Not applicableUnknownNCT04792671What this trial is testingPrevalence and Risk Factors of Women Mental Health DisordersWho this might be right forPsychiatric ProblemWolman Disease Assiut University 200
Not applicableLooking for participantsNCT07455864What this trial is testingLysosomal Acid Lipase Deficiency in Risk GroupsWho this might be right forLysosomal Acid Lipase Deficiency AstraZeneca 1,200
Testing effectiveness (Phase 2)Study completedNCT01307098What this trial is testingSafety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase DeficiencyWho this might be right forCholesterol Ester Storage Disease(CESD)Lysosomal Acid Lipase DeficiencyLAL-Deficiency Alexion Pharmaceuticals, Inc. 9
Not applicableStudy completedNCT01528917What this trial is testingAn Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease PhenotypeWho this might be right forCholesterol Ester Storage Disease(CESD)Lysosomal Acid Lipase Deficiency Alexion Pharmaceuticals, Inc. 49
Testing effectiveness (Phase 2)Ended earlyNCT02193867What this trial is testingClinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase DeficiencyWho this might be right forLysosomal Acid Lipase Deficiency Alexion Pharmaceuticals, Inc. 10
Not applicableUnknownNCT03564002What this trial is testingMetabolic Effects of Very Low Carbohydrate Ketogenic Diet in Subjects With Severe ObesityWho this might be right forLysosomal Acid Lipase DeficiencyObesityKetogenic Dieting+1 more University Of Perugia 60
Not applicableLooking for participantsNCT05619900What this trial is testingRegistry of Patients Diagnosed With Lysosomal Storage DiseasesWho this might be right forMucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV A+5 more University of California, San Francisco 250
Testing effectiveness (Phase 2)Study completedNCT00383448What this trial is testingHSCT for High Risk Inherited Inborn ErrorsWho this might be right forAdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+6 more Masonic Cancer Center, University of Minnesota 38
Not applicableUnknownNCT03984149What this trial is testingLipa Gene Mutation in PED-LIPIGEN (Pediatric FH Subjects)Who this might be right forLysosomal Acid Lipase Deficiency Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) 1,000
Large-scale testing (Phase 3)Study completedNCT01757184What this trial is testingAcid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase DeficiencyWho this might be right forLysosomal Acid Lipase Deficiency Alexion Pharmaceuticals, Inc. 66
Testing effectiveness (Phase 2)Ended earlyNCT00668564What this trial is testingHematopoietic Stem Cell Transplantation (HCT) for Inborn Errors of MetabolismWho this might be right forHurler's SyndromeMaroteaux-Lamy SyndromeSly Syndrome+8 more Masonic Cancer Center, University of Minnesota 18
Not applicableNo Longer AvailableNCT02376751What this trial is testingAn Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase DeficiencyWho this might be right forLysosomal Acid Lipase Deficiency Alexion Pharmaceuticals, Inc.
Not applicableStudy completedNCT04652713What this trial is testingBreakfast for Young WomenWho this might be right forObesityWolman DiseaseNutrition University of Aarhus 30
Not applicableStudy completedNCT05687474What this trial is testingBaby Detect : Genomic Newborn ScreeningWho this might be right forCongenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more Centre Hospitalier Universitaire de Liege 6,824
Not applicableStudy completedNCT01884220What this trial is testingWolman/CESD Natural History Chart Review and Longitudinal Follow-UpWho this might be right forWolman DiseaseCholesterol Ester Storage DiseaseAcid Cholesteryl Ester Hydrolase Deficiency, Type 2 Children's Hospital Medical Center, Cincinnati 4
Not applicableUnknownNCT01791452What this trial is testingNovel Association of Cholesterol Ester Storage Disease Due to Lysosomal Acid Lipase Deficiency and Non-Alcoholic Fatty Liver Disease: A Prospective Clinical StudyWho this might be right forNon-alcoholic Fatty Liver DiseaseCholesterol Ester Storage Disease Assy Nimer
Testing effectiveness (Phase 2)Study completedNCT02112994What this trial is testingSafety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase DeficiencyWho this might be right forLysosomal Acid Lipase Deficiency Alexion Pharmaceuticals, Inc. 31
Not applicableStudy completedNCT04678713What this trial is testingMaximal Fat Oxidation and Fuel Use During ExerciseWho this might be right forObesity University of Copenhagen 40
Not applicableWithdrawnNCT02383641What this trial is testingBiomarker for Wolman Disease (BioWolman)Who this might be right forAcid Lipase DeficiencyAcid Cholesteryl Ester Hydrolase Deficiency, Wolman TypeCholesterol Ester Storage Disease CENTOGENE GmbH Rostock
Testing effectiveness (Phase 2)Study completedNCT01488097What this trial is testingExtension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase DeficiencyWho this might be right forCholesterol Ester Storage Disease (CESD)Lysosomal Acid Lipase DeficiencyLAL-Deficiency Alexion Pharmaceuticals, Inc. 8