Effects of Coenzyme Q10 on Charcot-Marie-Tooth Disease
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ClinicalTrials.gov Identifier: NCT00541164 |
Recruitment Status :
Completed
First Posted : October 10, 2007
Last Update Posted : July 15, 2013
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Condition or disease | Intervention/treatment | Phase |
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Charcot Marie Tooth Disease | Drug: Coenzyme Q10 Dietary Supplement: Coenzyme Q10 | Phase 1 Phase 2 |
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.
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 23 participants |
Allocation: | Randomized |
Intervention Model: | Crossover Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Supportive Care |
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 |
Study Start Date : | September 2007 |
Actual Primary Completion Date : | January 2013 |
Actual Study Completion Date : | January 2013 |

Arm | Intervention/treatment |
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Experimental: 1
300 mg CoQ10 chewable wafer twice a day
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Drug: Coenzyme Q10
300 mg CoQ10 twice a day for 48 weeks
Other Names:
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Placebo Comparator: 2
Chewable placebo wafer twice a day for 24 weeks with crossover to 300mg CoQ10 twice a day for weeks 24-48.
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Dietary Supplement: Coenzyme Q10
300mg CoQ10 twice a day for 24 weeks beginning at week 24 of the study
Other Names:
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- Changes in weakness, fatigue and pain in persons with Charcot-Marie-Tooth disease after supplementation with 600 mgs a day of Coenzyme Q10. [ Time Frame: 60 weeks ]
- Improvements in quality of life in subjects with CMT before and after CoQ10 supplementation. [ Time Frame: 60 weeks ]
- Measure blood serum levels of the oxidized and reduced forms of CoQ10. [ Time Frame: 60 weeks ]
- Measure liver function tests [ Time Frame: visits 1, 6, 12 ]

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Ages Eligible for Study: | 18 Years to 75 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Subjects must have a diagnosis of CMT, confirmed by review of medical records by the study physician
- Subjects can be of either gender
- Subjects must be between the ages of 18 and 75
- Subjects must be able to complete all assessments at the designated time intervals
- Subjects must review and sign the informed consent statement according to Conemaugh Memorial Medical Center's (CMMC) Institutional Review Board (IRB) guidelines
- Subjects must receive approval from their primary care physician (PCP) to enroll in the study
- Regarding weakness, fatigue, and pain, subjects must experience at least two of the three symptoms on most days over the past month
- Regarding weakness, fatigue, and pain, subjects must report experiencing maximum levels of >/= 3.0 centimeters (cm) on the 10 cm visual analog scale (VAS) for any two of the three symptoms over the past month
- Female subjects must be willing to practice stable birth control during involvement in the study
- Subjects must agree to be randomized
Exclusion Criteria:
- Subjects having another general medical condition, which might confound the assessment of weakness, fatigue, and pain due to CMT
- Subjects taking warfarin or Coumadin
- Subjects who are pregnant, verified by a urine pregnancy test*
- Subjects having a cognitive impairment scoring < 20 on the Mini-Mental State Exam
- Subjects who are currently using CoQ10 supplementation or have used it in the past 6 months
- Subjects with a history of chronic liver disease or other condition causing malabsorption
- Drug intake that could modify lipid absorption (such as statins)
- Subjects who consume >3 alcoholic drinks per day on more than one occasion per month
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Subjects with abnormal liver function tests as defined through a Hepatic -Function Panel or a Liver Function Panel
- Women of childbearing age who have had at least one menstrual cycle within the past 12 months and who have not undergone a sterilization procedure will undergo a urine pregnancy test at visits 1-10 regardless of group assignment in order to maintain the single blind. The urine samples will be processed at CMMC's lab

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00541164
United States, Pennsylvania | |
John P Murtha Neuroscience and Pain Institute | |
Johnstown, Pennsylvania, United States, 15904 |
Principal Investigator: | Sharon Plank, MD | John P. Murtha Neuroscience and Pain Institute |
Responsible Party: | Memorial Medical Center |
ClinicalTrials.gov Identifier: | NCT00541164 |
Other Study ID Numbers: |
05-19 |
First Posted: | October 10, 2007 Key Record Dates |
Last Update Posted: | July 15, 2013 |
Last Verified: | June 2010 |
CMT Charcot-Marie Tooth CoQ10 Coenzyme Q10 |
Tooth Diseases Charcot-Marie-Tooth Disease Nerve Compression Syndromes Hereditary Sensory and Motor Neuropathy Stomatognathic Diseases Nervous System Malformations Nervous System Diseases Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Polyneuropathies |
Peripheral Nervous System Diseases Neuromuscular Diseases Congenital Abnormalities Genetic Diseases, Inborn Ubiquinone Coenzyme Q10 Micronutrients Physiological Effects of Drugs Vitamins |