Clinical Trial of High Dose CoQ10 in ALS
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| ClinicalTrials.gov Identifier: NCT00243932 |
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Recruitment Status :
Completed
First Posted : October 25, 2005
Results First Posted : March 17, 2011
Last Update Posted : February 28, 2014
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Amyotrophic Lateral Sclerosis Lou Gehrig's Disease | Drug: coenzyme Q10 Drug: Placebo | Phase 2 |
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 (Clinical Trial) |
| Actual Enrollment : | 185 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Double (Participant, Investigator) |
| Primary Purpose: | Treatment |
| Official Title: | Clinical Trial of High Dose CoQ10 in ALS |
| Study Start Date : | April 2005 |
| Actual Primary Completion Date : | March 2008 |
| Actual Study Completion Date : | March 2008 |
| Arm | Intervention/treatment |
|---|---|
| Experimental: 2,700 mg CoQ10 |
Drug: coenzyme Q10
antioxidant and mitochondrial cofactor, given in capsules three times daily
Other Names:
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| Placebo Comparator: placebo |
Drug: Placebo
Placebo capsules, indistinguishable from CoQ10 capsules, given three times daily |
| Experimental: 1,800 mg CoQ10 |
Drug: coenzyme Q10
antioxidant and mitochondrial cofactor, given in capsules three times daily
Other Names:
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- 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.
- 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 ]
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| Ages Eligible for Study: | 21 Years to 85 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00243932
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| Principal Investigator: | 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 |
| Responsible Party: | Hiroshi Mitsumoto, Wesley J. Howe Professor of Neurology at the New York Presbyterian Hospital/Columbia University Medical Center, Columbia University |
| ClinicalTrials.gov Identifier: | NCT00243932 |
| Other Study ID Numbers: |
AAAA1536 R01NS048125 ( U.S. NIH Grant/Contract ) |
| First Posted: | October 25, 2005 Key Record Dates |
| Results First Posted: | March 17, 2011 |
| Last Update Posted: | February 28, 2014 |
| Last Verified: | February 2013 |
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amyotrophic lateral sclerosis ALS Lou Gehrig's disease CoQ10 |
coenzyme Q10 antioxidants free radicals mitochondrial dysfunction |
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Motor Neuron Disease Amyotrophic Lateral Sclerosis Sclerosis Pathologic Processes Neurodegenerative Diseases Nervous System Diseases Neuromuscular Diseases Spinal Cord Diseases Central Nervous System Diseases |
TDP-43 Proteinopathies Proteostasis Deficiencies Metabolic Diseases Ubiquinone Coenzyme Q10 Micronutrients Physiological Effects of Drugs Vitamins |