Status | Study |
Recruiting |
Study Name: Biomarker for GM1/GM2 - Gangliosidoses Condition: Gangliosidosis GM1-Gangliosidosis Date: 2014-10-23 |
Recruiting |
Study Name: Synergistic Enteral Regimen for Treatment of the Gangliosidoses Condition: GM1 Gangliosidoses GM2 Gangliosidoses Date: 2013-12-17 Interventions: Drug: miglustat The Syner-G th |
Completed |
Study Name: Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders Condition: Lysosomal Storage Disease Peroxisomal Disorder Date: 2012-06-20 Interventions: Drug: Campath-1H A daily dose |
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 |
Terminated |
Study Name: ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases Condition: Inherited Metabolic Diseases Lysosomal Storage Disorders P Date: 2008-04-03 Interventions: Biological: ALD-101 A subpopulation of cord blood cells composed of cells that express a high level of t |
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 |