Condition
Late-onset Pompe Disease
Total Trials
5
Recruiting
0
Active
0
Completed
1
Success Rate
25.0%-62% vs avg
Key Insights
Highlights
Success Rate
25% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 65/100
Termination Rate
60.0%
3 terminated out of 5 trials
Success Rate
25.0%
-61.5% vs benchmark
Late-Stage Pipeline
20%
1 trials in Phase 3/4
Results Transparency
200%
2 of 1 completed with results
Key Signals
2 with results25% success
Data Visualizations
Phase Distribution
3Total
Not Applicable (1)
P 1 (1)
P 3 (1)
Trial Status
Terminated3
Completed1
Not Yet Recruiting1
Trial Success Rate
25.0%
Benchmark: 86.5%
Based on 1 completed trials
Clinical Trials (5)
Showing 5 of 5 trials
NCT07354724Phase 1Not Yet RecruitingPrimary
A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease
NCT05431127Not ApplicableCompletedPrimary
High Dose Inspiratory Muscle Training in LOPD
NCT03347253TerminatedPrimary
STRIDE Study - A Study in Subjects With LOPD Who Are Currently Being Treated With ERT
NCT02191917TerminatedPrimary
A Study of Respiratory Muscle Strength in Patients With Late-onset Pompe Disease (LOPD)
NCT01924845Phase 3TerminatedPrimary
BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study)
Showing all 5 trials