19 studies found for:    Tay-Sachs Disease
Show Display Options
RSS Create an RSS feed from your search for:
Tay-Sachs Disease
Need help? See RSS Feeds
Choose a feed type:
Show studies first received on any dateShow studies that were first received in the last 14 days
Show studies last updated date on any dateShow studies that were added or modified in the last 14 days
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2 Withdrawn A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis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 Recruiting A Natural History Study of Hexosaminidase Deficiency
Conditions: Tay Sachs Disease;   Sandhoff Disease;   Late Onset Tay Sachs Disease
Intervention:
4 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
5 Unknown  Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
Condition: Gangliosidoses GM2
Intervention: Drug: miglustat
6 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
7 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)
8 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: Hydroxyuria
9 Completed Diagnostic and Screening Study of Genetic Disorders
Conditions: Tay-Sachs Disease;   Porphyria, Erythropoietic;   Leukodystrophy, Globoid Cell;   Metabolism, Inborn Errors
Intervention:
10 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
11 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
12 Not yet recruiting Evaluating the Efficacy of a Web-based Educational Module Prior to Carrier Genetic Screening in Individuals of Ashkenazi Jewish Descent.
Conditions: Tay Sachs Disease;   Canavan Disease;   Familial Dysautonomia
Intervention: Other: Online pre-test genetic counselling tool
13 Suspended ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases
Conditions: Inherited Metabolic Diseases;   Lysosomal Storage Disease;   Peroxisomal Storage Diseases;   Inborn Errors of Metabolism;   Mucopolysaccharidosis
Intervention: Biological: ALD-101
14 Recruiting 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
15 Recruiting Nervous System Degeneration in Glycosphingolipid Storage Disorders
Conditions: Gangliosidoses;   Gaucher Disease
Intervention:
16 Recruiting Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases
Conditions: Lysosomal Storage Diseases;   Inborn Errors of Metabolism
Intervention: Biological: ALD-601
17 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
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
19 Completed Clinical Use of Parental Support To Detect Single Gene Mutations
Condition: Single Gene Disorders
Intervention: Other: Preimplantation Genetic Diagnosis

Indicates status has not been verified in more than two years