Comment Period Extended to 3/23/2015 for Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials
48 studies found for:    "leukoencephalopathy with vanishing white matter" OR "Hereditary Central Nervous System Demyelinating Diseases"
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Rank Status Study
1 Recruiting Natural History Study of Children With Metachromatic Leukodystrophy
Conditions: Leukodystrophy, Metachromatic;   Hereditary Central Nervous System Demyelinating Diseases;   Brain Diseases, Metabolic, Inborn;   Brain Diseases, Metabolic;   Brain Diseases;   Central Nervous System Diseases;   Nervous System Diseases;   Sulfatidosis;   Sphingolipidoses;   Lysosomal Storage Diseases, Nervous System;   Leukoencephalopathies;   Demyelinating Diseases;   Metabolism, Inborn Errors;   Genetic Diseases, Inborn;   Lipidoses;   Lipid Metabolism, Inborn Errors;   Lysosomal Storage Diseases;   Metabolic Diseases;   Lipid Metabolism Disorders
Intervention: Other: Natural History Study of Children With Metachromatic Leukodystrophy
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 Biomarker for Krabbe Disease
Conditions: Lysosomal Storage Diseases;   Krabbe Disease
Intervention:
4 Completed Randomized Study of Beta Interferon and Thalidomide in Patients With Adrenoleukodystrophy
Condition: Adrenoleukodystrophy
Interventions: Drug: glyceryl trierucate;   Drug: glyceryl trioleate;   Drug: interferon beta;   Drug: thalidomide
5 No longer available Single Patient Expanded Access Protocol: Metabolic Boost
Condition: Metachromatic Leukodystrophy
Intervention: Biological: Enriched Hematopoetic Stem Cell Transplant
6 Completed Stem Cell Transplantation (SCT) for Genetic Diseases
Conditions: Thrombocytopenia;   Metachromatic Leukodystrophy;   Fanconi's Anemia;   Thalassemia Major;   Pure Red-Cell Aplasia;   Inborn Errors of Metabolism
Intervention: Procedure: Stem Cell Transplantation
7 Withdrawn Safety and Pharmacodynamic Study of Sobetirome in X-Linked Adrenoleukodystrophy (X-ALD)
Conditions: X-Linked Adrenoleukodystrophy;   Adrenomyeloneuropathy
Intervention: Drug: Sobetirome
8 Unknown  Oral Glyceryl Triacetate (GTA) in Newborns With Canavan
Condition: Canavan Disease
Interventions: Dietary Supplement: GTA (Glyceryl triacetate);   Drug: GTA glyceryl triacetate
9 Recruiting A Phase 2/3 Study of the Efficacy and Safety of Hematopoietic Stem Cells Transduced With Lenti-D Lentiviral Vector for the Treatment of Childhood Cerebral Adrenoleukodystrophy (CCALD)
Conditions: Childhood Cerebral Adrenoleukodystrophy;   (X-linked Adrenoleukodystrophy Cerebral Childhood)
Interventions: Genetic: Lenti-D Drug Product;   Drug: Busulfan;   Drug: Cyclophosphamide;   Drug: Filgrastim
10 Unknown  Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
Conditions: I Cell Disease;   Fucosidosis;   Globoid Cell Leukodystrophy;   Adrenoleukodystrophy;   Mannosidosis;   Niemann-Pick Disease;   Pulmonary Complications;   Mucopolysaccharidosis I;   Mucopolysaccharidosis VI;   Metachromatic Leukodystrophy;   Gaucher's Disease;   Wolman Disease
Intervention:
11 Active, not recruiting Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)
Conditions: Pelizaeus-Merzbacher Disease;   PMD
Intervention: Biological: HuCNS-SC transplant in the lead-in phase
12 Withdrawn An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD)
Condition: Leukodystrophy, Globoid Cell
Intervention:
13 Enrolling by invitation The Natural History of Infantile Globoid Cell Leukodystrophy
Condition: Infantile Globoid Cell Leukodystrophy
Intervention:
14 Suspended Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
Condition: Adrenoleukodystrophy
Interventions: Drug: glyceryl trierucate;   Drug: glyceryl trioleate
15 Completed Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention: Drug: Warfarin
16 Completed Exercise Study of Function and Pathology for Women With X-linked Adrenoleukodystrophy
Condition: X-linked Adrenoleukodystrophy
Intervention: Behavioral: exercise training
17 Recruiting Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention: Genetic: intracerebral administration of AAVrh.10cuARSA
18 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
19 Recruiting Imaging Study of the White Matter Lesions in Children With Metachromatic Leucodystrophy
Condition: Late Infantile Metachromatic Leukodystrophy
Intervention: Other: High-field MRI (3 Teslas)
20 Active, not recruiting Diffusion Tensor Imaging (DTI) in Infants With Krabbe Disease
Condition: Krabbe Disease
Intervention:

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Indicates status has not been verified in more than two years