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266 studies found for:    "Sphingolipidosis"
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Rank Status Study
1 Recruiting LYSO-PROVE - Determine the Prognostic Value of Lyso-Gb1 for Monitoring the Progress of Gaucher Disease
Conditions: Lysosomal Storage Diseases;   Gaucher Disease;   Sphingolipidoses
Intervention:
2 Recruiting Biomarker for Gaucher Disease
Conditions: Lysosomal Storage Diseases;   Gaucher Disease;   Sphingolipidoses
Intervention:
3 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:
4 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
5 Recruiting MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Conditions: Mucopolysaccharidosis Disorders;   Hurler Syndrome;   Hunter Syndrome;   Maroteaux Lamy Syndrome;   Sly Syndrome;   Alpha-Mannosidosis;   Fucosidosis;   Aspartylglucosaminuria;   Glycoprotein Metabolic Disorders;   Sphingolipidoses;   Recessive Leukodystrophies;   Globoid Cell Leukodystrophy;   Metachromatic Leukodystrophy;   Niemann-Pick B;   Niemann-Pick C Subtype 2;   Sphingomyelin Deficiency;   Peroxisomal Disorders;   Adrenoleukodystrophy With Cerebral Involvement;   Zellweger Syndrome;   Neonatal Adrenoleukodystrophy;   Infantile Refsum Disease;   Acyl-CoA Oxidase Deficiency;   D-Bifunctional Enzyme Deficiency;   Multifunctional Enzyme Deficiency;   Alpha-methylacyl-CoA Racmase Deficiency;   Mitochondrial Neurogastrointestingal Encephalopathy;   Severe Osteopetrosis;   Hereditary Leukoencephalopathy;   Inherited Metabolic Disorders
Interventions: Biological: Stem Cell Transplantation;   Drug: IMD Preparative Regimen;   Drug: Osteopetrosis Only Preparative Regimen;   Drug: Osteopetrosis Haploidentical Only Preparative Regimen;   Drug: cALD SR-A (Standard-Risk, Regimen A);   Drug: cALD SR-B (Standard-Risk, Regimen B);   Drug: cALD HR-D (High-Risk, Regimen C);   Drug: cALD HR-D (High-Risk, Regimen D)
6 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:
7 Active, not recruiting Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders
Conditions: Mucopolysaccharidosis;   Hurler Syndrome;   Hunter Syndrome;   Maroteaux-Lamy Syndrome;   Sly Syndrome;   Alpha Mannosidosis;   Fucosidosis;   Aspartylglucosaminuria;   Adrenoleukodystrophy (ALD);   Krabbe Disease;   Metachromatic Leukodystrophy (MLD);   Sphingolipidoses;   Peroxisomal Disorders
Interventions: Drug: Campath-1H;   Drug: Cyclophosphamide;   Drug: Busulfan;   Procedure: Allogeneic stem cell transplantation;   Drug: Cyclosporine A;   Drug: Mycophenolate Mofetil
8 Completed Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease
Condition: Gaucher's Disease
Intervention: Genetic: human glucocerebrosidase gene into autologous peripheral blood stem cells
9 Unknown  Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease
Condition: Gaucher's Disease
Intervention: Drug: glucocerebrosidase
10 Completed Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease
Conditions: Gaucher's Disease;   Osteopenia
Interventions: Drug: alendronate sodium;   Drug: calcium carbonate;   Drug: cholecalciferol
11 Recruiting Krabbe Disease Global Patient Registry
Condition: Krabbe Disease
Intervention:
12 Completed Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease
Condition: Gaucher Disease
Intervention: Procedure: Blood sample
13 Recruiting Biomarker for Krabbe Disease
Conditions: Lysosomal Storage Diseases;   Krabbe Disease
Intervention:
14 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:
15 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
16 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
17 Suspended Effect of Glycerol Trierucate on Clinical Course of Adrenoleukodystrophy
Condition: Adrenoleukodystrophy
Interventions: Drug: glyceryl trierucate;   Drug: glyceryl trioleate
18 Recruiting Study of the Relation Between Lipid Myocardial Overload Evaluated by Cardiac Magnetic Resonance Imaging (MRI), Alteration of Longitudinal Myocardial Deformations by Echocardiography, and Clinical Achievements (Functional, Biological and Electrical) in Fabry Disease, and Its Outcomes.
Condition: Fabry Disease
Interventions: Diagnostic Test: Echocardiography at T0;   Diagnostic Test: Exercise test;   Biological: Biological assays;   Device: MRI with contrast agent injection;   Device: MRI without contrast agent injection;   Diagnostic Test: Echocardiography at M24
19 Not yet recruiting Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease
Condition: Type 1 Gaucher Disease
Intervention: Drug: Elelyso 60 units/kg
20 Not yet recruiting Safety and Efficacy of PRX 102 in Patients With Fabry Disease Currently Treated With REPLAGAL® (Agalsidase Alfa)
Condition: Fabry Disease
Intervention: Biological: PRX-102

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