Idebenone treatment fails to slow cognitive decline in Alzheimer’s disease
Abstract
OBJECTIVE: To determine the effect of idebenone on the rate of decline in Alzheimer's disease (AD). METHODS: A 1-year, multicenter, double-blind, placebo-controlled, randomized trial was conducted. Subjects were over age 50 with a diagnosis of probable AD and had Mini-Mental State Examination (MMSE) scores between 12 and 25. Subjects were treated with idebenone 120, 240, or 360 mg tid, each of which was compared with placebo. Primary outcome measures were the Alzheimer's Disease Assessment Scale-Cognitive Subcomponent (ADAS-Cog) and a Clinical Global Impression of Change (CGIC). Secondary outcome measures included measurements of activities of daily living, the Behavioral Pathology in Alzheimer's Disease Rating Scale, and the MMSE. RESULTS: Five hundred thirty-six subjects were enrolled and randomized to the four groups. Except for a slight difference in age, there were no differences in patient characteristics at baseline. For the primary outcome measures, there were no significant overall differences between the treatment groups in the prespecified four-group design. In an exploratory two-group analysis comparing all three treated groups combined with placebo, drug-treated patients performed better on the ADAS-Cog in both the intent-to-treat (ITT) and completers analyses. There were no differences in the CGIC scores for the ITT or completers analyses in either the four-group or the two-group analyses. There were no overall differences on any of the secondary outcome measures in any of the analyses. CONCLUSION: Idebenone failed to slow cognitive decline in AD that was of sufficient magnitude to be clinically significant.
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Another large trial showed no benefits of idebenone.
“These results are controversial since another large trial showed no benefits170.”
From Mitochondrial Approaches for Neuroprotection (2008) · cites this paper as [170]
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- Authors
- Thal, L. J., Grundman, M., Berg, J., Ernstrom, K., Margolin, R., Pfeiffer, E., Weiner, M. F., Zamrini, E., & Thomas, R. G.
- Affiliations
- University of South Florida (US)
- Venue
- Neurology
- Published
- 2003-12-09
- Volume
- 61
- Issue
- 11
- Pages
- 1498-1502
- ISSN
- 0028-3878 (ISSN-L)
- 1526-632X
- OpenAlex
- W2098790447
- Cited by
- 156
- References
- 29
- Citations by year
- 2026: 2 · 2025: 7 · 2024: 3 · 2023: 9 · 2022: 8 · 2021: 8 · 2020: 9 · 2019: 7 · 2018: 4 · 2017: 1 · 2016: 6 · 2015: 8 · 2014: 9 · 2013: 7 · 2012: 9
- FWCI
- 2.68
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Neurology (publishedVersion)
- PubMed (publishedVersion)
- Funders
- National Institute on Aging (AG 10483, U19AG010483)
- Topics
- Health Sciences › Medicine › Physiology › Alzheimer's disease research and treatments
- Health Sciences › Medicine › Psychiatry and Mental health › Dementia and Cognitive Impairment Research
- Life Sciences › Neuroscience › Neurology › Neurological Disorders and Treatments
- Keywords
- idebenone, Alzheimer's disease, cognitive decline, treatment efficacy, ADAS-Cog, Clinical Global Impression of Change, behavioral pathology, activities of daily living
- MeSH terms
- Aged, Alzheimer Disease, Cognition Disorders, Double-Blind Method, Female, Humans, Male, Middle Aged, Ubiquinone, Benzoquinones, Treatment Failure, Disease Progression
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