search
Back to results

Effects of Coenzyme Q10 on Charcot-Marie-Tooth Disease

Primary Purpose

Charcot Marie Tooth Disease

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
Coenzyme Q10
Coenzyme Q10
Sponsored by
Memorial Medical Center
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional supportive care trial for Charcot Marie Tooth Disease focused on measuring CMT, Charcot-Marie Tooth, CoQ10, Coenzyme Q10

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • John P Murtha Neuroscience and Pain Institute

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

1

2

Arm Description

300 mg CoQ10 chewable wafer twice a day

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

Outcomes

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.

Secondary Outcome Measures

Improvements in quality of life in subjects with CMT before and after CoQ10 supplementation.
Measure blood serum levels of the oxidized and reduced forms of CoQ10.
Measure liver function tests

Full Information

First Posted
October 9, 2007
Last Updated
July 11, 2013
Sponsor
Memorial Medical Center
Collaborators
United States Department of Defense
search

1. Study Identification

Unique Protocol Identification Number
NCT00541164
Brief Title
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

2. Study Status

Record Verification Date
June 2010
Overall Recruitment Status
Completed
Study Start Date
September 2007 (undefined)
Primary Completion Date
January 2013 (Actual)
Study Completion Date
January 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Memorial Medical Center
Collaborators
United States Department of Defense

4. Oversight

Data Monitoring Committee
No

5. 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Charcot Marie Tooth Disease
Keywords
CMT, Charcot-Marie Tooth, CoQ10, Coenzyme Q10

7. Study Design

Primary Purpose
Supportive Care
Study Phase
Phase 1, Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
23 (Actual)

8. Arms, Groups, and Interventions

Arm Title
1
Arm Type
Experimental
Arm Description
300 mg CoQ10 chewable wafer twice a day
Arm Title
2
Arm Type
Placebo Comparator
Arm Description
Chewable placebo wafer twice a day for 24 weeks with crossover to 300mg CoQ10 twice a day for weeks 24-48.
Intervention Type
Drug
Intervention Name(s)
Coenzyme Q10
Other Intervention Name(s)
CoQ10, Ubiquinone
Intervention Description
300 mg CoQ10 twice a day for 48 weeks
Intervention Type
Dietary Supplement
Intervention Name(s)
Coenzyme Q10
Other Intervention Name(s)
CoQ10, Ubiquinone
Intervention Description
300mg CoQ10 twice a day for 24 weeks beginning at week 24 of the study
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
Improvements in quality of life in subjects with CMT before and after CoQ10 supplementation.
Time Frame
60 weeks
Title
Measure blood serum levels of the oxidized and reduced forms of CoQ10.
Time Frame
60 weeks
Title
Measure liver function tests
Time Frame
visits 1, 6, 12

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Sharon Plank, MD
Organizational Affiliation
John P. Murtha Neuroscience and Pain Institute
Official's Role
Principal Investigator
Facility Information:
Facility Name
John P Murtha Neuroscience and Pain Institute
City
Johnstown
State/Province
Pennsylvania
ZIP/Postal Code
15904
Country
United States

12. IPD Sharing Statement

Citations:
Citation
Hendler SS, Rorvik D, eds. PDR for Nutritional Supplements. Montvale, NJ: Thomson PDR; 2001:105-106.
Results Reference
background
PubMed Identifier
15043967
Citation
Chaudhuri A, Behan PO. Fatigue in neurological disorders. Lancet. 2004 Mar 20;363(9413):978-88. doi: 10.1016/S0140-6736(04)15794-2.
Results Reference
background
PubMed Identifier
12069110
Citation
Beal MF. Coenzyme Q10 as a possible treatment for neurodegenerative diseases. Free Radic Res. 2002 Apr;36(4):455-60. doi: 10.1080/10715760290021315.
Results Reference
background
PubMed Identifier
12871093
Citation
Shults CW. Coenzyme Q10 in neurodegenerative diseases. Curr Med Chem. 2003 Oct;10(19):1917-21. doi: 10.2174/0929867033456882.
Results Reference
background
PubMed Identifier
16551570
Citation
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.
Results Reference
background
PubMed Identifier
12374491
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. doi: 10.1001/archneur.59.10.1541.
Results Reference
background
PubMed Identifier
11502903
Citation
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.
Results Reference
background
PubMed Identifier
12017500
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. No abstract available.
Results Reference
background
PubMed Identifier
8820103
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.
Results Reference
background

Learn more about this trial

Effects of Coenzyme Q10 on Charcot-Marie-Tooth Disease

We'll reach out to this number within 24 hrs