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261 studies found for:    Lipid Storage Diseases

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Rank Status Study
21 Completed
Has Results 		Stem Cell Transplant for Inborn Errors of Metabolism
Conditions: Adrenoleukodystrophy;   Metachromatic Leukodystrophy;   Globoid Cell Leukodystrophy;   Gaucher's Disease;   Fucosidosis;   Wolman Disease;   Niemann-Pick Disease;   Batten Disease;   GM1 Gangliosidosis;   Tay Sachs Disease;   Sandhoff Disease
Interventions: Procedure: Stem Cell Transplant;   Drug: Busulfan, Cyclophosphamide, Antithymocyte Globulin
22 Enrolling by invitation Identification of Undiagnosed Lysosomal Acid Lipase Deficiency
Conditions: Lysosomal Acid Lipase Deficiency;   Cholesterol Ester Storage Disease
Intervention: Other: Enzyme analysis
23 Completed Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Patients With Lysosomal Acid Lipase Deficiency
Conditions: Cholesterol Ester Storage Disease(CESD);   Lysosomal Acid Lipase Deficiency
Intervention: Drug: SBC-102 (sebelipase alfa)
24 Completed OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease
Condition: Gaucher Disease
Intervention: Drug: OGT 918
25 Recruiting Genetic Studies of Lysosomal Storage Disorders
Conditions: Gaucher's Disease;   Lysosomal Storage Disease
Intervention:
26 Completed Replagal Enzyme Replacement Therapy for Adults With Fabry Disease
Condition: Fabry Disease
Interventions: Drug: Replagal (Agalsidase Alfa);   Drug: Replagal
27 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:
28 Completed Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease
Condition: Gaucher Disease
Intervention: Procedure: Blood sample
29 Recruiting Exploration of Immunity in Gaucher Disease
Condition: Gaucher Disease
Intervention:
30 Terminated Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
Conditions: Acid Sphingomyelinase Deficiency;   Niemann-Pick Disease
Intervention: Drug: rhASM
31 Unknown † Detection of Fabry Disease in Chronic Renal Failure Patients in Area Provence - Alpes - Côte d'Azur
Condition: Fabry Disease
Intervention: Other: micromethod from samples taken from blood spots on filter paper
32 Completed A Study to Evaluate and Characterize the Effect of Pharmacological Chemicals on Blood From Patients With Gaucher Disease
Condition: Gaucher Disease
Intervention:
33 Recruiting Trial in Children With Growth Failure Due to Early Onset Lysosomal Acid Lipase (LAL) Deficiency/Wolman Disease
Conditions: Lysosomal Acid Lipase Deficiency;   Wolman Disease
Intervention: Drug: Sebelipase alfa (SBC-102)
34 Terminated Children With Lysosomal Acid Lipase Deficiency Who Previously Received Treatment With SBC-102
Conditions: Lysosomal Acid Lipase Deficiency;   Wolman Disease
Intervention: Drug: SBC-102
35 Enrolling by invitation Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)
Condition: Gaucher Disease
Intervention: Drug: Imiglucerase
36 Unknown † Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
Condition: Gangliosidoses GM2
Intervention: Drug: miglustat
37 Recruiting Physician Initiated Request for Migalastat in Individual Patients With Fabry Disease
Condition: Fabry Disease
Intervention: Drug: migalastat HCl
38 Recruiting Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients
Condition: Fabry Disease
Intervention: Drug: PRX-102
39 Recruiting Open-Label Phase 3 Long-Term Safety Study of Migalastat
Condition: Fabry Disease
Intervention: Drug: migalastat HCl 150mg
40 Enrolling by invitation An Extension of a Phase 1/2, Open Label, Dose Ranging Study of PRX-102 in Adult Fabry Patients
Condition: Fabry Disease
Intervention: Drug: PRX-102
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† Indicates status has not been verified in more than two years