Pelizaeus-Merzbacher Disease
8
5
5
3
Key Insights
Highlights
Success Rate
100% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
0.0%
0 terminated out of 8 trials
100.0%
+13.5% vs benchmark
0%
0 trials in Phase 3/4
33%
1 of 3 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 3 completed trials
Clinical Trials (8)
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
Longitudinal Study of Neurodegenerative Disorders
Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies
The Myelin Disorders Biorepository Project
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)
Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects