Scheie Syndrome
6
0
0
5
Key Insights
Highlights
Success Rate
83% trial completion
Treatment Research
2 treatments under study, led by Aldurazyme Recombinant Human Alphaliduronidase
Research Leaders
11 sponsors active, led by BioMarin/Genzyme LLC
Related Conditions
Often studied with 7 related conditions
Clinical Risk Assessment
Based on trial outcomes
Low Risk
Score: 27/100
16.7%
1 terminated out of 6 trials
83.3%
-3.3% vs benchmark
50%
3 trials in Phase 3/4
80%
4 of 5 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 5 completed trials
Clinical Trials (6)
A Study of Intrathecal Enzyme Therapy for Cognitive Decline in MPS I
A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease
A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old
Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
Extension Study of Intrathecal Enzyme Replacement Therapy for MPS I
Research Network
Common Treatments
- Aldurazyme Recombinant Human Alphaliduronidase3
- Laronidase1
Active Sponsors
- BioMarin/Genzyme LLC4
- Genzyme, a Sanofi Company4
- The Ryan Foundation2
- Patricia I. Dickson, M.D.2
- University of California, Los Angeles1
Top Investigators
Corp. GCP Compliance - Clinical Affairs, Genzyme Japan K.K.
Los Angeles Biomedical Institute at Harbor-UCLA
Related Conditions
- Mucopolysaccharidosis I6
- Hurlerscheie Syndrome6
- Lysosomal Storage Disease2
- Hurlers Syndrome2
- Hurler Syndrome2