Charcot Marie Tooth Disease Clinical Trial
Official title:
Effects of Coenzyme Q10 (CoQ10) on Subjects With Charcot-Marie-Tooth Disease (CMT):A Double Blind, Randomized, Controlled Trial With an Open Label Follow-up Study
The object of this research is to test the effectiveness of Coenzyme Q10 (CoQ10) on symptoms of weakness, fatigue, and pain in persons with Charcot-Marie-Tooth disease (CMT).In this study we also intend to examine the impact of daily supplementation on overall quality of life.We are also interested in identifying any differences in serum ratios of CoQ10 in the oxidized and reduced forms.
CoQ10 is an integral part of the electron transport chain in the mitochondria, or the energy
production centers of cells. Within recent years, there has been expanding interest in the
potential benefits of CoQ10 supplementation on a variety of neuromuscular diseases, some of
which involve mitochondrial dysfunction, such as CMT. Daily supplementation may have
cytoprotective and neuroprotective properties, which may improve symptoms of weakness,
fatigue, and pain, as well as increase quality of life (QOL) among persons with CMT.
With regards to within group comparisons we hypothesize that daily supplementation of CoQ10
taken as a 300 milligram wafer twice a day for 3 months will produce a statistically
significant reduction in weakness, fatigue, and pain, along with a significant improvement in
QOL as indicated from scores in both standardized physiological and scale measures.
The addition of serum level analysis will help to contextualize clinical results. We
hypothesize the ratios of the oxidized and reduced forms of CoQ10 will be modified upon
supplementation.
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