Clinical Trial of High Dose CoQ10 in ALS

NCT00243932 | Completed Phase 2 Results DMC

• 2 other identifiers: AAAA1536R01NS048125
• Study Type: interventional
• Target: 185
• Locations: 1 country
• Sites: 19

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.

Conditions

Amyotrophic Lateral Sclerosis; Lou Gehrig's Disease

Keywords

amyotrophic lateral sclerosis; ALS; Lou Gehrig's disease; CoQ10; coenzyme Q10; antioxidants; free radicals; mitochondrial dysfunction

Outcome Measures

Primary Outcomes (1)

• Change in the ALS Functional Rating Scale-revised (ALSFRSr) Score.

  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.

  9 months

Secondary Outcomes (4)

• Change in Fatigue Severity Scale

  9 months

• Change in Forced Vital Capacity

  9 months

• Change in Short Form (SF)-36 Score (Physical)

  9 months

• Change in Short Form (SF)-36 Score (Mental)

  9 months

Study Arms (3)

2,700 mg CoQ10

EXPERIMENTAL

Drug: coenzyme Q10

placebo

PLACEBO COMPARATOR

Drug: Placebo

1,800 mg CoQ10

EXPERIMENTAL

Drug: coenzyme Q10

Interventions

coenzyme Q10 DRUG

antioxidant and mitochondrial cofactor, given in capsules three times daily

Also known as: Coenzyme Q 10, CoQ10

1,800 mg CoQ10; 2,700 mg CoQ10

Placebo DRUG

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

placebo

Eligibility Criteria

• Age: 21 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Columbia University: lead
• National Institute of Neurological Disorders and Stroke (NINDS): collaborator

Study Sites (19)

University of Arkansas for Medical Sciences, Department of Neurology

Little Rock, Arkansas, 72201, United States

Location

California Pacific Medical Center

San Francisco, California, 94101, United States

Location

University of California at San Francisco

San Francisco, California, 94101, United States

Location

University of Colorado Health Sciences, Dept of Neurology

Denver, Colorado, 80221, United States

Location

Yale University School of Medicine, Department of Neurology

New Haven, Connecticut, 06501, United States

Location

Northwestern University, Department of Neurology,

Chicago, Illinois, 60290, United States

Location

University of Chicago, Department of Neurology

Chicago, Illinois, 60292, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 64116, United States

Location

University of Kentucky, Dept of Neurology, College of Medicine

Lexington, Kentucky, 40201, United States

Location

Brigham and Women's Hospital , Department of Neurology

Boston, Massachusetts, 02108, United States

Location

Baystate Medical Center, Division of Critical Care Research

Springfield, Massachusetts, 01101, United States

Location

Minneapolis Medical Research Foundation, ,

Minneapolis, Minnesota, 55421, United States

Location

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

St Louis, Missouri, 63101, United States

Location

Columbia Presbyterian Medical Center, The Neurological Institute

New York, New York, 10032, United States

Location

State University of New York Upstate Medical, Neurology Department

Syracuse, New York, 13201, United States

Location

Cleveland Clinic Foundation

Cleveland, Ohio, 44101, United States

Location

Drexel University, Dept of Neurology

Philadelphia, Pennsylvania, 19113, United States

Location

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

San Antonio, Texas, 78201, United States

Location

University of Vermont, Neurology Department

Burlington, Vermont, 05401, United States

Location

Related Publications (1)

• 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.

  PMID: 19743457 RESULT

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis; Mitochondrial Diseases

Interventions

coenzyme Q10

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Hiroshi Mitsumoto, MD
• Organization: Columbia University

hm264@cumc.columbia.edu

2123052940

Study Officials

• 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

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 24, 2005
• First Posted: October 25, 2005
• Study Start: April 1, 2005
• Primary Completion: March 1, 2008
• Study Completion: March 1, 2008
• Last Updated: May 7, 2024
• Results First Posted: March 17, 2011

Record last verified: 2024-04

Locations

US (19)