Clinical Trial of High Dose CoQ10 in ALS

Columbia University185 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

185

Conditions

Amyotrophic Lateral Sclerosis Lou Gehrig's Disease

Interventions

coenzyme Q10DRUG

antioxidant and mitochondrial cofactor, given in capsules three times daily

PlaceboDRUG

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

Eligibility

Sex: ALLMin age: 21 YearsMax age: 85 Years

Medical Language ↔ Plain English

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

Locations (19)

University of Arkansas for Medical Sciences, Department of Neurology

Little Rock, Arkansas, United States

Outcomes

Primary Outcomes

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.

Time frame: 9 months

Secondary Outcomes

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.

Time frame: 9 months

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.

Time frame: 9 months

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.

Time frame: 9 months

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.

Time frame: 9 months

Data from ClinicalTrials.gov

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