Type 1 Gaucher Disease
6
0
0
3
Key Insights
Highlights
Success Rate
60% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 65/100
33.3%
2 terminated out of 6 trials
60.0%
-26.5% vs benchmark
17%
1 trials in Phase 3/4
100%
3 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 (6)
A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02
Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease
A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients
A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease
A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101