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55 studies found for:    Leukodystrophy
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Leukodystrophy
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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 Recruiting Natural History of the Leukodystrophies
Condition: Leukodystrophies
Intervention:
4 Recruiting Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention: Genetic: intracerebral administration of AAVrh.10cuARSA
5 Completed Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)
Condition: Metachromatic Leukodystrophy (MLD)
Intervention: Biological: Recombinant human arylsulfatase A
6 Withdrawn An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD)
Condition: Leukodystrophy, Globoid Cell
Intervention:
7 Completed The Natural History of Infantile Globoid Cell Leukodystrophy
Condition: Infantile Globoid Cell Leukodystrophy
Intervention:
8 Recruiting Aclarubicin for the Treatment of Retinal Vasculopathy With Cerebral Leukodystrophy
Conditions: Vasculopathy, Retinal, With Cerebral Leukodystrophy;   Cerebroretinal Vasculopathy, Hereditary
Intervention: Drug: aclarubicin
9 Recruiting LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
Conditions: Leukodystrophy;   White Matter Disease
Interventions: Other: Whole Genome Sequencing (WGS);   Other: Standard Clinical Care and Diagnostic Approaches
10 Completed Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention: Drug: Warfarin
11 Active, not recruiting The Natural History of Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention:
12 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
13 Recruiting The Nosology and Etiology of Leukodystrophies of Unknown Causes
Conditions: Leukodystrophy;   Leukoencephalopathy
Intervention:
14 Recruiting Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy
Conditions: Metachromatic Leukodystrophy;   Adrenoleukodystrophy
Intervention: Genetic: transduced CD34+ hematopoietic stem cell
15 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)
16 Active, not recruiting Gene Therapy for Metachromatic Leukodystrophy
Condition: Lysosomal Storage Disease
Intervention: Genetic: Autologous CD34+ stem cells transduced with ARSA encoding lentiviral vector
17 Completed
Has Results
Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Condition: Metachromatic Leukodystrophy (MLD)
Intervention: Drug: rhASA
18 Recruiting The Myelin Disorders Biorepository Project
Conditions: Leukodystrophy;   White Matter Disease;   Leukoencephalopathies
Intervention:
19 Completed Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation
Condition: Metachromatic Leukodystrophy
Intervention: Drug: rhARSA
20 Completed The Classification and Cause of Leukodystrophies of Unknown Cause
Condition: Lysosomal Storage Disease
Intervention:

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Study has passed its completion date and status has not been verified in more than two years.