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53 studies found for:    Acid Lipase Disease
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Rank Status Study
1 Active, not recruiting
Has Results
SurVival of Lysosomal Acid Lipase Deficiency (LAL-D) Infants Treated With SebelipAse aLfa
Conditions: Lysosomal Acid Lipase Deficiency;   Wolman Disease
Intervention: Drug: Sebelipase alfa (SBC-102)
2 Active, not recruiting Clinical Trial in Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
Condition: Lysosomal Acid Lipase Deficiency
Intervention: Drug: sebelipase alfa
3 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
4 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
5 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
6 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)
7 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)
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 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
10 Enrolling by invitation Identification of Undiagnosed Lysosomal Acid Lipase Deficiency
Conditions: Lysosomal Acid Lipase Deficiency;   Cholesterol Ester Storage Disease
Intervention: Other: enzyme analysis
11 Completed A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype
Conditions: Lysosomal Acid Lipase Deficiency;   Wolman Disease
Intervention:
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;   LAL-Deficiency
Intervention:
13 Recruiting Assessement of the Prevalence of Lysosomal Acid Lipase Deficiency in Liver Post-transplant Patients
Condition: Liver Post-transplant Patients
Intervention:
14 Terminated A Study to Identify and Characterize LAL-D Patients in High-risk Populations
Condition: Lysosomal Acid Lipase Deficiency
Intervention:
15 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:
16 Unknown  Novel Association of Cholesterol Ester Storage Disease Due to Lysosomal Acid Lipase Deficiency and Non-Alcoholic Fatty Liver Disease: A Prospective Clinical Study
Conditions: Non-alcoholic Fatty Liver Disease;   Cholesterol Ester Storage Disease
Intervention:
17 Recruiting National Lysosomal Acid Lipase Deficiency Study
Condition: Cholesteryl Ester Storage Disease
Intervention:
18 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.
19 Recruiting Hypertriglyceridaemia - Cause and Effects
Condition: Hypertriglyceridaemia
Intervention:
20 Recruiting Biomarker for Wolman Disease
Conditions: Wolman Disease;   Acid Lipase Deficiency;   Acid Cholesteryl Ester Hydrolase Deficiency, Wolman Type;   Cholesterol Ester Storage Disease
Intervention:

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Indicates status has not been verified in more than two years