| Status | Study |
| Recruiting |
Study Name: Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy Condition: Metachromatic Leukodystrophy Adrenoleukodystrophy Date: 2015-08-12 Interventions: Genetic: transduced CD34+ hematopoietic stem cell Autologous hematopoietic stem cells (HSCs) collected f |
| 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 |
| No longer available |
Study Name: Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use) Condition: Metachromatic Leukodystrophy Date: 2014-03-07 Interventions: Biological: Enriched Hematopoetic Stem Cell Infusion Enriched Hematopoetic Stem Cell Infusion |
| No longer available |
Study Name: Single Patient Expanded Access Protocol: Metabolic Boost Condition: Metachromatic Leukodystrophy Date: 2013-12-19 Interventions: Biological: Enriched Hematopoetic Stem Cell Infusion Enriched Hematopoetic Stem Cell Infusion |
| Terminated |
Study Name: Natural History Study of Children With Metachromatic Leukodystrophy Condition: Lipid Metabolism Disorders Metachromatic Leukodystrophy (MLD) Date: 2013-10-11 |
| Enrolling by invitation |
Study Name: Open-Label Extension Study Evaluating Safety and Efficacy of HGT-1110 in Patients With Metachromatic Leukodystrophy Condition: Metachromatic Leukodystrophy (MLD) Date: 2013-06-20 Interventions: Biological: Recombinant human arylsulfatase A |
| Recruiting |
Study Name: Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy Condition: Metachromatic Leukodystrophy Date: 2013-01-28 Interventions: Genetic: intracerebral administration of AAVrh.10cuARSA |
| 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. |
| Active, not recruiting |
Study Name: Gene Therapy for Metachromatic Leukodystrophy Condition: Lysosomal Storage Disease Date: 2012-03-16 Interventions: Genetic: Autologous CD34+ stem cells transduced with ARSA encoding lentiviral vector Autologous hematopo |