Table 3 Examples of the clinical trials currently open or completed for mitochondrial diseases
TreatmentDiseaseTrial numberDesignTarget of interventionOutcome
Currently open
 EPI-743Metabolism or mitochondrial disordersNCT01642056Randomized, double-blindROSOngoing
 BezafibrateMitochondrial myopathyNCT02398201Open-labelMitochondrial biogenesisOngoing
 RTA 408Mitochondrial myopathyNCT02255422Randomized, double- blindROS/NRF2Ongoing
 KH176MELASNCT02544217Randomized, double-blindROSOngoing
 scAAV2-ND4LHONNCT02161380Open-labelND4Ongoing
Completed
 KetonesMELASNCT01252979Open-labelHeteroplasmyN/A
l-ArginineMELASNCT01603446Open-labelNitric oxideImprovement in aerobic capacity and muscle metabolism
 IdebenoneLHONNCT00747487Randomized, double-blindROSNo recovery in visual acuity, but improvements in secondary end points (e.g. changes in visual acuity of the best eye at baseline)
 Coenzyme Q10Mitochondrial diseaseNCT00432744Randomized, double-blindROSN/A
 MTP-131Mitochondrial myopathyNCT02367014Randomized, double-blindCardiolipinN/A