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Clinical Trial of High Dose CoQ10 in ALS

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00243932
Recruitment Status : Completed
First Posted : October 25, 2005
Results First Posted : March 17, 2011
Last Update Posted : February 28, 2014
National Institute of Neurological Disorders and Stroke (NINDS)
Information provided by (Responsible Party):
Hiroshi Mitsumoto, Columbia University

Brief Summary:
The purpose of this study is to determine the efficacy and preferred dose of CoQ10 in individuals with ALS for a possible future phase III study.

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Lou Gehrig's Disease Drug: coenzyme Q10 Drug: Placebo Phase 2

Detailed Description:

Amyotrophic lateral sclerosis (ALS) is a progressive and devastating neurodegenerative disorder. Available treatment for ALS remains scarce. Oxidative stress and mitochondrial dysfunction have been implicated in the pathophysiology of ALS. Oxidative stress refers to the effects of cell-damaging reactive oxygen species, also known as free radicals. Oxidative stress is thought to contribute to nerve cell loss in ALS. Mitochondria are organelles within each cell that are sometimes called "powerhouses of the cell" because cellular energy metabolism is located within the mitochondria.

Coenzyme Q10 (CoQ10), a mitochondrial cofactor known for its antioxidant properties, has prolonged survival in the mouse model of ALS and has slowed functional decline in another neurodegenerative disorder, Parkinson's disease. The goals of this double-blind, placebo-controlled, two-dose comparison phase II study are to obtain preliminary efficacy data and to select the preferred dose for a larger phase III study.

Participants were randomly assigned to CoQ10 (at two different dose levels) or placebo in the first stage, then the 2,700 mg dose was selected in the second stage. Duration of the trial was 9 months with a total of 7 visits.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 185 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: Clinical Trial of High Dose CoQ10 in ALS
Study Start Date : April 2005
Actual Primary Completion Date : March 2008
Actual Study Completion Date : March 2008

Arm Intervention/treatment
Experimental: 2,700 mg CoQ10 Drug: coenzyme Q10
antioxidant and mitochondrial cofactor, given in capsules three times daily
Other Names:
  • Coenzyme Q 10
  • CoQ10

Placebo Comparator: placebo Drug: Placebo
Placebo capsules, indistinguishable from CoQ10 capsules, given three times daily

Experimental: 1,800 mg CoQ10 Drug: coenzyme Q10
antioxidant and mitochondrial cofactor, given in capsules three times daily
Other Names:
  • Coenzyme Q 10
  • CoQ10

Primary Outcome Measures :
  1. Change in the ALS Functional Rating Scale-revised (ALSFRSr) Score. [ Time Frame: 9 months ]
    The ALSFRSr, a questionnaire-based scale assessing daily living function ranging from 48 (best score) to 0 (worst), was administered to the patient, or to a proxy if the patient could not communicate effectively. Decline was defined as ALSFRSr at baseline minus ALSFRSr at month 9. Thus a positive value indicates worsening.

Secondary Outcome Measures :
  1. The Change Over 9 Months in Forced Vital Capacity; Fatigue Severity Scale; Short Form-36; and 8OH2dG (a Biomarker of Oxidative Stress Measured in a Blood Sample). [ Time Frame: 9 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   21 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Clinical diagnosis of definite, probable, or laboratory-supported probable ALS
  • Negative pregnancy test for women of childbearing age and adequate birth control measures
  • Subjects must be able and willing to give informed consent and must be capable of complying with the trial procedures
  • Forced Vital Capacity (FVC) >/= 60% of predicted
  • Age 21 to 85 years, inclusive
  • Disease duration of less than 5 years
  • Subjects may take riluzole (without change in dose for more than 30 days before enrollment)
  • Patients who have taken CoQ10 in the past will be eligible if they stop at least 30 days before enrollment
  • Patients who have taken vitamin E in the past will be eligible if they stop at least 14 days before enrollment

Exclusion Criteria:

  • Dependency on mechanical ventilation (non-invasive ventilation > 23 hours)
  • Severe and unstable concomitant medical or psychiatric illness
  • Insufficiently controlled diabetes mellitus
  • Concomitant warfarin therapy
  • Women who are breast feeding or have a high likelihood of pregnancy
  • Significant hepatic dysfunction
  • Forced Vital Capacity (FVC) less than 60%
  • Exposure to CoQ10 within 30 days of enrollment
  • Exposure to other experimental medications within 30 days of enrollment
  • Exposure to vitamin E within 14 days of enrollment
  • Sensitivity to color additive FD&C Yellow No. 5
  • Sensitivity to aspirin

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00243932

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United States, Arkansas
University of Arkansas for Medical Sciences, Department of Neurology
Little Rock, Arkansas, United States, 72201
United States, California
California Pacific Medical Center
San Francisco, California, United States, 94101
University of California at San Francisco
San Francisco, California, United States, 94101
United States, Colorado
University of Colorado Health Sciences, Dept of Neurology
Denver, Colorado, United States, 80221
United States, Connecticut
Yale University School of Medicine, Department of Neurology
New Haven, Connecticut, United States, 06501
United States, Illinois
Northwestern University, Department of Neurology,
Chicago, Illinois, United States, 60290
University of Chicago, Department of Neurology
Chicago, Illinois, United States, 60292
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 64116
United States, Kentucky
University of Kentucky, Dept of Neurology, College of Medicine
Lexington, Kentucky, United States, 40201
United States, Massachusetts
Brigham and Women's Hospital , Department of Neurology
Boston, Massachusetts, United States, 02108
Baystate Medical Center, Division of Critical Care Research
Springfield,, Massachusetts, United States, 01101
United States, Minnesota
Minneapolis Medical Research Foundation, ,
Minneapolis, Minnesota, United States, 55421
United States, Missouri
Washington University in St. Louis School of Medicine, Department of Neurology
St. Louis, Missouri, United States, 63101
United States, New York
Columbia Presbyterian Medical Center, The Neurological Institute
New York, New York, United States, 10032
State University of New York Upstate Medical, Neurology Department
Syracuse, New York, United States, 13201
United States, Ohio
Cleveland Clinic Foundation
Cleveland, Ohio, United States, 44101
United States, Pennsylvania
Drexel University, Dept of Neurology
Philadelphia, Pennsylvania, United States, 19113
United States, Texas
University of Texas, Health Science Center at San Antonio, Division of Neurology
San Antonio, Texas, United States, 78201
United States, Vermont
University of Vermont, Neurology Department
Burlington, Vermont, United States, 05401
Sponsors and Collaborators
Columbia University
National Institute of Neurological Disorders and Stroke (NINDS)
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Principal Investigator: Petra Kaufmann, MD Assistant Professor, Division of Neuromuscular Disease, Columbia University Medical Center (Clinical Principal Investigator)
Principal Investigator: J. L. P. Thompson, Ph.D. Director, Statistical Analysis Center, Department of Biostatistics, Mailman School of Public Health (Statistical Principal Investigator)
Principal Investigator: Hiroshi Mitsumoto Wesley J. Howe Professor of Neurology at the New York Presbyterian Hospital/Columbia University Medical Center

Publications of Results:
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Responsible Party: Hiroshi Mitsumoto, Wesley J. Howe Professor of Neurology at the New York Presbyterian Hospital/Columbia University Medical Center, Columbia University Identifier: NCT00243932     History of Changes
Other Study ID Numbers: AAAA1536
R01NS048125 ( U.S. NIH Grant/Contract )
First Posted: October 25, 2005    Key Record Dates
Results First Posted: March 17, 2011
Last Update Posted: February 28, 2014
Last Verified: February 2013
Keywords provided by Hiroshi Mitsumoto, Columbia University:
amyotrophic lateral sclerosis
Lou Gehrig's disease
coenzyme Q10
free radicals
mitochondrial dysfunction
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Coenzyme Q10
Physiological Effects of Drugs
Growth Substances