Table 1. Selected clinical trials on drugs affecting mitochondrial function.

Trial statuses were confirmed as of 26 January 2016. AMP, adenosine-5′-monophosphate.

Class	Compound name (manufacturer)	Targeting site	Disease	Phase	Outcome	ClinicalTrials.gov identifier
Antioxidant	MitoQ	ROS scavenger	Chronic kidney disease (CKD)	4*	Ongoing	NCT02364648
	Resveratrol	SIRT1 activator	CKD	3	Completed	NCT02433925
	EPI-743 (Edison Pharmaceuticals)	Coenzyme Q10 (CoQ10)–based	Rett syndrome	2	Completed	NCT01822249
	Idebenone (Santhera Pharmaceuticals)	CoQ10-based	Duchenne muscular dystrophy	3	Positive (74)	NCT01027884
	Edaravone	Nonspecific ROS scavenger	Acute myocardial infarction (AMI)	4	Positive (75)	NCT00265239
	Edaravone	Nonspecific ROS scavenger	Amyotrophic lateral sclerosis	3	Negative (76)	NCT00330681
	CoQ10	ROS scavenger	Parkinson’s disease	3	Negative (77)	NCT00740714
	SS31 (bendavia) (Stealth BioTherapeutics Inc.)	Unknown	AMI	2	Negative†	NCT01572909
Metabolism modulator	Acipimox	Niacin derivative	Obesity	2	Negative (78)	NCT01488409
	Metformin		Type 1 diabetes	4	Ongoing	NCT01813929
	Metformin	Metabolism/unknown	Nonalcoholic fatty liver disease	4	Terminated	NCT00736385
	Creatine	Metabolism	Huntington’s disease	3	Terminated‡	NCT00712426
	Acadesine (Merck Sharp & Dohme Corp.)	AMP-activated protein kinase, nonspecific	Myocardial infarction	3	Negative (79)	NCT00872001
Mitochondrial permeability transition pore (mPTP) inhibitor	Cyclosporine A	Cyclophilin D	Acute kidney injury	2	Ongoing	NCT02397213
	Cyclosporine A	Cyclophilin D	AMI	3	Negative (46)	NCT01502774
	Dimebon (Pfizer)	Unknown	Alzheimer’s disease	3	Negative (80)	NCT00838110
	TRO40303 (Trophos)	Mitochondrial translocator protein	AMI	2	Negative (81)	NCT01374321

^*The compound entered a phase 4 trial on the basis that it is used as a nutritional supplement.

^‡Terminated as unlikely to be effective.

^†Results reported at the American College of Cardiology 2015 Scientific Sessions.