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Ide3.0

Idebenone Research

Mostly mechanism / observationalModerate safety

4 peer-reviewed studies

What the evidence says

Mostly mechanism / observational

Most Idebenone studies are mechanism or observational rather than RCTs that measure a clinical effect — keep findings provisional.

Most evidence is from medium-quality randomised trials published 1998–2011 with a typical study size of 85 participants.

Based on 4 studies · 4 RCTs · 675 total participants

Confidence

Moderate

By outcome

Vision & eye healthApproved (EU) for Leber hereditary optic neuropathy, but the pivotal trial missed its primary endpoint · 6+ months
Too few graded studies1 study

Older research base

Newest study from 2011

199820042011

1RCTn=85 · small study2011

The primary end-point did not reach statistical significance in the intention-to-treat population.

Klopstock T, Yu-Wai-Man P, Dimitriadis K, et al. · Brain (2011)

24-week multicentre double-blind RCT in 85 LHON patients; idebenone 900 mg/day
Primary endpoint (best recovery in visual acuity) did NOT reach statistical significance
Only a post-hoc subgroup with discordant baseline visual acuity showed significant secondary-endpoint differences

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2RCTn=70 · small study2010

Idebenone did not significantly alter neurological function in Friedreich ataxia during the 6-month study.

Lynch DR, Perlman SL, Meier T. · Arch Neurol (2010)

70 pediatric patients randomized to two idebenone dose tiers or placebo for 6 months
Difference in ICARS and FARS scores between idebenone and placebo was NOT statistically significant
No demonstrated neurological benefit over the trial period

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3RCTn=70 · small study2011

Idebenone did not decrease LV hypertrophy or improve cardiac function in subjects with FRDA.

Lagedrost SJ, Sutton MS, Cohen MS, et al. · Am Heart J (2011)

70 pediatric Friedreich ataxia subjects treated with idebenone or placebo for 6 months
Left-ventricular mass, wall thickness, ejection fraction and ECG parameters were not significantly improved
No evidence of cardiac benefit over 6 months

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4RCTn=450 · medium study1998

Idebenone showed statistically significant dose-dependent improvement on the ADAS in mild-to-moderate Alzheimer's during the placebo-controlled year.

Gutzmann H, Hadler D. · J Neural Transm Suppl (1998)

450 mild-to-moderate Alzheimer patients; idebenone 90-120 mg three times daily over 2 years
Dose-dependent improvement on ADAS-Total and secondary measures during the placebo-controlled first year
Older positive trial — note a later large US trial of idebenone in Alzheimer's was negative

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View in Research Library

Idebenone Research

Mostly mechanism / observational

A randomized placebo-controlled trial of idebenone in Leber's hereditary optic neuropathy.

A phase 3, double-blind, placebo-controlled trial of idebenone in Friedreich ataxia.

Idebenone in Friedreich ataxia cardiomyopathy - results from a 6-month phase III study (IONIA).

Sustained efficacy and safety of idebenone in the treatment of Alzheimer's disease: update on a 2-year double-blind multicentre study.