Condition
Pompe disease / glycogenosis type II / acid maltase deficiency
Total Trials
2
Recruiting
0
Active
0
Completed
0
Key Insights
Highlights
Treatment Research
4 treatments under study, led by Alglucosidase Alfa
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 30/100
Termination Rate
0.0%
0 terminated out of 2 trials
Late-Stage Pipeline
100%
2 trials in Phase 3/4
Results Transparency
0%
0 of 0 completed with results
Key Signals
Data Visualizations
Phase Distribution
2Total
Phase 4 (2)
Trial Status
Not Yet Recruiting2
Clinical Trials (2)
Showing 2 of 2 trials
SYF3c654df2a748407abb3d7087d1b70a17Phase 4Not Yet Recruiting
Pompe disease; new Outcomes measures, Treatment Effect and Novel Targeted Investigations for Advancing Long-term follow-up (POTENTIAL).
SYFf46bd0c6e82b470bb755a2eaa2199d98Phase 4Not Yet Recruiting
Effects and health economic aspects of enzyme therapy in children and adults with Pompe disease
Showing all 2 trials
Research Network
Common Treatments
- Alglucosidase Alfa2
- Miglustat1
- Avalglucosidase Alfa1
- Cipaglucosidase Alfa1
Active Sponsors
- Erasmus Universitair Medisch Centrum Rotterdam (Erasmus MC)2