Condition
Acid Maltase Deficiency
Total Trials
5
Recruiting
0
Active
1
Completed
2
Success Rate
66.7%-20% vs avg
Key Insights
Highlights
Success Rate
67% trial completion
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 39/100
Termination Rate
20.0%
1 terminated out of 5 trials
Success Rate
66.7%
-19.8% vs benchmark
Late-Stage Pipeline
40%
2 trials in Phase 3/4
Results Transparency
50%
1 of 2 completed with results
Key Signals
1 with results67% success
Data Visualizations
Phase Distribution
5Total
Not Applicable (1)
P 1 (2)
P 4 (2)
Trial Status
Completed2
Not Yet Recruiting1
Terminated1
Active Not Recruiting1
Trial Success Rate
66.7%
Benchmark: 86.5%
Based on 2 completed trials
Clinical Trials (5)
Showing 5 of 5 trials
NCT04093349Phase 1Active Not Recruiting
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)
NCT06575829Phase 4Not Yet Recruiting
Treatment Frequency Reduction in Pompe Disease
NCT03893240Not ApplicableCompleted
Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease
NCT01898364Phase 1Completed
Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.
NCT01597596Phase 4Terminated
A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease
Showing all 5 trials