Status | Study |
Completed |
Study Name: Pharmacokinetics, Safety and Tolerability of Zavesca (Miglustat) in Patients With Infantile Onset Gangliosidosis: Single and Steady State Oral Doses Condition: GM2 Gangliosidoses Tay-Sachs Date: 2008-05-02 Interventions: Drug: Zavesca (Miglustat) |
Recruiting |
Study Name: A Natural History Study of the Gangliosidoses Condition: Tay-Sachs Disease Sandhoff Disease Late Onset Tay-Sachs Di Date: 2008-04-25 |
Completed |
Study Name: Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis Condition: Gangliosidoses GM2 Date: 2007-01-04 Interventions: Drug: miglustat Target dose of 320 mg/m^2/day (divided in 3 doses) will be based on the Body Surface Are |
Completed |
Study Name: HSCT for High Risk Inherited Inborn Errors Condition: Adrenoleukodystrophy Metachromatic Leukodystrophy Globoid Date: 2006-09-29 Interventions: Drug: Clofarabine days -7 thro |
Completed |
Study Name: Stem Cell Transplant for Inborn Errors of Metabolism Condition: Adrenoleukodystrophy Metachromatic Leukodystrophy Globoid Date: 2005-09-12 Interventions: Procedure: Stem Cell Transplant |
Recruiting |
Study Name: Nervous System Degeneration in Glycosphingolipid Storage Disorders Condition: Gangliosidoses Gaucher Disease Date: 2002-01-27 |
Completed |
Study Name: Diagnostic and Screening Study of Genetic Disorders Condition: Tay-Sachs Disease Porphyria, Erythropoietic Leukodystrophy Date: 2000-07-05 |