Acid Sphingomyelinase Deficiency (ASMD)
3
1
2
1
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Treatment Research
1 treatments under study, led by Olipudase Alfa
Research Leaders
20 sponsors active, led by Hospital Avicenne
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
0.0%
0 terminated out of 3 trials
100.0%
+13.4% vs benchmark
0%
0 trials in Phase 3/4
0%
0 of 1 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 1 completed trials
Clinical Trials (3)
National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children
Diagnostic Creteria of Acid Sphingomyelinase Deficiency (ASMD)
Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France
Research Network
Common Treatments
- Olipudase Alfa1
Active Sponsors
- Hospital Avicenne1
- Centre Hospitalier Eure-Seine1
- CH Henri Mondor (Aurillac)1
- Sohag University1
- Sanofi1
Top Investigators
Groupe Hospitalier Diaconesses Croix Saint-Simon
Related Conditions
- Niemann Pick Disease1