Acid Sphingomyelinase Deficiency
7
2
2
3
Key Insights
Highlights
Success Rate
75% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 72/100
14.3%
1 terminated out of 7 trials
75.0%
-11.5% vs benchmark
0%
0 trials in Phase 3/4
33%
1 of 3 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 3 completed trials
Clinical Trials (7)
A Study to Evaluate Safety and Tolerability of Olipudase Alfa in Pediatric and Adult Participants With Acid Sphingomyelinase Deficiency (ASMD) Who Completed the DFI12712 or the LTS13632 Study in France
PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"
GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma
A Real-world Long-term Safety and Immunogenicity Study of Olipudase Alfa Therapy in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency (ASMD)
A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)