Condition
Mucopolysaccharidosis Type I (MPS I)
Total Trials
3
Recruiting
0
Active
0
Completed
2
Success Rate
100.0%+13% vs avg
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
Termination Rate
0.0%
0 terminated out of 3 trials
Success Rate
100.0%
+13.5% vs benchmark
Late-Stage Pipeline
0%
0 trials in Phase 3/4
Results Transparency
0%
0 of 2 completed with results
Key Signals
100% success
Data Visualizations
Phase Distribution
1Total
P 1 (1)
Trial Status
Completed2
Suspended1
Trial Success Rate
100.0%
Benchmark: 86.5%
Based on 2 completed trials
Clinical Trials (3)
Showing 3 of 3 trials
NCT03580083Phase 1SuspendedPrimary
RGX-111 Gene Therapy in Patients With MPS I
NCT05073783Completed
A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting
NCT01938014CompletedPrimary
Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children
Showing all 3 trials