Amyotrophic Lateral SclerosisPhase 2NCT00243932
Clinical Trial of High Dose CoQ10 in ALS
A Phase 2 study testing coenzyme Q10 in people with Amyotrophic Lateral Sclerosis and Lou Gehrig's Disease, run by Columbia University at 19 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 21 to 85 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, Lou Gehrig's Disease
- What is being tested?
- coenzyme Q10
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 185 participants
- Where
- 19 sitesUnited States
- Study dates
- Apr 2005to Mar 2008
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 21 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
19 sites in 1 country.
19 sites in 1 country
United States19 sites
Brigham and Women's Hospital , Department of Neurology
Boston, Massachusetts, 02108
University of Vermont, Neurology Department
Burlington, Vermont, 05401
Northwestern University, Department of Neurology,
Chicago, Illinois, 60290
University of Chicago, Department of Neurology
Chicago, Illinois, 60292
Cleveland Clinic Foundation
Cleveland, Ohio, 44101
University of Colorado Health Sciences, Dept of Neurology
Denver, Colorado, 80221
University of Kansas Medical Center
Kansas City, Kansas, 64116
University of Kentucky, Dept of Neurology, College of Medicine
Lexington, Kentucky, 40201
University of Arkansas for Medical Sciences, Department of Neurology
Little Rock, Arkansas, 72201
Minneapolis Medical Research Foundation, ,
Minneapolis, Minnesota, 55421
Yale University School of Medicine, Department of Neurology
New Haven, Connecticut, 06501
Columbia Presbyterian Medical Center, The Neurological Institute
New York, New York, 10032
Drexel University, Dept of Neurology
Philadelphia, Pennsylvania, 19113
University of Texas, Health Science Center at San Antonio, Division of Neurology
San Antonio, Texas, 78201
California Pacific Medical Center
San Francisco, California, 94101
University of California at San Francisco
San Francisco, California, 94101
Baystate Medical Center, Division of Critical Care Research
Springfield, Massachusetts, 01101
Washington University in St. Louis School of Medicine, Department of Neurology
St Louis, Missouri, 63101
State University of New York Upstate Medical, Neurology Department
Syracuse, New York, 13201
About this study
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.
Read the 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.
- Being tested
- coenzyme Q10, Placebo
- Keywords
- amyotrophic lateral sclerosis, ALS, Lou Gehrig's disease, CoQ10, coenzyme Q10, antioxidants, free radicals, mitochondrial dysfunction
- Medical subject headings
- Amyotrophic Lateral Sclerosis, Mitochondrial Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
coenzyme Q10Drug
Also called Coenzyme Q 10, CoQ10
antioxidant and mitochondrial cofactor, given in capsules three times daily
PlaceboDrug
Placebo capsules, indistinguishable from CoQ10 capsules, given three times daily
| Study group | Role | What they receive |
|---|---|---|
| 2,700 mg CoQ10 | Experimental | Not described Receives: Drug: coenzyme Q10 |
| placebo | Placebo comparator | Not described Receives: Drug: Placebo |
| 1,800 mg CoQ10 | Experimental | Not described Receives: Drug: coenzyme Q10 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 9 months
Secondary outcomes (4)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Oct 2005 (estimated)
- Study startApr 2005
- Primary completionMar 2008
- Study completionMar 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- More trials run by Columbia UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 185 participants (actual)
Sponsor and investigators
- Lead sponsor
- Columbia University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Petra Kaufmann, MD, Principal investigator, Assistant Professor, Division of Neuromuscular Disease, Columbia University Medical Center (Clinical Principal Investigator)J. L. P. Thompson, Ph.D., Principal investigator, Director, Statistical Analysis Center, Department of Biostatistics, Mailman School of Public Health (Statistical Principal Investigator)Hiroshi Mitsumoto, Principal investigator, Wesley J. Howe Professor of Neurology at the New York Presbyterian Hospital/Columbia University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00243932
- Sponsor's protocol ID
- AAAA1536
- Other IDs
- R01NS048125
- Status last verified
- Apr 2024
- First submitted
- 24 Oct 2005
- Last updated
- 7 May 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
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.
PubMed 19743457
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Trial of High Dose CoQ10 in ALS
- ClinicalTrials.gov ID
- NCT00243932
- Acronym / sponsor ID
- AAAA1536
- Keywords
- amyotrophic lateral sclerosis, ALS, Lou Gehrig's disease, CoQ10, coenzyme Q10, antioxidants, free radicals, mitochondrial dysfunction
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 185 participants (actual)
- Lead sponsor
- Columbia UniversityUniversity, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS)
- Study start
- Apr 2005
- Primary completion
- Mar 2008 (actual)
- Study completion
- Mar 2008 (actual)
- First posted
- 25 Oct 2005 (estimated)
- Results
- Posted 17 Mar 2011
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| 2,700 mg CoQ10 | Experimental | Drug: coenzyme Q10 |
| placebo | Placebo comparator | Drug: Placebo |
| 1,800 mg CoQ10 | Experimental | Drug: coenzyme Q10 |
Who may qualify
- Ages
- 21 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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
Key exclusion criteria (12 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| coenzyme Q10Also: Coenzyme Q 10, CoQ10antioxidant and mitochondrial cofactor, given in capsules three times daily | Drug | 1,800 mg CoQ10, 2,700 mg CoQ10 |
| PlaceboPlacebo capsules, indistinguishable from CoQ10 capsules, given three times daily | Drug | placebo |
Primary outcome
Change in the ALS Functional Rating Scale-revised (ALSFRSr) Score.
Time frame: 9 months
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.
4 secondary outcomes
Change in Fatigue Severity Scale
Time frame: 9 months
Change in Forced Vital Capacity
Time frame: 9 months
Change in Short Form (SF)-36 Score (Physical)
Time frame: 9 months
Change in Short Form (SF)-36 Score (Mental)
Time frame: 9 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 May 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 19743457Kaufmann 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Petra Kaufmann, MD principal investigator
Assistant Professor, Division of Neuromuscular Disease, Columbia University Medical Center (Clinical Principal Investigator)
J. L. P. Thompson, Ph.D. principal investigator
Director, Statistical Analysis Center, Department of Biostatistics, Mailman School of Public Health (Statistical Principal Investigator)
Hiroshi Mitsumoto principal investigator
Wesley J. Howe Professor of Neurology at the New York Presbyterian Hospital/Columbia University Medical Center
19 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing coenzyme Q10 in people with Amyotrophic Lateral Sclerosis and Lou Gehrig's Disease, run by Columbia University at 19 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 7 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 17 Mar 2011). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in the ALS Functional Rating Scale-revised (ALSFRSr) Score. (time frame: 9 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00243932, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 May 2024.