MPS II
7
2
3
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)
RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)
PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)
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