Scheie Syndrome
6
0
0
5
Key Insights
Highlights
Success Rate
83% trial completion
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
16.7%
1 terminated out of 6 trials
83.3%
-3.2% vs benchmark
50%
3 trials in Phase 3/4
80%
4 of 5 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 5 completed trials
Clinical Trials (6)
A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old
A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I