Condition
Jansky-Bielschowsky Disease
Total Trials
3
Recruiting
0
Active
0
Completed
3
Success Rate
100.0%+13% vs avg
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 25/100
Termination Rate
0.0%
0 terminated out of 3 trials
Success Rate
100.0%
+13.5% vs benchmark
Late-Stage Pipeline
0%
0 trials in Phase 3/4
Results Transparency
100%
3 of 3 completed with results
Key Signals
3 with results100% success
Data Visualizations
Phase Distribution
3Total
P 1 (2)
P 2 (1)
Trial Status
Completed3
Trial Success Rate
100.0%
Benchmark: 86.5%
Based on 3 completed trials
Clinical Trials (3)
Showing 3 of 3 trials
NCT02678689Phase 2CompletedPrimary
A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease
NCT02485899Phase 1CompletedPrimary
An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease
NCT01907087Phase 1CompletedPrimary
A Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of Intracerebroventricular BMN 190 in Patients With Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN2) Disease
Showing all 3 trials