Tier 3 — preclinical

A randomized trial of coenzyme Q10 in mitochondrial disorders

Elisa I Glover, Joan Martin, Amy Maher, Rebecca E Thornhill, Gerald R Moran, Mark A Tarnopolsky
Muscle & Nerve 2010 42(5):739-748

Bibliography

PubMed
PMID 20886510
Funding
Not reported in the accessible PubMed abstract.
Competing interests
Not reported in the accessible PubMed abstract.

Study snapshot

DesignRandomized, double-blind crossover trial
ModelHuman mitochondrial cytopathy
Sample30 participants
InterventionCoQ10 1,200 mg/day versus placebo
Duration60 days per treatment period
EndpointsBlood lactate; Oxidative-stress markers; Activities of daily living; Quality of life; Handgrip strength; Exercise physiology; Brain metabolites

What the study showed, in plain terms

High-dose CoQ10 modestly attenuated the rise in lactate after exercise and changed a few exercise and brain-metabolic measures, but did not improve strength, resting lactate or most clinically relevant functional outcomes.

Key findings

The trial found minor physiologic effects rather than broad clinical improvement. It is direct evidence against assuming that mitochondrial target engagement automatically improves everyday function.

What this study can and cannot tell us

Small sample, heterogeneous mitochondrial disorders, short treatment periods and many surrogate outcomes.

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