51 studies found for:    "leukoencephalopathy with vanishing white matter" OR "Hereditary Central Nervous System Demyelinating Diseases"
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Rank Status Study
1 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:
2 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
3 Recruiting Biomarker for Krabbe Disease
Conditions: Lysosomal Storage Diseases;   Krabbe Disease
Intervention:
4 No longer available Single Patient Expanded Access Protocol: Metabolic Boost
Condition: Metachromatic Leukodystrophy
Intervention: Biological: Enriched Hematopoetic Stem Cell Transplant
5 Terminated
Has Results
Hematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of Metabolism
Conditions: Hurler's Syndrome;   Maroteaux-Lamy Syndrome;   Sly Syndrome;   Alpha Mannosidosis;   Fucosidosis;   Aspartylglucosaminuria;   Sphingolipidoses;   Krabbe Disease;   Wolman's Disease;   Niemann-Pick Disease Type B;   Niemann-Pick Disease, Type C
Interventions: Procedure: Stem Cell Transplantation;   Drug: Cyclophosphamide;   Drug: Campath-1H;   Drug: Busulfan
6 Recruiting Gene Therapy for Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention: Genetic: Autologous CD34+ stem cells transduced with ARSA encoding lentiviral vector
7 Unknown  Study of Bile Acids in Patients With Peroxisomal Disorders
Conditions: Infantile Refsum's Disease;   Zellweger Syndrome;   Bifunctional Enzyme Deficiency;   Adrenoleukodystrophy
Interventions: Drug: chenodeoxycholic acid;   Drug: cholic acid;   Drug: ursodiol
8 Completed Effect of Warfarin in the Treatment of Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention: Drug: Warfarin
9 Unknown  Oral Glyceryl Triacetate (GTA) in Newborns With Canavan
Condition: Canavan Disease
Interventions: Dietary Supplement: GTA (Glyceryl triacetate);   Drug: GTA glyceryl triacetate
10 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
11 Completed A Clinical Trial for AMN: Validation of Biomarkers of Oxidative Stress, Efficacy and Safety of a Mixture of Antioxidants
Condition: Adrenomyeloneuropathy
Interventions: Drug: N-acetylcysteine;   Drug: lipoic acid;   Drug: vitamin E
12 Completed The Effect of Bezafibrate on the Level of Very Long Chain Fatty Acids (VLCFA) in X-linked Adrenoleukodystrophy (X-ALD)
Conditions: X-linked Adrenoleukodystrophy;   Adrenomyeloneuropathy
Intervention: Drug: Bezafibrate
13 Recruiting The Natural History of Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention:
14 Recruiting Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy
Condition: Metachromatic Leukodystrophy
Intervention: Genetic: intracerebral administration of AAVrh.10cuARSA
15 Active, not recruiting Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
Conditions: Hurler Syndrome (MPS I);   Hurler-Scheie Syndrome With Early Neurologic Involvement and/or Sensitization to Enzyme Replacement Therapy (ERT);   Hunter Syndrome (MPS II);   Sanfilippo Syndrome (MPS III);   Krabbe Disease (Globoid Leukodystrophy);   Metachromatic Leukodystrophy (MLD);   Adrenoleukodystrophy (ALD and AMN);   Sandhoff Disease;   Tay Sachs Disease;   Pelizaeus Merzbacher (PMD);   Niemann-Pick Disease;   Alpha-mannosidosis
Intervention: Biological: Enriched Hematopoetic Stem Cell Transplantation/novel platform technology
16 Active, not recruiting Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)
Condition: Metachromatic Leukodystrophy
Intervention: Biological: Recombinant human arylsulfatase A
17 Recruiting A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Conditions: Hematologic Malignancies;   Certain:;   Inherited Disorders of Metabolism;   Inherited Abnormalities of Erythrocyte Differentiation or Function;   Inherited Abnormalities of Platelets;   Disorders of the Immune System;   Histiocytic Disorders;   X-linked Adrenoleukodystrophy;   Primary Immunodeficiency Diseases;   Bone Marrow Failure;   Beta-thalassemia
Intervention: Drug: A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)
18 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)
19 Recruiting HSCT for High Risk Inherited Inborn Errors
Conditions: Adrenoleukodystrophy;   Metachromatic Leukodystrophy;   Globoid Cell Leukodystrophy;   Tay Sachs Disease;   Sandhoffs Disease;   Wolman Disease;   I-Cell Disease;   Sanfilippo Syndrome;   GM1 Gangliosidosis
Interventions: Drug: Clofarabine;   Procedure: Total body Irradiation;   Drug: Melphalan;   Biological: Hematopoietic Stem Cell Transplantation;   Drug: Alemtuzumab;   Drug: mycophenylate mofetil;   Device: Cyclosporine A;   Drug: Hydroxyurea
20 Suspended Lithium and Acetate for Canavan Disease
Conditions: Canavan Disease;   Infantile;   Deficiency Disease;   Aspartoacylase;   Leukodystrophy, Spongiform
Intervention: Drug: Lithium Gluconate (drug) Glyceryl Triacetate GTA (drug)

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Indicates status has not been verified in more than two years