| ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program (NCT05368038) | Albert Einstein College of Medicine | 2021-05-10 |
| Early Check: Expanded Screening in Newborns (NCT03655223) | RTI International | 2018-10-15 |
| Lipa Gene Mutation in PED-LIPIGEN (Pediatric FH Subjects) (NCT03984149) | Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) | 2017-09-01 |
| Screening for Lysosomal Acid Lipase Deficiency (NCT02926872) | Alexion Pharmaceuticals, Inc. | 2016-11-01 |
| Metabolic Effects of Very Low Carbohydrate Ketogenic Diet in Subjects With Severe Obesity (NCT03564002) | University Of Perugia | 2016-10-01 |
| A Study to Identify and Characterize LAL-D Patients in High-risk Populations (NCT02345421) | Alexion Pharmaceuticals, Inc. | 2014-12-01 |
| Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency (NCT02112994) | Alexion Pharmaceuticals, Inc. | 2014-06-24 |
| Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency (NCT02193867) | Alexion Pharmaceuticals, Inc. | 2014-06-06 |
| Lysosomal Acid Lipase (LAL) Deficiency Registry (NCT01633489) | Alexion Pharmaceuticals, Inc. | 2013-05-30 |
| Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency (NCT01757184) | Alexion Pharmaceuticals, Inc. | 2013-01-22 |
| Identification of Undiagnosed Lysosomal Acid Lipase Deficiency (NCT01716728) | Massachusetts General Hospital | 2012-08-01 |
| Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency (NCT01488097) | Alexion Pharmaceuticals, Inc. | 2011-12-12 |
| An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype (NCT01528917) | Alexion Pharmaceuticals, Inc. | 2011-06-01 |
| Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency (NCT01371825) | Alexion Pharmaceuticals, Inc. | 2011-05-04 |
| Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency (NCT01307098) | Alexion Pharmaceuticals, Inc. | 2011-04-25 |
| A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype (NCT01358370) | Alexion Pharmaceuticals, Inc. | 2010-11-01 |
| An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency (NCT02376751) | Alexion Pharmaceuticals, Inc. | |