Status | Study |
Active, not recruiting |
Study Name: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders Condition: Hurler Syndrome (MPS I) Hurler-Scheie Syndrome With Early Neurologic Involvement and/or Sens Date: 2011-06-10 Interventions: Biological: FCRx infusion Enriched hematopoetic stem cell infusion |
Withdrawn |
Study Name: An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD) Condition: Leukodystrophy, Globoid Cell Date: 2010-03-24 |
Active, not recruiting |
Study Name: Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Condition: Mucopolysaccharidosis Hurler Syndrome Hunter Syndrome Date: 2010-01-05 Interventions: Drug: Campath-1H Administered |
Completed |
Study Name: The Natural History of Infantile Globoid Cell Leukodystrophy Condition: Infantile Globoid Cell Leukodystrophy Date: 2009-09-23 |
Active, not recruiting |
Study Name: Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease Condition: Krabbe Disease Date: 2008-11-07 |
Terminated |
Study Name: Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism Condition: Hurler's Syndrome Maroteaux-Lamy Syndrome Sly Syndrome Date: 2008-04-25 Interventions: Procedure: Stem Cell Transplantation |
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 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 Date: 2005-09-12 Interventions: Procedure: Stem Cell Transplant |
Completed |
Study Name: Diagnostic and Screening Study of Genetic Disorders Condition: Tay-Sachs Disease Porphyria, Erythropoietic Date: 2000-07-05 |