Clinical Trial of High Dose CoQ10 in ALS
This study has been completed.
Sponsor:
Columbia University
Collaborator:
Information provided by:
Columbia University
ClinicalTrials.gov Identifier:
NCT00243932
First received: October 24, 2005
Last updated: February 22, 2011
Last verified: February 2011
- Full Text View
- Tabular View
- Study Results
- Disclaimer
- How to Read a Study Record
Purpose
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 | Intervention | Phase |
|---|---|---|
|
Amyotrophic Lateral Sclerosis Lou Gehrig's Disease |
Drug: coenzyme Q10 Drug: Placebo |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Investigator) Primary Purpose: Treatment |
| Official Title: | Clinical Trial of High Dose CoQ10 in ALS |
Resource links provided by NLM:
Genetics Home Reference related topics:
amyotrophic lateral sclerosis
Drug Information available for:
Ubidecarenone
U.S. FDA Resources
Further study details as provided by Columbia University:
Primary Outcome Measures:
- Decline in the ALS Functional Rating Scale-revised (ALSFRSr) Score. [ Time Frame: 9 months ] [ Designated as safety issue: No ]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:
- 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 ] [ Designated as safety issue: No ]
| Enrollment: | 185 |
| Study Start Date: | April 2005 |
| Study Completion Date: | March 2008 |
| Primary Completion Date: | March 2008 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: 2,700 mg CoQ10 |
Drug: coenzyme Q10
antioxidant and mitochondrial cofactor, given in capsules three times daily
Other Names:
|
| 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:
|
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.
Eligibility| Ages Eligible for Study: | 21 Years to 85 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
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
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00243932
Locations
| 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
Investigators
| 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) |
More Information
Publications:
| Responsible Party: | Petra Kaufmann, MD, Associate Professor, Division of Neuromuscular Disease, Columbia University |
| ClinicalTrials.gov Identifier: | NCT00243932 History of Changes |
| Other Study ID Numbers: | AAAA1536, R01NS048125 |
| Study First Received: | October 24, 2005 |
| Results First Received: | March 29, 2010 |
| Last Updated: | February 22, 2011 |
| Health Authority: | United States: Food and Drug Administration United States: Federal Government |
Keywords provided by Columbia University:
|
amyotrophic lateral sclerosis ALS Lou Gehrig's disease CoQ10 |
coenzyme Q10 antioxidants free radicals mitochondrial dysfunction |
Additional relevant MeSH terms:
|
Amyotrophic Lateral Sclerosis Sclerosis Motor Neuron Disease Spinal Cord Diseases Central Nervous System Diseases Nervous System Diseases Neurodegenerative Diseases TDP-43 Proteinopathies Neuromuscular Diseases Proteostasis Deficiencies Metabolic Diseases |
Pathologic Processes Antioxidants Coenzyme Q10 Ubiquinone Molecular Mechanisms of Pharmacological Action Pharmacologic Actions Protective Agents Physiological Effects of Drugs Micronutrients Growth Substances Vitamins |
ClinicalTrials.gov processed this record on May 19, 2013