Condition
Gangliosidoses, GM2
Total Trials
3
Recruiting
1
Active
1
Completed
2
Success Rate
100.0%+13% vs avg
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 30/100
Termination Rate
0.0%
0 terminated out of 3 trials
Success Rate
100.0%
+13.5% vs benchmark
Late-Stage Pipeline
33%
1 trials in Phase 3/4
Results Transparency
0%
0 of 2 completed with results
Key Signals
100% success
Data Visualizations
Phase Distribution
3Total
P 1 (1)
P 2 (1)
P 3 (1)
Trial Status
Completed2
Recruiting1
Trial Success Rate
100.0%
Benchmark: 86.5%
Based on 2 completed trials
Clinical Trials (3)
Showing 3 of 3 trials
NCT07082543Phase 3RecruitingPrimary
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
NCT00418847Phase 2CompletedPrimary
Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis
NCT01102686Phase 1CompletedPrimary
Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)
Showing all 3 trials