Now Available: Final Rule for FDAAA 801 and NIH Policy on Clinical Trial Reporting
54 studies found for:    Leukodystrophy
Show Display Options
RSS Create an RSS feed from your search for:
Leukodystrophy
Need help? See RSS Feeds
Choose a feed type:
Show studies first received on any dateShow studies that were first received in the last 14 days
Show studies last updated date on any dateShow studies that were added or modified in the last 14 days
Rank Status Study
1 Completed Imaging Study of the White Matter Lesions in Children With Metachromatic Leucodystrophy
Condition: Late Infantile Metachromatic Leukodystrophy
Intervention: Other: High-field MRI (3 Teslas)
2 Recruiting Biomarker for Metachromatic Leukodystrophy Disease
Conditions: Leukodystrophy, Metachromatic;   Hereditary Central Nervous System Demyelinating Diseases;   Brain Diseases, Metabolic, Inborn;   Sphingolipidoses;   Lysosomal Storage Diseases, Nervous System
Intervention:
3 Active, not recruiting Gene Therapy for Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention: Genetic: Autologous CD34+ stem cells transduced with ARSA encoding lentiviral vector
4 Recruiting Natural History of the Leukodystrophies
Condition: Leukodystrophies
Intervention:
5 Recruiting Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention: Genetic: intracerebral administration of AAVrh.10cuARSA
6 Active, not recruiting Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)
Condition: Metachromatic Leukodystrophy (MLD)
Intervention: Biological: Recombinant human arylsulfatase A
7 Withdrawn An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD)
Condition: Leukodystrophy, Globoid Cell
Intervention:
8 Completed The Natural History of Infantile Globoid Cell Leukodystrophy
Condition: Infantile Globoid Cell Leukodystrophy
Intervention:
9 Not yet recruiting Aclarubicin for the Treatment of Retinal Vasculopathy With Cerebral Leukodystrophy
Conditions: Vasculopathy, Retinal, With Cerebral Leukodystrophy;   Cerebroretinal Vasculopathy, Hereditary
Intervention: Drug: aclarubicin
10 Completed Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention: Drug: Warfarin
11 Recruiting New Diagnostic and Therapeutic Approaches in Leukodystrophy
Conditions: White Matter Diseases;   Leukodystrophy
Interventions: Other: Whole Genome Sequencing (WGS);   Other: Standard Clinical Care and Diagnostic Approaches
12 Active, not recruiting The Natural History of Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention:
13 Enrolling by invitation Open-Label Extension Study Evaluating Safety and Efficacy of HGT-1110 in Patients With Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy (MLD)
Intervention: Biological: Recombinant human arylsulfatase A
14 Recruiting The Nosology and Etiology of Leukodystrophies of Unknown Causes
Conditions: Leukodystrophy;   Leukoencephalopathy
Intervention:
15 Recruiting Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy
Conditions: Metachromatic Leukodystrophy;   Adrenoleukodystrophy
Intervention: Genetic: transduced CD34+ hematopoietic stem cell
16 Completed
Has Results
Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Condition: Late Infantile Metachromatic Leukodystrophy
Intervention: Biological: Recombinant human Arylsulfatase A (rhASA)
17 Completed
Has Results
Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Condition: Metachromatic Leukodystrophy (MLD)
Intervention: Drug: rhASA
18 Completed Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation
Condition: Metachromatic Leukodystrophy
Intervention: Drug: rhARSA
19 Completed The Classification and Cause of Leukodystrophies of Unknown Cause
Condition: Lysosomal Storage Disease
Intervention:
20 Terminated Natural History Study of Children With Metachromatic Leukodystrophy
Conditions: Lipid Metabolism Disorders;   Metachromatic Leukodystrophy (MLD);   Nervous System Diseases;   Brain Diseases;   Central Nervous System Diseases;   Demyelinating Diseases;   Metabolism, Inborn Errors;   Genetic Diseases, Inborn;   Sphingolipidoses;   Hereditary Central Nervous System Demyelinating Diseases;   Metabolic Inborn Brain Diseases;   Lysosomal Storage Diseases;   Metabolic Diseases;   Sulfatidosis
Intervention:

   Previous Page Studies Shown (1-20) Next Page (21-40) Show next page of results    Last Page
Study has passed its completion date and status has not been verified in more than two years.