Trials / Conditions / Lysosomal Acid Lipase Deficiency

Lysosomal Acid Lipase Deficiency

19 registered clinical trials studyying Lysosomal Acid Lipase Deficiency — 4 currently recruiting.

Status	Trial	Sponsor	Phase
Recruiting	Lysosomal Acid Lipase Deficiency in Risk Groups NCT07455864	AstraZeneca	—
Completed	Baby Detect : Genomic Newborn Screening NCT05687474	Centre Hospitalier Universitaire de Liege	—
Enrolling By Invitation	ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program NCT05368038	Albert Einstein College of Medicine	—
Enrolling By Invitation	Early Check: Expanded Screening in Newborns NCT03655223	RTI International	—
Unknown	Lipa Gene Mutation in PED-LIPIGEN (Pediatric FH Subjects) NCT03984149	Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi)	—
Terminated	Screening for Lysosomal Acid Lipase Deficiency NCT02926872	Alexion Pharmaceuticals, Inc.	—
Unknown	Metabolic Effects of Very Low Carbohydrate Ketogenic Diet in Subjects With Severe Obesity NCT03564002	University Of Perugia	—
Terminated	A Study to Identify and Characterize LAL-D Patients in High-risk Populations NCT02345421	Alexion Pharmaceuticals, Inc.	—
Completed	Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency NCT02112994	Alexion Pharmaceuticals, Inc.	Phase 2
Terminated	Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency NCT02193867	Alexion Pharmaceuticals, Inc.	Phase 2
Recruiting	Lysosomal Acid Lipase (LAL) Deficiency Registry NCT01633489	Alexion Pharmaceuticals, Inc.	—
Completed	Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase D NCT01757184	Alexion Pharmaceuticals, Inc.	Phase 3
Unknown	Identification of Undiagnosed Lysosomal Acid Lipase Deficiency NCT01716728	Massachusetts General Hospital	N/A
Completed	Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in A NCT01488097	Alexion Pharmaceuticals, Inc.	Phase 2
Completed	An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phe NCT01528917	Alexion Pharmaceuticals, Inc.	—
Completed	Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Gro NCT01371825	Alexion Pharmaceuticals, Inc.	Phase 2 / Phase 3
Completed	Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Ac NCT01307098	Alexion Pharmaceuticals, Inc.	Phase 1 / Phase 2
Completed	A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype NCT01358370	Alexion Pharmaceuticals, Inc.	—
No Longer Available	An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency NCT02376751	Alexion Pharmaceuticals, Inc.	—