Glycogen Storage Disease Type II (GSD-II)
9
1
1
6
Key Insights
Highlights
Success Rate
86% trial completion
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
11.1%
1 terminated out of 9 trials
85.7%
-0.8% vs benchmark
67%
6 trials in Phase 3/4
83%
5 of 6 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 6 completed trials
Clinical Trials (9)
Pompe Pregnancy Sub-Registry
Growth and Development Study of Alglucosidase Alfa
Immune Tolerance Induction Study
A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease
High Dose or High Dose Frequency Study of Alglucosidase Alfa
Late-Onset Treatment Study Extension Protocol
Alglucosidase Alfa Temporary Access Program
A Study of rhGAA in Patients With Late-Onset Pompe Disease
Safety and Effectiveness Study of rhGAA in Patients With Advanced Late-Onset Pompe Disease Receiving Respiratory Support