Clinical trial NCT00541164: Effects of Coenzyme Q10 on Charcot-Marie-Tooth Disease
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 type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Charcot Marie Tooth Disease
Keywords: CMT
Charcot-Marie Tooth
CoQ10
Coenzyme Q10
Lead sponsor: Memorial Medical Center
Sponsor class: OTHER
Collaborators: name: United States Department of Defense
First posted: 2007-10-10
Enrollment: 23
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: SUPPORTIVE_CARE

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.

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 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

Primary outcomes:
measure: Changes in weakness, fatigue and pain in persons with Charcot-Marie-Tooth disease after supplementation with 600 mgs a day of Coenzyme Q10.
timeFrame: 60 weeks
Secondary outcomes:
measure: Improvements in quality of life in subjects with CMT before and after CoQ10 supplementation.
timeFrame: 60 weeks
measure: Measure blood serum levels of the oxidized and reduced forms of CoQ10.
timeFrame: 60 weeks
measure: Measure liver function tests
timeFrame: visits 1, 6, 12
Other outcomes:
Locations:
city: Johnstown
country: United States
facility: John P Murtha Neuroscience and Pain Institute
state: Pennsylvania
Study officials:
affiliation: John P. Murtha Neuroscience and Pain Institute
name: Sharon Plank, MD
role: PRINCIPAL_INVESTIGATOR
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