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 |
Recruiting |
Study Name: Dose Finding Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis Condition: Alpha Mannosidosis Date: 2011-01-25 Interventions: Drug: Lamazym ERT, infusion weekly Other Name: rhLAM |
Completed |
Study Name: Safety Study of Recombinant Human Alpha-mannosidase for the Treatment of Patients With Alpha-mannosidosis Condition: Alpha Mannosidosis Date: 2010-12-29 Interventions: Drug: Lamazym Lamazym, ERT, infusion weekly Other Na |
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 |
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: The Natural History of Alpha-Mannosidosis Condition: Alpha Mannosidosis Date: 2007-07-09 |
Completed |
Study Name: Stem Cell Transplantation for Hurler Condition: Mucopolysaccharidosis I Mucopolysaccharidosis VI Date: 2005-09-12 Interventions: Procedure: Stem Cell Transplant |
Completed |
Study Name: Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation Condition: I Cell Disease Fucosidosis Globoid Cell Leukodystrophy Date: 2000-06-02 |