Effects of Coenzyme Q10 on Charcot-Marie-Tooth Disease

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00541164
• Other study ID #: 05-19
• Status: Completed
• Phase: Phase 1/Phase 2
• First received: October 9, 2007
• Last updated: July 11, 2013
• Start date: September 2007
• Est. completion date: January 2013

Study information

• Verified date: June 2010
• Source: Memorial Medical Center
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 23
• Est. completion date: January 2013
• Est. primary completion date: January 2013
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

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

Related Conditions & MeSH terms

• Charcot Marie Tooth Disease
• Charcot-Marie-Tooth Disease
• Hereditary Sensory and Motor Neuropathy
• Nerve Compression Syndromes
• Tooth Diseases

Intervention

Drug:
• Coenzyme Q10
300 mg CoQ10 twice a day for 48 weeks

Dietary Supplement:
• Coenzyme Q10
300mg CoQ10 twice a day for 24 weeks beginning at week 24 of the study

Locations

Country	Name	City	State
United States	John P Murtha Neuroscience and Pain Institute	Johnstown	Pennsylvania

Sponsors (2)

Lead Sponsor	Collaborator
Memorial Medical Center	United States Department of Defense

Country where clinical trial is conducted

United States, 

References & Publications (9)

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

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

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

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

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. — View Citation

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. — View Citation

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. — View Citation

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. — View Citation

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Changes in weakness, fatigue and pain in persons with Charcot-Marie-Tooth disease after supplementation with 600 mgs a day of Coenzyme Q10.		60 weeks
Secondary	Improvements in quality of life in subjects with CMT before and after CoQ10 supplementation.		60 weeks
Secondary	Measure blood serum levels of the oxidized and reduced forms of CoQ10.		60 weeks
Secondary	Measure liver function tests		visits 1, 6, 12