Niemann-Pick Disease
8
0
0
3
Key Insights
Highlights
Success Rate
60% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 100/100
25.0%
2 terminated out of 8 trials
60.0%
-26.5% 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 (8)
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
Biomarker for Niemann Pick Type C Disease (BioNPC)
Human Placental-Derived Stem Cell Transplantation
Stem Cell Transplant for Inborn Errors of Metabolism
Investigating Lysosomal Storage Diseases in Minority Groups
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells
Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation