Status | Study |
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 |
Recruiting |
Study Name: Biomarker for Metachromatic Leukodystrophy Disease Condition: Leukodystrophy, Metachromatic Hereditary Central Nervous System Date: 2012-02-21 |
Completed |
Study Name: Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD) Condition: Metachromatic Leukodystrophy (MLD) Date: 2011-12-14 Interventions: Biological: Recombinant human arylsulfatase A 6 patients treated with HGT-1110 EOW by IT injection |
Completed |
Study Name: Imaging Study of the White Matter Lesions in Children With Metachromatic Leucodystrophy Condition: Late Infantile Metachromatic Leukodystrophy Date: 2011-03-09 Interventions: Other: High-field MRI (3 Teslas) |
Completed |
Study Name: Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation Condition: Metachromatic Leukodystrophy Date: 2011-02-23 Interventions: Drug: rhARSA intravenous infusion 100U/kg every other week for 18 months |
Withdrawn |
Study Name: An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD) Condition: Leukodystrophy, Globoid Cell Date: 2010-03-24 |
Completed |
Study Name: The Natural History of Infantile Globoid Cell Leukodystrophy Condition: Infantile Globoid Cell Leukodystrophy Date: 2009-09-23 |
Recruiting |
Study Name: The Nosology and Etiology of Leukodystrophies of Unknown Causes Condition: Leukodystrophy Leukoencephalopathy Date: 2009-04-25 |
Completed |
Study Name: Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy Condition: Metachromatic Leukodystrophy Date: 2008-05-21 Interventions: Drug: Warfarin Oral administration (QD), variable dosage: patients will undergo blood test for PT/INR at |