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Clinical Trial of High Dose CoQ10 in ALS

Primary Purpose

Amyotrophic Lateral Sclerosis, Lou Gehrig's Disease

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
coenzyme Q10
Placebo
Sponsored by
Columbia University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring amyotrophic lateral sclerosis, ALS, Lou Gehrig's disease, CoQ10, coenzyme Q10, antioxidants, free radicals, mitochondrial dysfunction

Eligibility Criteria

21 Years - 85 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University of Arkansas for Medical Sciences, Department of Neurology
  • California Pacific Medical Center
  • University of California at San Francisco
  • University of Colorado Health Sciences, Dept of Neurology
  • Yale University School of Medicine, Department of Neurology
  • Northwestern University, Department of Neurology,
  • University of Chicago, Department of Neurology
  • University of Kansas Medical Center
  • University of Kentucky, Dept of Neurology, College of Medicine
  • Brigham and Women's Hospital , Department of Neurology
  • Baystate Medical Center, Division of Critical Care Research
  • Minneapolis Medical Research Foundation, ,
  • Washington University in St. Louis School of Medicine, Department of Neurology
  • Columbia Presbyterian Medical Center, The Neurological Institute
  • State University of New York Upstate Medical, Neurology Department
  • Cleveland Clinic Foundation
  • Drexel University, Dept of Neurology
  • University of Texas, Health Science Center at San Antonio, Division of Neurology
  • University of Vermont, Neurology Department

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Placebo Comparator

Experimental

Arm Label

2,700 mg CoQ10

placebo

1,800 mg CoQ10

Arm Description

Outcomes

Primary Outcome Measures

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.

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).

Full Information

First Posted
October 24, 2005
Last Updated
January 28, 2014
Sponsor
Columbia University
Collaborators
National Institute of Neurological Disorders and Stroke (NINDS)
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1. Study Identification

Unique Protocol Identification Number
NCT00243932
Brief Title
Clinical Trial of High Dose CoQ10 in ALS
Official Title
Clinical Trial of High Dose CoQ10 in ALS
Study Type
Interventional

2. Study Status

Record Verification Date
February 2013
Overall Recruitment Status
Completed
Study Start Date
April 2005 (undefined)
Primary Completion Date
March 2008 (Actual)
Study Completion Date
March 2008 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Columbia University
Collaborators
National Institute of Neurological Disorders and Stroke (NINDS)

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis, Lou Gehrig's Disease
Keywords
amyotrophic lateral sclerosis, ALS, Lou Gehrig's disease, CoQ10, coenzyme Q10, antioxidants, free radicals, mitochondrial dysfunction

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigator
Allocation
Randomized
Enrollment
185 (Actual)

8. Arms, Groups, and Interventions

Arm Title
2,700 mg CoQ10
Arm Type
Experimental
Arm Title
placebo
Arm Type
Placebo Comparator
Arm Title
1,800 mg CoQ10
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
coenzyme Q10
Other Intervention Name(s)
Coenzyme Q 10, CoQ10
Intervention Description
antioxidant and mitochondrial cofactor, given in capsules three times daily
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo capsules, indistinguishable from CoQ10 capsules, given three times daily
Primary Outcome Measure Information:
Title
Change in the ALS Functional Rating Scale-revised (ALSFRSr) Score.
Description
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 Outcome Measure Information:
Title
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
21 Years
Maximum Age & Unit of Time
85 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Petra Kaufmann, MD
Organizational Affiliation
Assistant Professor, Division of Neuromuscular Disease, Columbia University Medical Center (Clinical Principal Investigator)
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
J. L. P. Thompson, Ph.D.
Organizational Affiliation
Director, Statistical Analysis Center, Department of Biostatistics, Mailman School of Public Health (Statistical Principal Investigator)
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Hiroshi Mitsumoto
Organizational Affiliation
Wesley J. Howe Professor of Neurology at the New York Presbyterian Hospital/Columbia University Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
University of Arkansas for Medical Sciences, Department of Neurology
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72201
Country
United States
Facility Name
California Pacific Medical Center
City
San Francisco
State/Province
California
ZIP/Postal Code
94101
Country
United States
Facility Name
University of California at San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94101
Country
United States
Facility Name
University of Colorado Health Sciences, Dept of Neurology
City
Denver
State/Province
Colorado
ZIP/Postal Code
80221
Country
United States
Facility Name
Yale University School of Medicine, Department of Neurology
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06501
Country
United States
Facility Name
Northwestern University, Department of Neurology,
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60290
Country
United States
Facility Name
University of Chicago, Department of Neurology
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60292
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
64116
Country
United States
Facility Name
University of Kentucky, Dept of Neurology, College of Medicine
City
Lexington
State/Province
Kentucky
ZIP/Postal Code
40201
Country
United States
Facility Name
Brigham and Women's Hospital , Department of Neurology
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02108
Country
United States
Facility Name
Baystate Medical Center, Division of Critical Care Research
City
Springfield,
State/Province
Massachusetts
ZIP/Postal Code
01101
Country
United States
Facility Name
Minneapolis Medical Research Foundation, ,
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55421
Country
United States
Facility Name
Washington University in St. Louis School of Medicine, Department of Neurology
City
St. Louis
State/Province
Missouri
ZIP/Postal Code
63101
Country
United States
Facility Name
Columbia Presbyterian Medical Center, The Neurological Institute
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
State University of New York Upstate Medical, Neurology Department
City
Syracuse
State/Province
New York
ZIP/Postal Code
13201
Country
United States
Facility Name
Cleveland Clinic Foundation
City
Cleveland
State/Province
Ohio
ZIP/Postal Code
44101
Country
United States
Facility Name
Drexel University, Dept of Neurology
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19113
Country
United States
Facility Name
University of Texas, Health Science Center at San Antonio, Division of Neurology
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78201
Country
United States
Facility Name
University of Vermont, Neurology Department
City
Burlington
State/Province
Vermont
ZIP/Postal Code
05401
Country
United States

12. IPD Sharing Statement

Citations:
PubMed Identifier
19743457
Citation
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.
Results Reference
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Clinical Trial of High Dose CoQ10 in ALS

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