Mucopolysaccharidosis II
36
3
12
12
Key Insights
Highlights
Success Rate
67% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 72/100
16.7%
6 terminated out of 36 trials
66.7%
-19.9% vs benchmark
8%
3 trials in Phase 3/4
33%
4 of 12 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 12 completed trials
Clinical Trials (36)
A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)
An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Long-term Follow-Up for RGX-121
Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
Registry of Patients Diagnosed With Lysosomal Storage Diseases
Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type II
MPS (RaDiCo Cohort) (RaDiCo-MPS)
Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI
An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects
Baby Detect : Genomic Newborn Screening
A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome
A Study of JR-141 in Patients With Mucopolysaccharidosis II (BR21)
A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ
MPS II Immunophenotyping
A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome