MPS II
7
2
2
2
Key Insights
Highlights
Success Rate
50% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 55/100
28.6%
2 terminated out of 7 trials
50.0%
-36.5% vs benchmark
43%
3 trials in Phase 3/4
100%
2 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 (7)
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)
Longitudinal Study of Neurodegenerative Disorders
An Extension Study to Assess the Long-term Safety and Efficacy of Hunterase (Idursulfase Beta)
MPS II Immunophenotyping
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy