AstraZeneca: Wolman Disease trials
The registry lists 13 Wolman Disease studies with AstraZeneca as lead sponsor. 1 are open to participants now and 6 have results posted.
Phase 1 1Phase 2 5Phase 3 2No phase 7(a study in two phases counts in both)
Studies
Open to participants first, then studies with results posted, then the rest; newest registration first within each.
- Lysosomal Acid Lipase (LAL) Deficiency RegistryRecruitingNCT01633489
- Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase DeficiencyTerminatedNCT02193867Phase 2Results posted
- Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase DeficiencyCompletedNCT02112994Phase 2Results posted
- Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase DeficiencyCompletedNCT01757184Phase 3Results posted
- Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase DeficiencyCompletedNCT01488097Phase 2Results posted
- Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase DeficiencyCompletedNCT01371825Phase 2/3Results posted
- Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase DeficiencyCompletedNCT01307098Phase 1/2Results posted
- Lysosomal Acid Lipase Deficiency in Risk GroupsTerminatedNCT07455864
- Screening for Lysosomal Acid Lipase DeficiencyTerminatedNCT02926872
- An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase DeficiencyNo longer availableNCT02376751
- A Study to Identify and Characterize LAL-D Patients in High-risk PopulationsTerminatedNCT02345421
- An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease PhenotypeCompletedNCT01528917
- A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman PhenotypeCompletedNCT01358370