Clinical Trial of High Dose CoQ10 in ALS

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

Clinical Trial of High Dose CoQ10 in ALS

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00243932
• Other study ID #: AAAA1536
• Secondary ID: R01NS048125
• Status: Completed
• Phase: Phase 2
• Start date: April 2005
• Est. completion date: March 2008

Study information

• Verified date: April 2024
• Source: Columbia University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 185
• Est. completion date: March 2008
• Est. primary completion date: March 2008
• Accepts healthy volunteers: No
• Gender: All
• Age group: 21 Years to 85 Years

Eligibility

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

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Lou Gehrig's Disease
• Motor Neuron Disease
• Sclerosis

Intervention

Drug:
• coenzyme Q10
antioxidant and mitochondrial cofactor, given in capsules three times daily
• Placebo
Placebo capsules, indistinguishable from CoQ10 capsules, given three times daily

Locations

Country	Name	City	State
United States	Brigham and Women's Hospital , Department of Neurology	Boston	Massachusetts
United States	University of Vermont, Neurology Department	Burlington	Vermont
United States	Northwestern University, Department of Neurology,	Chicago	Illinois
United States	University of Chicago, Department of Neurology	Chicago	Illinois
United States	Cleveland Clinic Foundation	Cleveland	Ohio
United States	University of Colorado Health Sciences, Dept of Neurology	Denver	Colorado
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	University of Arkansas for Medical Sciences, Department of Neurology	Little Rock	Arkansas
United States	Minneapolis Medical Research Foundation, ,	Minneapolis	Minnesota
United States	Yale University School of Medicine, Department of Neurology	New Haven	Connecticut
United States	Columbia Presbyterian Medical Center, The Neurological Institute	New York	New York
United States	Drexel University, Dept of Neurology	Philadelphia	Pennsylvania
United States	Washington University in St. Louis School of Medicine, Department of Neurology	Saint Louis	Missouri
United States	University of Texas, Health Science Center at San Antonio, Division of Neurology	San Antonio	Texas
United States	California Pacific Medical Center	San Francisco	California
United States	University of California at San Francisco	San Francisco	California
United States	Baystate Medical Center, Division of Critical Care Research	Springfield	Massachusetts
United States	State University of New York Upstate Medical, Neurology Department	Syracuse	New York

Sponsors (2)

Lead Sponsor	Collaborator
Columbia University	National Institute of Neurological Disorders and Stroke (NINDS)

Country where clinical trial is conducted

United States, 

References & 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 — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Change in Fatigue Severity Scale	The change over 9 months in fatigue severity scale. A 9-item scale measuring the impact of fatigue. Scores range from 7 (strongly disagree)-63 (strongly agree) with higher scores indicating a worse outcome.	9 months
Secondary	Change in Forced Vital Capacity	The change over 9 months in forced vital capacity is the volume (liters) of gas that can be exhaled by maximum voluntary effort following deep inspiration. The best of three trials will be recorded. The result is recorded as percentage of predicted for age, height and weight.	9 months
Secondary	Change in Short Form (SF)-36 Score (Physical)	The change over 9 months in SF-36 score, which is a quality of life measure. Scores range from 0-100 with a higher score indicating a better outcome.	9 months
Secondary	Change in Short Form (SF)-36 Score (Mental)	The change over 9 months in SF-36 score, which is a quality of life measure. Scores range from 0-100 with a higher score indicating a better outcome.	9 months