MPS IV A
6
1
1
2
Key Insights
Highlights
Success Rate
50% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 65/100
33.3%
2 terminated out of 6 trials
50.0%
-36.5% vs benchmark
17%
1 trials in Phase 3/4
150%
3 of 2 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 2 completed trials
Clinical Trials (6)
Cardiac Structure and Function in MPS
Evaluation of Losartan on Cardiovascular Disease in Patients With Mucopolysaccharidoses IV A and VI
A Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
A Clinical Assessment Study of Subjects With Mucopolysaccharidosis IVA (Morquio Syndrome)
A Double-Blind Study to Evaluate the Efficacy and Safety of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)
A Study to Evaluate the Safety, Tolerability and Efficacy of BMN 110 in Subjects With Mucopolysaccharidosis IVA