Hereditary Central Nervous System Demyelinating Diseases - 25 Studies Found
Recruiting |
: Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy : Metachromatic Leukodystrophy : 2013-01-28 : Genetic: intracerebral administration of AAVrh.10cuARSA |
Active, not recruiting |
: Gene Therapy for Metachromatic Leukodystrophy : Lysosomal Storage Disease : 2012-03-16 : Genetic: Autologous CD34+ stem cells transduced with ARSA encoding lentiviral vector Autologous hematopo |
Completed |
: Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD) : Metachromatic Leukodystrophy (MLD) : 2011-12-14 : Biological: Recombinant human arylsulfatase A 6 patients treated with HGT-1110 EOW by IT injection |
Completed |
: Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD) :
: 2011-07-06 : Biological: HuCNS-SC transplant in the lead-in phase Long-term safety follow-up study |
Completed |
: Imaging Study of the White Matter Lesions in Children With Metachromatic Leucodystrophy : Late Infantile Metachromatic Leukodystrophy : 2011-03-09 : Other: High-field MRI (3 Teslas) |
Completed |
: Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation : Metachromatic Leukodystrophy : 2011-02-23 : Drug: rhARSA intravenous infusion 100U/kg every other week for 18 months |
Withdrawn |
: An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD) : Leukodystrophy, Globoid Cell : 2010-03-24 |
Completed |
: Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects : Pelizaeus-Merzbacher Disease : 2009-10-28 : Biological: HuCNS-SC cells intracerebral transplantation |
Completed |
: The Natural History of Infantile Globoid Cell Leukodystrophy : Infantile Globoid Cell Leukodystrophy : 2009-09-23 |
Active, not recruiting |
: Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease : Krabbe Disease : 2008-11-07 |