Clinical Trial of High Dose CoQ10 in ALS

NCT ID: NCT00243932

Last Updated: 2024-05-07

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 185 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2005-04-30
• Study Completion Date: 2008-03-31

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.

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.

Conditions

Amyotrophic Lateral Sclerosis; Lou Gehrig's Disease

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

2,700 mg CoQ10

Group Type: EXPERIMENTAL

coenzyme Q10

Intervention Type: DRUG

antioxidant and mitochondrial cofactor, given in capsules three times daily

placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo capsules, indistinguishable from CoQ10 capsules, given three times daily

1,800 mg CoQ10

Group Type: EXPERIMENTAL

coenzyme Q10

Intervention Type: DRUG

antioxidant and mitochondrial cofactor, given in capsules three times daily

Interventions

coenzyme Q10

antioxidant and mitochondrial cofactor, given in capsules three times daily

Intervention Type: DRUG

Placebo

Placebo capsules, indistinguishable from CoQ10 capsules, given three times daily

Intervention Type: DRUG

Other Intervention Names

Coenzyme Q 10; CoQ10

Eligibility 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

• Minimum Eligible Age: 21 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Institute of Neurological Disorders and Stroke (NINDS)

NIH

Sponsor Role: collaborator

Columbia University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Petra Kaufmann, MD

Role: PRINCIPAL_INVESTIGATOR

Assistant Professor, Division of Neuromuscular Disease, Columbia University Medical Center (Clinical Principal Investigator)

J. L. P. Thompson, Ph.D.

Role: PRINCIPAL_INVESTIGATOR

Director, Statistical Analysis Center, Department of Biostatistics, Mailman School of Public Health (Statistical Principal Investigator)

Hiroshi Mitsumoto

Role: PRINCIPAL_INVESTIGATOR

Wesley J. Howe Professor of Neurology at the New York Presbyterian Hospital/Columbia University Medical Center

Locations

University of Arkansas for Medical Sciences, Department of Neurology

Little Rock, Arkansas, United States

California Pacific Medical Center

San Francisco, California, United States

University of California at San Francisco

San Francisco, California, United States

University of Colorado Health Sciences, Dept of Neurology

Denver, Colorado, United States

Yale University School of Medicine, Department of Neurology

New Haven, Connecticut, United States

Northwestern University, Department of Neurology,

Chicago, Illinois, United States

University of Chicago, Department of Neurology

Chicago, Illinois, United States

University of Kansas Medical Center

Kansas City, Kansas, United States

University of Kentucky, Dept of Neurology, College of Medicine

Lexington, Kentucky, United States

Brigham and Women's Hospital , Department of Neurology

Boston, Massachusetts, United States

Baystate Medical Center, Division of Critical Care Research

Springfield, Massachusetts, United States

Minneapolis Medical Research Foundation, ,

Minneapolis, Minnesota, United States

Washington University in St. Louis School of Medicine, Department of Neurology

St Louis, Missouri, United States

Columbia Presbyterian Medical Center, The Neurological Institute

New York, New York, United States

State University of New York Upstate Medical, Neurology Department

Syracuse, New York, United States

Cleveland Clinic Foundation

Cleveland, Ohio, United States

Drexel University, Dept of Neurology

Philadelphia, Pennsylvania, United States

University of Texas, Health Science Center at San Antonio, Division of Neurology

San Antonio, Texas, United States

University of Vermont, Neurology Department

Burlington, Vermont, United States

Countries

United States

References

Kaufmann P, Thompson JL, Levy G, Buchsbaum R, Shefner J, Krivickas LS, Katz J, Rollins Y, Barohn RJ, Jackson CE, Tiryaki E, Lomen-Hoerth C, Armon C, Tandan R, Rudnicki SA, Rezania K, Sufit R, Pestronk A, Novella SP, Heiman-Patterson T, Kasarskis EJ, Pioro EP, Montes J, Arbing R, Vecchio D, Barsdorf A, Mitsumoto H, Levin B; QALS Study Group. Phase II trial of CoQ10 for ALS finds insufficient evidence to justify phase III. Ann Neurol. 2009 Aug;66(2):235-44. doi: 10.1002/ana.21743.

Reference Type: RESULT

PMID: 19743457 (View on PubMed)

Other Identifiers

R01NS048125

Identifier Type: NIH

Identifier Source: secondary_id

View Link

AAAA1536

Identifier Type: -

Identifier Source: org_study_id