Niemann-Pick Disease Type C1
9
0
1
4
Key Insights
Highlights
Success Rate
80% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 62/100
11.1%
1 terminated out of 9 trials
80.0%
-6.5% vs benchmark
11%
1 trials in Phase 3/4
25%
1 of 4 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 4 completed trials
Clinical Trials (9)
Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Disease as Compared to Healthy Neurotypical Controls
Early Check: Expanded Screening in Newborns
Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1
Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1
Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients
Hydroxypropyl Beta Cyclodextrin for Niemann-Pick Type C1 Disease
Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C
Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)
Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease