| Status | Study |
| Recruiting |
Study Name: Krabbe Disease Global Patient Registry Condition: Krabbe Disease Date: 2016-12-06 |
| Recruiting |
Study Name: UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells Condition: Adrenoleukodystrophy Batten Disease Mucopolysaccharidosis Date: 2014-09-23 Interventions: Biological: DUOC-01 Intrathecal administration of DUOC-01 |
| Recruiting |
Study Name: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Condition: Mucopolysaccharidosis Disorders Hurler Syndrome Hunter Syn Date: 2014-06-20 Interventions: Biological: Stem Cell Transplantation |
| Recruiting |
Study Name: Genetic Characterization of Movement Disorders and Dementias Condition: Ataxia Dystonia Parkinson's Disease Date: 2013-12-12 |
| Recruiting |
Study Name: Reduced Intensity Conditioning in Patients Aged ≤35 With Non-Malignant Disorders Undergoing UCBT, BMT, or PBSCT Condition: Primary Immunodeficiency Syndromes Congenital Bone Marrow Failure Syndromes Date: 2013-10-10 Interventions: Drug: Hydroxyurea Hydroxyurea |
| Recruiting |
Study Name: Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children Condition: Mucopolysaccharidosis Type I (MPS I) Mucopolysaccharidosis Type II (MPS II) Date: 2013-08-06 |
| 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: Human Placental-Derived Stem Cell Transplantation Condition: Mucopolysaccharidosis I Mucopolysaccharidosis VI Adrenoleu Date: 2012-04-25 Interventions: Drug: Human Placental Derived Stem Cell Infusions of thawed HPDSC to be given following UCB infusion. |
| Recruiting |
Study Name: Biomarker for Krabbe Disease Condition: Lysosomal Storage Diseases Krabbe Disease Date: 2011-08-29 |
| 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 |