18 studies found for:    TAY-SACHS DISEASE
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Rank Status Study
1 Recruiting Gene Therapy for Tay-Sachs Disease
Conditions: Tay Sachs Disease;   Sandhoff Disease;   Late Onset Tay Sachs Disease
Intervention:
2 Withdrawn A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis
Conditions: G(M2) Ganglioside;   Tay-Sachs Disease Ganglioside;   Sandhoff Disease Ganglioside
Intervention: Drug: Pyrimethamine
3 Completed Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)
Conditions: Gangliosidoses, GM2;   Sandhoff Disease;   Tay-Sachs Disease
Interventions: Drug: Pyrimethamine;   Drug: Leucovorin
4 Recruiting A Natural History Study of the Gangliosidoses
Conditions: Tay Sachs Disease;   Sandhoff Disease;   Late Onset Tay Sachs Disease
Intervention:
5 Recruiting Synergistic Enteral Regimen for Treatment of the Gangliosidoses
Conditions: GM1 Gangliosidoses;   GM2 Gangliosidoses;   Tay-Sachs Disease;   Sandhoff Disease
Interventions: Drug: miglustat;   Behavioral: Ketogenic Diet
6 Recruiting HSCT for High Risk Inherited Inborn Errors
Conditions: Adrenoleukodystrophy;   Metachromatic Leukodystrophy;   Globoid Cell Leukodystrophy;   Tay Sachs Disease;   Sandhoffs Disease;   Wolman Disease;   I-Cell Disease;   Sanfilippo Syndrome;   GM1 Gangliosidosis
Interventions: Drug: Clofarabine;   Procedure: Total body Irradiation;   Drug: Melphalan;   Biological: Hematopoietic Stem Cell Transplantation;   Drug: Alemtuzumab;   Drug: mycophenylate mofetil;   Device: Cyclosporine A;   Drug: Hydroxyurea
7 Active, not recruiting 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: Enriched Hematopoetic Stem Cell Transplantation/novel platform technology
8 Completed
Has Results
Stem Cell Transplant for Inborn Errors of Metabolism
Conditions: Adrenoleukodystrophy;   Metachromatic Leukodystrophy;   Globoid Cell Leukodystrophy;   Gaucher's Disease;   Fucosidosis;   Wolman Disease;   Niemann-Pick Disease;   Batten Disease;   GM1 Gangliosidosis;   Tay Sachs Disease;   Sandhoff Disease
Interventions: Procedure: Stem Cell Transplant;   Drug: Busulfan, Cyclophosphamide, Antithymocyte Globulin
9 Completed Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses
Conditions: GM2 Gangliosidoses;   Tay-Sachs;   Sandhoff Disease
Intervention: Drug: Zavesca (Miglustat)
10 Recruiting 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
11 Completed Diagnostic and Screening Study of Genetic Disorders
Conditions: Tay-Sachs Disease;   Porphyria, Erythropoietic;   Leukodystrophy, Globoid Cell;   Metabolism, Inborn Errors
Intervention:
12 Unknown  Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
Condition: Gangliosidoses GM2
Intervention: Drug: miglustat
13 Completed 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
14 Terminated ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Conditions: Inherited Metabolic Diseases;   Lysosomal Storage Disorders;   Peroxisomal Storage Diseases;   Inborn Errors of Metabolism;   Mucopolysaccharidosis
Intervention: Biological: ALD-101
15 Active, not recruiting Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases
Conditions: Lysosomal Storage Diseases;   Inborn Errors of Metabolism
Intervention: Biological: ALD-601
16 Completed Concurrent Single Gene and 24 Chromosome Aneuploidy Preimplantation Genetic Diagnosis (PGD)
Condition: Any Single Gene Disorder (Cystic Fibrosis, Tay-Sachs)
Intervention: Other: preimplantation diagnosis
17 Recruiting Nervous System Degeneration in Glycosphingolipid Storage Disorders
Conditions: Gangliosidoses;   Gaucher Disease
Intervention:
18 Completed Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells
Conditions: MDS;   Anemia, Aplastic;   Inborn Errors of Metabolism;   Congenital Marrow Failure;   Congenital Immunodeficiency Syndrome
Intervention: Biological: ALDHbr Umbilical Cord Blood Cells

Indicates status has not been verified in more than two years