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Charcot Marie Tooth DiseasePhase 1/2NCT00541164

Effects of Coenzyme Q10 on Charcot-Marie-Tooth Disease

A Phase 1/2 study testing Coenzyme Q10 in people with Charcot Marie Tooth Disease, run by Memorial Medical Center at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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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… Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Charcot Marie Tooth Disease
What is being tested?
Coenzyme Q10
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
23 participants
Where
1 siteUnited States
Study dates
Sep 2007to Jan 2013
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
Show all 10Show fewer
  • 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

Who cannot join

  • 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
Show all 10Show fewer
  • 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

Trial locations and contacts

1 site in 1 country.

United States1 site

  • John P Murtha Neuroscience and Pain Institute

    Johnstown, Pennsylvania, 15904

Other trials in United States

About this 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

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.

Read the 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.

Being tested
Coenzyme Q10
Keywords
CMT, Charcot-Marie Tooth, CoQ10, Coenzyme Q10
Medical subject headings
Charcot-Marie-Tooth Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Coenzyme Q10Drug

    Also called CoQ10, Ubiquinone

    300 mg CoQ10 twice a day for 48 weeks

  • Coenzyme Q10Dietary supplement

    Also called CoQ10, Ubiquinone

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

Study groups in NCT00541164
Study groupRoleWhat they receive
1Experimental300 mg CoQ10 chewable wafer twice a day
Receives: Drug: Coenzyme Q10
2Placebo comparatorChewable placebo wafer twice a day for 24 weeks with crossover to 300mg CoQ10 twice a day for weeks 24-48.
Receives: Dietary Supplement: Coenzyme Q10

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Changes in weakness, fatigue and pain in persons with Charcot-Marie-Tooth disease after supplementation with 600 mgs a day of Coenzyme Q10.

    Measured: 60 weeks

Secondary outcomes (3)

  1. Improvements in quality of life in subjects with CMT before and after CoQ10 supplementation.

    Measured: 60 weeks

  2. Measure blood serum levels of the oxidized and reduced forms of CoQ10.

    Measured: 60 weeks

  3. Measure liver function tests

    Measured: visits 1, 6, 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted10 Oct 2007 (estimated)
  2. Study startSep 2007
  3. Primary completionJan 2013
  4. Study completionJan 2013

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Supportive care
Allocation
Randomised
Intervention model
Crossover
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
23 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00541164
Sponsor's protocol ID
05-19
Status last verified
Jun 2010
First submitted
9 Oct 2007
Last updated
15 Jul 2013
Results posted
No
Data monitoring committee
No

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. doi: 10.1016/S0140-6736(04)15794-2.

    PubMed 15043967

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

    PubMed 12069110

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

    PubMed 12871093

  • 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.

    PubMed 16551570

  • 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.

    PubMed 12374491

  • 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.

    PubMed 11502903

  • 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.

    PubMed 12017500

  • 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.

    PubMed 8820103

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Coenzyme Q10 in people with Charcot Marie Tooth Disease, run by Memorial Medical Center at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 15 Jul 2013. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT00541164, which also lists 9 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jul 2013.

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