Pearson Syndrome
6
2
2
2
Key Insights
Highlights
Success Rate
67% trial completion
Clinical Risk Assessment
Based on trial outcomes
High Risk
Score: 72/100
16.7%
1 terminated out of 6 trials
66.7%
-19.8% vs benchmark
0%
0 trials in Phase 3/4
0%
0 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 (6)
Natural History of Pearson Syndrome
Evaluate the Safety and Therapeutic Effects of a Single Intravenous Infusion (IV) of Autologous CD34+ Cells Enriched With Allogenic Placenta-derived Mitochondria in Patients With a Diagnosis of Pearson Syndrome (PS)
Global Registry and Natural History Study for Mitochondrial Disorders
SLSMDS Natural History Study
A Study to Evaluate the Safety and Therapeutic Effects of Transplantation of MNV-BM-BLD in Pediatric Patients With Pearson Syndrome
Phase 2 Study of EPI-743 in Children With Pearson Syndrome