Effects of Coenzyme Q10 on Charcot-Marie-Tooth Disease - Full Text View

Sponsors and Collaborators:	Memorial Medical Center Department of Defense
Information provided by:	Memorial Medical Center
ClinicalTrials.gov Identifier:	NCT00541164

Purpose

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	Intervention	Phase
Charcot Marie Tooth Disease	Dietary Supplement: Coenzyme Q10	Phase I Phase II

Genetics Home Reference related topics: Charcot-Marie-Tooth disease familial encephalopathy with neuroserpin inclusion bodies hereditary neuropathy with liability to pressure palsies

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

Drug Information available for: Coenzyme Q10

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Supportive Care, Randomized, Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Placebo Control, Crossover Assignment, Efficacy Study
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

Further study details as provided by Memorial Medical Center:

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 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Improvements in quality of life in subjects with CMT before and after CoQ10 supplementation. [ Time Frame: 60 weeks ] [ Designated as safety issue: No ]
Measure blood serum levels of the oxidized and reduced forms of CoQ10. [ Time Frame: 60 weeks ] [ Designated as safety issue: No ]
Measure liver function tests [ Time Frame: visits 1, 6, 12 ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	46
Study Start Date:	September 2007
Estimated Study Completion Date:	September 2012
Estimated Primary Completion Date:	June 2012 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
1: Experimental 300 mg CoQ10 chewable wafer twice a day	Dietary Supplement: Coenzyme Q10 300 mg CoQ10 twice a day for 48 weeks
2: Placebo Comparator 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

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.

Eligibility

Ages Eligible for Study:	18 Years to 75 Years
Genders Eligible for Study:	Both
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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00541164

Contacts

Contact: Janet Goodard, RN, BC BSN	814-269-5288	jgoodar@conemaugh.org
Contact: Sharon Plank, MD	814-269-5204	splank@conemaugh.org

Locations

United States, Pennsylvania
John P Murtha Neuroscience and Pain Institute	Recruiting
Johnstown, Pennsylvania, United States, 15904
Principal Investigator: Sharon Plank, MD
Sub-Investigator: Lisa Pasierb, PhD

Sponsors and Collaborators

Memorial Medical Center

Department of Defense

Investigators

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. No abstract available.

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.

Responsible Party:	Memorial Medical Center ( Sharon Plank, MD, )
Study ID Numbers:	05-19
Study First Received:	October 9, 2007
Last Updated:	February 5, 2008
ClinicalTrials.gov Identifier:	NCT00541164
Health Authority:	United States: Food and Drug Administration

Keywords provided by Memorial Medical Center:

CMT
Charcot-Marie Tooth
CoQ10
Coenzyme Q10

Study placed in the following topic categories:

Tooth Diseases
Nervous System Malformations
Roussy Levy hereditary areflexic dystasia
Charcot-Marie-Tooth Disease
Polyneuropathies
Neurodegenerative Diseases
Coenzyme Q10
Nerve Compression Syndromes
Tomaculous neuropathy

Heredodegenerative Disorders, Nervous System
Neuromuscular Diseases
Genetic Diseases, Inborn
Peripheral Nervous System Diseases
Ubiquinone
Hereditary Motor and Sensory Neuropathies
Stomatognathic Diseases
Congenital Abnormalities
Charcot Marie Tooth disease

Additional relevant MeSH terms:

Vitamins
Growth Substances
Physiological Effects of Drugs

Nervous System Diseases
Micronutrients
Pharmacologic Actions

ClinicalTrials.gov processed this record on January 14, 2009