Status | Study |
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: 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 |