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27 studies found for:    "Wolman disease" OR "Wolman Disease"
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Rank Status Study
1 Completed A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype
Conditions: Lysosomal Acid Lipase Deficiency;   Wolman Disease
Intervention:
2 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
3 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)
4 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
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 Active, not recruiting Clinical Trial in Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
Condition: Lysosomal Acid Lipase Deficiency
Intervention: Drug: sebelipase alfa
7 Recruiting Screening for Lysosomal Acid Lipase Deficiency
Condition: Lysosomal Acid Lipase Deficiency
Intervention: Other: There is no intervention in the study
8 Recruiting Biomarker for Wolman Disease
Conditions: Wolman Disease;   Acid Lipase Deficiency;   Acid Cholesteryl Ester Hydrolase Deficiency, Wolman Type;   Cholesterol Ester Storage Disease
Intervention:
9 Recruiting National Lysosomal Acid Lipase Deficiency Study
Condition: Cholesteryl Ester Storage Disease
Intervention:
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 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
12 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)
13 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)
14 Terminated A Study to Identify and Characterize LAL-D Patients in High-risk Populations
Condition: Lysosomal Acid Lipase Deficiency
Intervention:
15 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:
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 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:
18 Recruiting Assessement of the Prevalence of Lysosomal Acid Lipase Deficiency in Liver Post-transplant Patients
Condition: Liver Post-transplant Patients
Intervention:
19 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:
20 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:

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