome (MPS II);   Sanfilippo Syndrome (MPS III);   Krabbe Disease (Globoid Leukodystrophy);   Metachromatic Leukodystrophy (MLD);   Adrenoleukodystrophy (ALD and AMN);   Sandhoff Disease;   Tay Sachs Disease;   Pelizaeus Merzbacher (PMD);   Niemann-Pick Disease;   Alpha-mannosidosis
Intervention:   Biological: FCRx infusion
Sponsors:   University of Louisville;   Duke University
Active, not recruiting - verified November 2016


Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases


Conditions:   Lysosomal Storage Diseases;   Inborn Errors of Metabolism
Intervention:   Biological: ALD-601
Sponsors:   Joanne Kurtzberg, MD;   Aldagen
Withdrawn - verified April 2017


A Natural History Study of the Gangliosidoses


Conditions:   Tay-Sachs Disease;   Sandhoff Disease;   Late Onset Tay-Sachs Disease;   GM1 Gangliosidosis;   GM2 Gangliosidosis
Intervention:  
Sponsors:   University of Minnesota - Clinical and Translational Science Institute;   Rare Diseases Clinical Research Network;   National Center for Advancing Translational Science (NCATS);   National Institute of Neurological Disorders and Stroke (NINDS);   National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);   Lysosomal Disease Network
Recruiting - verified January 2017


Nervous System Degeneration in Glycosphingolipid Storage Disorders


Conditions:   Gangliosidoses;   Gaucher Disease
Intervention:  
Sponsor:   National Human Genome Research Institute (NHGRI)
Recruiting - verified April 14, 2017

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