Lysosomal Acid Lipase Deficiency
19
2
2
8
Key Insights
Highlights
Success Rate
73% trial completion
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 47/100
15.8%
3 terminated out of 19 trials
72.7%
-13.8% vs benchmark
5%
1 trials in Phase 3/4
75%
6 of 8 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 8 completed trials
Clinical Trials (19)
Lysosomal Acid Lipase Deficiency in Risk Groups
Lysosomal Acid Lipase (LAL) Deficiency Registry
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Baby Detect : Genomic Newborn Screening
Early Check: Expanded Screening in Newborns
Lipa Gene Mutation in PED-LIPIGEN (Pediatric FH Subjects)
Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency
Metabolic Effects of Very Low Carbohydrate Ketogenic Diet in Subjects With Severe Obesity
Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency
Clinical Study In Infants With Rapidly Progressive Lysosomal Acid Lipase Deficiency
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency
Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of SBC-102 (Sebelipase Alfa) in Adult Subjects With Lysosomal Acid Lipase Deficiency
Screening for Lysosomal Acid Lipase Deficiency
An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype
A Retrospective Natural History Study of Patients With Lysosomal Acid Lipase Deficiency/Wolman Phenotype
An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency
A Study to Identify and Characterize LAL-D Patients in High-risk Populations
Identification of Undiagnosed Lysosomal Acid Lipase Deficiency