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50 studies found for:    "Leukodystrophy"
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Study Details
Participant Details
Identifiers
Dates
Rank Status Study
1 Active, not recruiting The Natural History of Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention:
2 Recruiting Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention: Genetic: intracerebral administration of AAVrh.10cuARSA
3 Completed Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)
Condition: Metachromatic Leukodystrophy (MLD)
Intervention: Biological: Recombinant human arylsulfatase A
4 Completed Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention: Drug: Warfarin
5 Withdrawn An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD)
Condition: Leukodystrophy, Globoid Cell
Intervention:
6 Completed The Natural History of Infantile Globoid Cell Leukodystrophy
Condition: Infantile Globoid Cell Leukodystrophy
Intervention:
7 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)
8 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)
9 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
10 Completed
Has Results
Metazym for the Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)
Condition: Metachromatic Leukodystrophy (MLD)
Intervention: Drug: rhASA
11 Recruiting Natural History of the Leukodystrophies
Condition: Leukodystrophies
Intervention:
12 Completed Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation
Condition: Metachromatic Leukodystrophy
Intervention: Drug: rhARSA
13 Recruiting Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy
Conditions: Metachromatic Leukodystrophy;   Adrenoleukodystrophy
Intervention: Genetic: transduced CD34+ hematopoietic stem cell
14 Terminated
Has Results
Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD
Condition: Late Infantile Metachromatic Leukodystrophy
Intervention: Drug: HGT-1111
15 Recruiting The Nosology and Etiology of Leukodystrophies of Unknown Causes
Conditions: Leukodystrophy;   Leukoencephalopathy
Intervention:
16 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
17 No longer available Allogeneic Stem Cell Transplantation for the Treatment of Multiple Sclerosis (Compassionate Use)
Condition: Metachromatic Leukodystrophy
Intervention: Biological: Enriched Hematopoetic Stem Cell Infusion
18 Recruiting Aclarubicin for the Treatment of Retinal Vasculopathy With Cerebral Leukodystrophy
Conditions: Vasculopathy, Retinal, With Cerebral Leukodystrophy;   Cerebroretinal Vasculopathy, Hereditary
Intervention: Drug: aclarubicin
19 No longer available Single Patient Expanded Access Protocol: Metabolic Boost
Condition: Metachromatic Leukodystrophy
Intervention: Biological: Enriched Hematopoetic Stem Cell Infusion
20 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:

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