Mucopolysaccharidosis I
32
2
4
20
Key Insights
Highlights
Success Rate
77% trial completion
Published Results
12 trials with published results (38%)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 49/100
18.8%
6 terminated out of 32 trials
76.9%
-9.6% vs benchmark
22%
7 trials in Phase 3/4
60%
12 of 20 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 20 completed trials
Clinical Trials (32)
Long Term Follow-Up for RGX-111
Registry of Patients Diagnosed With Lysosomal Storage Diseases
MPS (RaDiCo Cohort) (RaDiCo-MPS)
Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI
China Post-marketing Surveillance (PMS) Study of Aldurazyme®
An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I)
Baby Detect : Genomic Newborn Screening
A Study of JR-171 in Patients With Mucopolysaccharidosis I
Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products
Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
A Study of the Effect of Aldurazyme® (Laronidase) Treatment on Lactation in Female Patients With Mucopolysaccharidosis I (MPS I) and Their Breastfed Infants
Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I
Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I
Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS I
Safety and Dose Ranging Study of Insulin Receptor MAb-IDUA Fusion Protein in Patients With MPS I
Human Placental-Derived Stem Cell Transplantation
Stem Cell Transplant w/Laronidase for Hurler
Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181
Clinical Trial of Growth Hormone in MPS I, II, and VI
Stem Cell Transplantation for Hurler