Mitochondrial Diseases
75
18
21
36
Key Insights
Highlights
Success Rate
90% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
5.3%
4 terminated out of 75 trials
90.0%
+3.4% vs benchmark
5%
4 trials in Phase 3/4
22%
8 of 36 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.6%
Based on 36 completed trials
Clinical Trials (75)
The Precision Mito- Mito Map COMPASS Study
Metabolic and Functional Study of γδ T Cells in Critically Ill Patients
Open Label Extension to Assess Long Term Safety and Efficacy of KL1333 in Patients With Primary Mitochondrial Disease
Enhancement of Quality of Work And Life
Global Registry and Natural History Study for Mitochondrial Disorders
KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
Longitudinal Study of Mitochondrial Hepatopathies
Genetic Diagnosis in Inborn Errors of Metabolism
A Multi-Dimensional Model of cAre and transItion for Patients With cOmplex RAre Diseases
Transgenerational Metabolic-Immune Biomarkers of Neurological and Neurodevelopmental Disorders
Digital Health Technology for People With Mitochondrial Disease
The Natural History of Mitochondrial Diseases
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
Exercise Training Effects on Muscle Function in Adults With Mitochondrial Myopathy
Mitochondrial Substrate Utilization in the Diabetic Human Heart
Validation of Oxygen Nanosensor in Mitochondrial Myopathy
North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)
CrCest Study in Primary Mitochondrial Disease
Reference Values of Intraepidermal Nerve Fiber Density in Children and Small Fiber Neuropathy in Neurodevelopmental Disorders
Applying pGz in Mitochondrial Disease