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Clinical Trials on Cholesterol Ester Storage Disease

Total 28 results

    • NCT01528917
      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
    • NCT01791452
      Unknown status
      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
    • NCT02372513
      Completed
      National Lysosomal Acid Lipase Deficiency Study
      Conditions: Cholesteryl Ester Storage Disease
    • NCT01716728
      Unknown status
      Identification of Undiagnosed Lysosomal Acid Lipase Deficiency
      Conditions: Lysosomal Acid Lipase Deficiency; Cholesterol Ester Storage Disease
    • NCT01884220
      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
    • NCT02383641
      Active, not recruiting
      Biomarker for Wolman Disease (BioWolman)
      Conditions: Acid Lipase Deficiency; Acid Cholesteryl Ester Hydrolase Deficiency, Wolman Type; Cholesterol Ester Storage Disease
    • NCT01488097
      Completed
      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; LAL-Deficiency
    • NCT01307098
      Completed
      Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency
      Conditions: Cholesterol Ester Storage Disease(CESD); Lysosomal Acid Lipase Deficiency; LAL-Deficiency
    • NCT01633489
      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
    • NCT02112994
      Completed
      Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency
      Conditions: Lysosomal Acid Lipase Deficiency
    • NCT02376751
      No longer available
      An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
      Conditions: Lysosomal Acid Lipase Deficiency
    • NCT01757184
      Completed
      Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency
      Conditions: Lysosomal Acid Lipase Deficiency
    • NCT01371825
      Completed
      Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
      Conditions: Lysosomal Acid Lipase Deficiency; Wolman Disease
    • NCT02193867
      Terminated
      Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
      Conditions: Lysosomal Acid Lipase Deficiency
    • NCT04532047
      Not yet recruiting
      In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases
      Conditions: MPS I; MPS II; MPS IVA; MPS VI; Mps VII; Gaucher Disease, Type 2; Gaucher Disease, Type 3; Pompe Disease Infantile-Onset; Wolman Disease
    • NCT04652713
      Recruiting
      Breakfast for Young Women
      Conditions: Obesity; Wolman Disease; Nutrition
    • NCT01358370
      Completed
      A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype
      Conditions: Lysosomal Acid Lipase Deficiency; Wolman Disease
    • NCT03984149
      Recruiting
      Lipa Gene Mutation in PED-LIPIGEN (Pediatric FH Subjects)
      Conditions: Lysosomal Acid Lipase Deficiency
    • NCT02926872
      Terminated
      Screening for Lysosomal Acid Lipase Deficiency
      Conditions: Lysosomal Acid Lipase Deficiency
    • NCT02852304
      Unknown status
      Assessment of the Prevalence of Lysosomal Acid Lipase Deficiency in Patients Waiting for a Liver Transplant.
      Conditions: Patients Waiting for a Liver Transplant.
    • NCT02851550
      Unknown status
      Assessement of the Prevalence of Lysosomal Acid Lipase Deficiency in Liver Post-transplant Patients
      Conditions: Liver Post-transplant Patients
    • NCT02345421
      Terminated
      A Study to Identify and Characterize LAL-D Patients in High-risk Populations
      Conditions: Lysosomal Acid Lipase Deficiency
    • NCT01586455
      Active, not recruiting
      Human Placental-Derived Stem Cell Transplantation
      Conditions: Mucopolysaccharidosis I; Mucopolysaccharidosis VI; Adrenoleukodystrophy; Niemann-Pick Disease; Metachromatic Leukodystrophy; Wolman Disease; Krabbe's Disease; Gaucher's Disease; Fucosidosis; Batten Disease; Severe Aplastic Anemia; Diamond-Blackfan Anemia; Amegakaryocytic Thrombocytopenia; Myelodysplastic Syndrome; Acute Myelogenous Leukemia; Acute Lymphocytic Leukemia
    • NCT00176904
      Completed
      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
    • NCT00005900
      Unknown status
      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
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