Status | Study |
Recruiting |
Study Name: Krabbe Disease Global Patient Registry Condition: Krabbe Disease Date: 2016-12-06 |
Recruiting |
Study Name: A Natural History Study of Canavan Disease Condition: Canavan Disease Date: 2016-07-28 |
Recruiting |
Study Name: Aclarubicin for the Treatment of Retinal Vasculopathy With Cerebral Leukodystrophy Condition: Vasculopathy, Retinal, With Cerebral Leukodystrophy Cerebroretinal Vasculopathy, Hereditary Date: 2016-03-02 Interventions: Drug: aclarubicin Aclarubicin (3 mg/m²) will be administered intravenously through a central venous |
Recruiting |
Study Name: Natural History and Outcome Measures in Alexander Disease Condition: Alexander Disease Date: 2016-02-19 |
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 |
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 |
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 |