Gaucher Disease, Type 3
13
2
4
2
Key Insights
Highlights
Success Rate
67% trial completion
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
7.7%
1 terminated out of 13 trials
66.7%
-19.9% vs benchmark
15%
2 trials in Phase 3/4
100%
2 of 2 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 2 completed trials
Clinical Trials (13)
MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)
Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)
Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension
A Phase I/II Clinical Study to Evaluate the Safety and Efficacy of VGN-R08b in Patients With Type III Gaucher's Disease
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3)
A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease
Effect of Enzyme Replacement Therapy on Cardiac Function in Children With Gaucher Disease Type 3
An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3
Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3
Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease
Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease
The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease