Mitochondrial Disease
22
3
4
13
Key Insights
Highlights
Success Rate
93% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 50/100
4.5%
1 terminated out of 22 trials
92.9%
+6.4% vs benchmark
0%
0 trials in Phase 3/4
23%
3 of 13 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 13 completed trials
Clinical Trials (22)
Acute Infection in Mitochondrial Disease: Metabolism, Infection and Immunity
Vatiquinone Expanded Access Protocol
Validation of Nanosensor Oxygen Measurement
Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels
Mitochondrial Dysfunction in Autism Spectrum Disorder
Tissue Sample Study for Mitochondrial Disorders
Pilot Study of the Efficacy of Nicotinamide (Vitamin B3) in Leber's Hereditary Optic Neuropathy
Expanded Access Program for R007 (Probucol) Tablets in Adults With Mitochondrial Disease and Chronic Kidney Disease
Magnetic Resonance Imaging (MRI) Muscle Phenotyping in Mitochondrial Disease
Mitochondria Inborn Errors of Metabolism and ANT Defects in Mitochondria Diseases
Drug-drug Interaction Study of KL1333 in Healthy Subjects
A Dose-escalating Clinical Trial With KH176
Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan
EPI-743 for Metabolism or Mitochondrial Disorders
GDF-15 as a Biomarker for Mitochondrial Disease
Calf Muscle Strength in Mitochondrial Diseases
Energy Requirements in Mitochondrial Disease
Survey on Supplement Use in Mitochondrial Disease
Mechanisms of Mitochondrial Defects in Gulf War Syndrome
Anesthetic Effects in Mitochondrial Disease