Effects of Coenzyme Q10 on Charcot-Marie-Tooth Disease

NCT ID: NCT00541164

Last Updated: 2013-07-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

23 participants

Study Classification

INTERVENTIONAL

Study Start Date

2007-09-30

Study Completion Date

2013-01-31

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Charcot Marie Tooth Disease

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

SUPPORTIVE_CARE

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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1

300 mg CoQ10 chewable wafer twice a day

Group Type EXPERIMENTAL

Coenzyme Q10

Intervention Type DRUG

300 mg CoQ10 twice a day for 48 weeks

2

Chewable placebo wafer twice a day for 24 weeks with crossover to 300mg CoQ10 twice a day for weeks 24-48.

Group Type PLACEBO_COMPARATOR

Coenzyme Q10

Intervention Type DIETARY_SUPPLEMENT

300mg CoQ10 twice a day for 24 weeks beginning at week 24 of the study

Interventions

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Coenzyme Q10

300 mg CoQ10 twice a day for 48 weeks

Intervention Type DRUG

Coenzyme Q10

300mg CoQ10 twice a day for 24 weeks beginning at week 24 of the study

Intervention Type DIETARY_SUPPLEMENT

Other Intervention Names

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CoQ10 Ubiquinone CoQ10 Ubiquinone

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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United States Department of Defense

FED

Sponsor Role collaborator

Memorial Medical Center

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Sharon Plank, MD

Role: PRINCIPAL_INVESTIGATOR

John P. Murtha Neuroscience and Pain Institute

Locations

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John P Murtha Neuroscience and Pain Institute

Johnstown, Pennsylvania, United States

Site Status

Countries

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United States

References

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

Reference Type BACKGROUND

Chaudhuri A, Behan PO. Fatigue in neurological disorders. Lancet. 2004 Mar 20;363(9413):978-88. doi: 10.1016/S0140-6736(04)15794-2.

Reference Type BACKGROUND
PMID: 15043967 (View on PubMed)

Beal MF. Coenzyme Q10 as a possible treatment for neurodegenerative diseases. Free Radic Res. 2002 Apr;36(4):455-60. doi: 10.1080/10715760290021315.

Reference Type BACKGROUND
PMID: 12069110 (View on PubMed)

Shults CW. Coenzyme Q10 in neurodegenerative diseases. Curr Med Chem. 2003 Oct;10(19):1917-21. doi: 10.2174/0929867033456882.

Reference Type BACKGROUND
PMID: 12871093 (View on PubMed)

Bhagavan HN, Chopra RK. Coenzyme Q10: absorption, tissue uptake, metabolism and pharmacokinetics. Free Radic Res. 2006 May;40(5):445-53. doi: 10.1080/10715760600617843.

Reference Type BACKGROUND
PMID: 16551570 (View on PubMed)

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. doi: 10.1001/archneur.59.10.1541.

Reference Type BACKGROUND
PMID: 12374491 (View on PubMed)

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. doi: 10.1212/wnl.57.3.397.

Reference Type BACKGROUND
PMID: 11502903 (View on PubMed)

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. No abstract available.

Reference Type BACKGROUND
PMID: 12017500 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 8820103 (View on PubMed)

Other Identifiers

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05-19

Identifier Type: -

Identifier Source: org_study_id

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