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

Sponsor:

Collaborator:

United States Department of Defense

Information provided by (Responsible Party):

Memorial Medical Center

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
Charcot Marie Tooth Disease	Drug: Coenzyme Q10 Dietary Supplement: Coenzyme Q10	Phase 1 Phase 2

Detailed Description:

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 Design

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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

Resource links provided by the National Library of Medicine

MedlinePlus Genetics related topics: Hereditary neuropathy with liability to pressure palsies Charcot-Marie-Tooth disease

MedlinePlus related topics: Charcot-Marie-Tooth Disease Tooth Disorders

Drug Information available for: Ubidecarenone

Genetic and Rare Diseases Information Center resources: Charcot-Marie-Tooth Disease Hereditary Neuropathy With Liability to Pressure Palsies Roussy Levy Syndrome

U.S. FDA Resources

Arms and Interventions

Arm	Intervention/treatment
Experimental: 1 300 mg CoQ10 chewable wafer twice a day	Drug: Coenzyme Q10 300 mg CoQ10 twice a day for 48 weeks Other Names: CoQ10 Ubiquinone
Placebo Comparator: 2 Chewable placebo wafer twice a day for 24 weeks with crossover to 300mg CoQ10 twice a day for weeks 24-48.	Dietary Supplement: Coenzyme Q10 300mg CoQ10 twice a day for 24 weeks beginning at week 24 of the study Other Names: CoQ10 Ubiquinone

Outcome Measures

Primary Outcome Measures :

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 ]

Secondary Outcome Measures :

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 ]

Eligibility Criteria

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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

Criteria

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
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

Contacts and Locations

Information from the National Library of Medicine

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

Locations

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United States, Pennsylvania
John P Murtha Neuroscience and Pain Institute
Johnstown, Pennsylvania, United States, 15904

Sponsors and Collaborators

Memorial Medical Center

United States Department of Defense

Investigators

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Principal Investigator:	Sharon Plank, MD	John P. Murtha Neuroscience and Pain Institute

More Information

Publications:

Hendler SS, Rorvik D, eds. PDR for Nutritional Supplements. Montvale, NJ: Thomson PDR; 2001:105-106.

Chaudhuri A, Behan PO. Fatigue in neurological disorders. Lancet. 2004 Mar 20;363(9413):978-88. Review.

Beal MF. Coenzyme Q10 as a possible treatment for neurodegenerative diseases. Free Radic Res. 2002 Apr;36(4):455-60. Review.

Shults CW. Coenzyme Q10 in neurodegenerative diseases. Curr Med Chem. 2003 Oct;10(19):1917-21. Review.

Bhagavan HN, Chopra RK. Coenzyme Q10: absorption, tissue uptake, metabolism and pharmacokinetics. Free Radic Res. 2006 May;40(5):445-53. Review.

Shults CW, Oakes D, Kieburtz K, Beal MF, Haas R, Plumb S, Juncos JL, Nutt J, Shoulson I, Carter J, Kompoliti K, Perlmutter JS, Reich S, Stern M, Watts RL, Kurlan R, Molho E, Harrison M, Lew M; Parkinson Study Group. Effects of coenzyme Q10 in early Parkinson disease: evidence of slowing of the functional decline. Arch Neurol. 2002 Oct;59(10):1541-50.

Huntington Study Group. A randomized, placebo-controlled trial of coenzyme Q10 and remacemide in Huntington's disease. Neurology. 2001 Aug 14;57(3):397-404.

Jones K, Hughes K, Mischley L, McKenna DJ. Coenzyme Q-10: efficacy, safety, and use. Altern Ther Health Med. 2002 May-Jun;8(3):42-55; quiz 56, 138. Review.

Lagendijk J, Ubbink JB, Vermaak WJ. Measurement of the ratio between the reduced and oxidized forms of coenzyme Q10 in human plasma as a possible marker of oxidative stress. J Lipid Res. 1996 Jan;37(1):67-75.

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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

Keywords provided by Memorial Medical Center:


CMT Charcot-Marie Tooth CoQ10 Coenzyme Q10

Additional relevant MeSH terms:

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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

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