Other
Beyond Batten Disease Foundation
Total Trials
3
Recruiting
1
Active
1
Completed
1
Success Rate
100.0%+13% vs avg
Key Highlights
Risk & Performance
Pipeline Risk Assessment
Pipeline Risk Assessment
Based on historical performance
Low Risk
Score: 5/100
Failure Rate
0.0%
0 terminated/withdrawn out of 3 trials
Success Rate
100.0%
+13.4% vs industry average
Late-Stage Pipeline
33%
1 trials in Phase 3/4
Results Transparency
100%
1 of 1 completed trials have results
Key Signals
1 recruiting1 with results
Enrollment reporting
Analytics
Phase 3
1(50.0%)
Phase 1/2
1(50.0%)
2Total
Phase 3(1)
Phase 1/2(1)
Activity Timeline
Global Presence
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Clinical Trials (3)
Showing 3 of 3 trials
NCT01793168Recruiting
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Role: collaborator
NCT07882290Phase 3Not Yet Recruiting
Study With Batten-1 (Miglustat Liquid Formulation) to Treat Ceroid Lipofuscinosis Neuronal 3 (CLN3) Disease
Role: lead
NCT05174039Phase 1/2Completed
An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease
Role: lead
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