Leigh Syndrome
11
3
3
3
Key Insights
Highlights
Success Rate
75% trial completion
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 37/100
9.1%
1 terminated out of 11 trials
75.0%
-11.5% vs benchmark
0%
0 trials in Phase 3/4
67%
2 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 (11)
Sirolimus for Leigh Syndrome
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
A Dose-ranging Study of TTI-0102 in Adults and Children With Leigh Syndrome Spectrum (LSS)
Global Registry and Natural History Study for Mitochondrial Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
The International Registry for Leigh Syndrome
A Study of the Natural Course of SURF1 Deficiency
ABI-009 (Nab-sirolimus) in Patients With Genetically-confirmed Leigh or Leigh-like Syndrome
A Dose-escalating Clinical Trial With KH176
Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome