Status | Study |
Recruiting |
Study Name: The Myelin Disorders Biorepository Project Condition: Leukodystrophy White Matter Disease Date: 2017-02-01 |
Recruiting |
Study Name: Krabbe Disease Global Patient Registry Condition: Krabbe Disease Date: 2016-12-06 |
Recruiting |
Study Name: Natural History of the Leukodystrophies Condition: Leukodystrophies Date: 2016-04-07 |
Recruiting |
Study Name: Aclarubicin for the Treatment of Retinal Vasculopathy With Cerebral Leukodystrophy Condition: Vasculopathy, Retinal, With Cerebral Leukodystrophy Cerebroreti Date: 2016-03-02 Interventions: Drug: aclarubicin Aclarubicin (3 mg/m²) will be administered intravenously through a central venous |
Recruiting |
Study Name: LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies Condition: Leukodystrophy White Matter Disease Date: 2016-02-16 Interventions: Other: Whole Genome Sequencing (WGS) |
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: 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 |