26 studies found for:    LYSOSOMAL ACID LIPASE DEFICIENCY
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Rank Status Study
1 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
2 Completed A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype
Conditions: Lysosomal Acid Lipase Deficiency;   Wolman Disease
Intervention:
3 No longer available An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
Condition: Lysosomal Acid Lipase Deficiency
Intervention: Drug: sebelipase alfa
4 Enrolling by invitation Identification of Undiagnosed Lysosomal Acid Lipase Deficiency
Conditions: Lysosomal Acid Lipase Deficiency;   Cholesterol Ester Storage Disease
Intervention: Other: enzyme analysis
5 Active, not recruiting Clinical Trial in Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
Condition: Lysosomal Acid Lipase Deficiency
Intervention: Drug: sebelipase alfa
6 Active, not recruiting Safety and Efficacy Study of Sebelipase Alfa in Patients With Lysosomal Acid Lipase Deficiency
Condition: Lysosomal Acid Lipase Deficiency
Intervention: Drug: sebelipase alfa
7 Active, not recruiting
Has Results
A Multicenter Study of SBC-102 (Sebelipase Alfa) in Patients With Lysosomal Acid Lipase Deficiency/ ARISE (Acid Lipase Replacement Investigating Safety and Efficacy)
Conditions: Cholesterol Ester Storage Disease (CESD);   Lysosomal Acid Lipase Deficiency
Interventions: Drug: SBC-102 [sebelipase alfa] (1 mg/kg);   Drug: Placebo
8 Completed An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype
Conditions: Cholesterol Ester Storage Disease(CESD);   Lysosomal Acid Lipase Deficiency
Intervention:
9 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)
10 Active, not recruiting
Has Results
Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency
Conditions: Cholesterol Ester Storage Disease(CESD);   Lysosomal Acid Lipase Deficiency
Intervention: Drug: SBC-102 (sebelipase alfa)
11 Active, not recruiting
Has Results
SurVival of LAL-D Infants Treated With SebelipAse ALfa
Conditions: Lysosomal Acid Lipase Deficiency;   Wolman Disease
Intervention: Drug: Sebelipase alfa (SBC-102)
12 Recruiting Lysosomal Acid Lipase (LAL) Deficiency Registry
Conditions: Lysosomal Acid Lipase Deficiency;   Cholesterol Ester Storage Disease;   Wolman Disease;   Acid Cholesteryl Ester Hydrolase Deficiency, Type 2;   Acid Lipase Deficiency;   LIPA Deficiency
Intervention:
13 Terminated A Study to Identify and Characterize LAL-D Patients in High-risk Populations
Condition: Lysosomal Acid Lipase Deficiency
Intervention:
14 Recruiting National Lysosomal Acid Lipase Deficiency Study
Condition: Cholesteryl Ester Storage Disease
Intervention:
15 Recruiting Biomarker for Wolman Disease
Conditions: Wolman Disease;   Acid Lipase Deficiency;   Acid Cholesteryl Ester Hydrolase Deficiency, Wolman Type;   Cholesterol Ester Storage Disease
Intervention:
16 Completed Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up
Conditions: Wolman Disease;   Cholesterol Ester Storage Disease;   Acid Cholesteryl Ester Hydrolase Deficiency, Type 2
Intervention: Other: There are no interventions in this study.
17 Recruiting Assessement of the Prevalence of Lysosomal Acid Lipase Deficiency in Liver Post-transplant Patients
Condition: Liver Post-transplant Patients
Intervention:
18 Recruiting Assessment of the Prevalence of Lysosomal Acid Lipase Deficiency in Patients Waiting for a Liver Transplant.
Condition: Patients Waiting for a Liver Transplant.
Intervention:
19 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:
20 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

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Indicates status has not been verified in more than two years